Temsirolimus Adventitial Delivery to Improve ANGioplasty And/or Atherectomy Revascularization Outcomes Below the Knee: a Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Effect of Temsirolimus Perivascular Injection 0.1 Mg/mL on the Incidence of Ischemia-Driven Major Amputation, Clinically Driven Target Lesion Revascularization, and Clinically Relevant Target Lesion Occlusion After Revascularization of Lesions Below the Knee in Patients with Symptomatic Rutherford 3-5 Peripheral Artery Disease

A multicenter, randomized, double-blind, placebo-controlled trial to evaluate the effect of Temsirolimus Perivascular Injection 0.1 mg/mL on the incidence of ischemia-driven major amputation, clinically driven target lesion revascularization, and clinically relevant target lesion occlusion after revascularization of lesions below the knee in patients with symptomatic Rutherford 3-5 peripheral artery disease. The primary safety endpoint will be gathered at 1-month post-index procedure. The primary efficacy endpoint will be gathered at 6 months post-index procedure. Participants will be followed for up to 5 years post-index procedure.

Start Date30 Apr 2025

Sponsor / Collaborator

Mercator MedSystems, Inc.

NCT04199026

/ RecruitingNot ApplicableIIT

Pilot Trial of an Implantable Microdevice for In Vivo Drug Sensitivity Testing in Patients With Sarcomas

This early phase I trial studies the side effects of implanting and removing a microdevice in patients with sarcomas that have spread to other places in the body (metastatic) or have come back (recurrent). Microdevices are rice-sized devices that are implanted into tumor tissue and are loaded with 10 different drugs that are delivered at very small doses, or "microdoses," which may only affect a very small, local area inside the tumor. The purpose of this study is to determine which drugs delivered in the microdevice affect tumor tissue in patients with sarcomas.

Start Date25 Feb 2025

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

[+1]

NCT05773326

/ RecruitingEarly Phase 1IIT

A Phase 0, Single-center, Open-label, Dose-escalating Trial Using Super-selective Intra-arterial Infusion of a Single Dose of Temsirolimus for the Treatment of Recurrent High-grade Glioma

This is a single-center, open-label, dose-escalating Phase 0 trial that will enroll participants with a confirmed diagnosed recurrent high-grade glioma (grade 3 or 4 per WHO criteria) targeting the mTOR pathway. Eligible participants will be administered a single infusion of temsirolimus through super-selective intra-arterial infusion or intravenous infusion. Participants will receive the study drug administration on the same day as the planned surgical resection of the tumor.

Start Date15 May 2023

Sponsor / Collaborator

St. Joseph's Medical Center, Inc.

[+1]

100 Clinical Results associated with Temsirolimus

100 Translational Medicine associated with Temsirolimus

100 Patents (Medical) associated with Temsirolimus

1,606

Literatures (Medical) associated with Temsirolimus

31 Dec 2025·ANNALS OF MEDICINE

A novel glycogene-related signature for prognostic prediction and immune microenvironment assessment in kidney renal clear cell carcinoma

Article

Author: Zhao, Danni ; Cui, Hanxiao ; Zhao, Xuyan ; Ren, Xueting ; Kang, Huafeng ; Liu, Peinan ; Li, Zihao ; Lin, Shuai ; Zhou, Mingjing

BACKGROUND:

Kidney Renal Clear Cell Carcinoma (KIRC) is a prevalent urinary malignancies worldwide. Glycosylation is a key post-translational modification that is essential in cancer progression. However, its relationship with prognosis, tumour microenvironment (TME), and treatment response in KIRC remains unclear.

METHOD:

Expression profiles and clinical data were retrieved from The Cancer Genome Atlas and Gene Expression Omnibus databases. Consensus clustering, Cox regression, and LASSO regression analyses were conducted to develop an optimal glycogene-related signature. The prognostic relevance of this molecular signature was rigorously analyzed, along with its connections to tumour microenvironment (TME), tumour mutation burden, immune checkpoint activity, cancer-immunity cycle regulation, immunomodulatory gene expression patterns, and therapeutic response profiles. Validation was performed using real-world clinical specimens, quantitative PCR (qPCR), and immunohistochemistry (IHC), supported by cohort analyses from the Human Protein Atlas (HPA) database.

RESULTS:

A glycogene-associated prognostic scoring system was established to categorize patients into risk-stratified subgroups. Patients in the high-risk cohort exhibited significantly poorer survival outcomes (p < 0.001). By incorporating clinicopathological variables into this framework, we established a predictive nomogram demonstrating strong calibration and a concordance index (C-index) of 0.78. The high-risk subgroup displayed elevated immune infiltration scores (p < 0.001), upregulated expression of immune checkpoint-related genes (p < 0.05), and an increased frequency of somatic mutations (p = 0.043). The risk score positively correlated with cancer-immunity cycle activation and immunotherapy-related signals. The high-risk groups also showed associations with T cell exhaustion, immune-activating genes, chemokines, and receptors. Drug sensitivity analysis revealed that low-risk patients were more sensitive to sorafenib, pazopanib, and erlotinib, whereas high-risk individuals responded better to temsirolimus (p < 0.01). qPCR and IHC analyses consistently revealed distinct expression patterns of MX2 and other key genes across the risk groups, further corroborated by the HPA findings.

CONCLUSION:

This glycogene-based signature provides a robust tool for predicting prognosis, TME characteristics, and therapeutic responses in KIRC, offering potential clinical utility in patient management.

01 Jul 2025·COMPUTERS IN BIOLOGY AND MEDICINE

Integrating AI/ML and multi-omics approaches to investigate the role of TNFRSF10A/TRAILR1 and its potential targets in pancreatic cancer

Article

Author: Kant, Shiva ; Sharma, Sudhanshu ; Mishra, Manoj K ; Singh, Rajesh

Pancreatic ductal adenocarcinoma (PDAC) remains one of the most lethal malignancies, with a five-year survival of under 10 % despite current therapies. Aggressive tumor biology, a desmoplastic stroma that limits drug delivery and immune cell infiltration, and profound resistance to apoptosis make it more complex to treat. Here, we describe a multi-layered system biology and drug discovery pipeline that integrates bulk genomics, single-cell spatial transcriptomics, proteomics, competing endogenous RNA (ceRNA) network analysis, and deep learning-driven quantitative structure-activity relationship (QSAR) modeling. By implementing this pipeline, we predicted that TNFRSF10A encodes for the TRAILR1 death receptor as a potential therapeutic target in PDAC. Mutational and expressional analysis also confirmed TNFRSF10A as a putative target in PDAC. Cancer cells within the PDAC microenvironment exhibit aberrantly elevated TNFRSF10A expression. Immune-excluded tumor niches and pro-survival signaling link this elevated expression. Using an advanced transformer-based deep learning approach, SELFormer, combined with QSAR analysis-based virtual screening, we identified previously unexplored FDA-approved drugs and natural compounds, i.e., Temsirolimus, Ergotamine, and capivasertib, with potential TRAILR1 modulatory effects. During molecular dynamics simulations, these repurposed candidates showed the highest binding affinities against TNFRSF10A for 300 ns. These showed favorable binding energies (MM-PBSA), minimal RMSD drift, PCA, and SASA. We propose TNFRSF10A as a therapeutically important PDAC vulnerability nurtured by spatially resolved expression patterns and dynamic molecular modeling. This study has used a novel integration of AI-implemented chemical modeling, high-throughput screening, and a multi-omics approach to unravel and pharmacologically target a cancer compartment-specific weakness in a notoriously drug-resistant cancer.

01 Jul 2025·PEDIATRIC BLOOD & CANCER

Strategies for the Treatment of Desmoplastic Small Round Cell Tumor: A Case Series

Article

Author: Lazar, Paige ; Lee, Alice ; Fabish, Lara ; Offenbacher, Rachel ; Fox, Jana ; Baker, Alissa ; Loeb, David M.

ABSTRACT:

Desmoplastic small round cell tumor (DSRCT) is a rare, aggressive pediatric sarcoma that has a poor prognosis despite a multimodal approach of surgical resection, chemotherapy, and radiation. Incidence is only approximately 0.2 cases per million, limiting clinical trials from which to derive a standard of treatment. Advancement instead relies on case reports and series. The Children's Hospital at Montefiore in the Bronx, New York, a tertiary care hospital associated with the Montefiore Einstein Comprehensive Cancer Center, has treated eight such patients in the last decade, resulting in one of the largest single‐institution case series of DSRCT patients to date. Though these patients are demographically unusual for DSRCT, including two women and two with rare extra‐abdominal tumors of the brain and bone, through treatment of these patients we have accrued experience regarding various treatment strategies in both primary and refractory DSRCT. We treat primary DSRCT with interval‐compressed vincristine/doxorubicin/cyclophosphamide alternating with cycles of ifosfamide/etoposide and irinotecan/temozolomide/temsirolimus (ITT). This is the first descriptive series of this size describing the use of ITT in DSRCT. Treatment for refractory cases focuses on targeted therapies when available, emphasizing the personalization that DSRCT treatment requires.

News (Medical) associated with Temsirolimus

18 Jun 2025

·www.einpresswire.com

Palvella Therapeutics Granted Sixth U.S. Patent Covering 0.1–20% Anhydrous Compositions of Rapamycin and Other mTOR Inhibitors

New intellectual property builds on Palvella’s multi-layered exclusivity strategy Patent term expected through at least 2038 WAYNE, Pa., June 18, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: PVLA) Palvella Therapeutics, Inc . (Palvella), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced that the United States Patent and Trademark Office (USPTO) issued patent No. 12,329,748 for claims related to the Company's lead product candidate QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin). The patent provides broad protection for the proprietary anhydrous topical composition and methods of use for QTORIN™ rapamycin. “Our sixth issued U.S. patent marks another meaningful milestone in solidifying the exclusivity position of QTORIN™ rapamycin,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella. “We’re executing a multi-layered strategy to protect and maximize the long-term value of QTORIN™ rapamycin—anchored by strong intellectual property, proprietary formulation and manufacturing trade secrets, and regulatory exclusivities.” The newly granted U.S. patent encompasses a wide range of composition and method-of-use claims for QTORIN™ rapamycin and other mTOR inhibitors—including temsirolimus and everolimus—formulated in anhydrous compositions. It also covers treatment of a broad spectrum of dermatologic conditions, with emphasis on rare and difficult-to-treat diseases such as microcystic lymphatic malformations and venous malformations. QTORIN™ rapamycin has been granted Breakthrough Therapy, Orphan Drug, and Fast Track Designations by the FDA for the treatment of microcystic lymphatic malformations. If approved, QTORIN™ rapamycin is expected to qualify for seven years of orphan drug market exclusivity in the U.S. Palvella has also been named an awardee of an FDA Orphan Products Grant from the FDA’s Office of Orphan Products Development and is eligible to receive up to $2.6 million over the life of the grant to support the ongoing SELVA Phase 3 trial of QTORIN™ rapamycin. About Palvella Therapeutics Founded and led by rare disease drug development veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies. Palvella is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare genetic skin diseases, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently being evaluated in the Phase 3 SELVA clinical trial in microcystic lymphatic malformations and the Phase 2 TOIVA clinical trial in cutaneous venous malformations. For more information, please visit www.palvellatx.com or follow Palvella on LinkedIn or X (formerly known as Twitter). QTORIN™ rapamycin is for investigational use only and has not been approved or cleared by the FDA or by any other regulatory agency for any indication. Forward-Looking Statements This press release contains forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended (Securities Act)). These statements may discuss goals, intentions, and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the management of Palvella, as well as assumptions made by, and information currently available to, the management of Palvella. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Statements that are not historical facts are forward-looking statements. Forward-looking statements include, but are not limited to, statements regarding the expected timing of the presentation of data from ongoing clinical trials, Palvella’s clinical development plans and related anticipated development milestones, Palvella’s cash and financial resources and expected cash runway, and the potential of, and expectations regarding, Palvella’s programs, including QTORIN™ rapamycin, and its research-stage opportunities, including its expected therapeutic potential and market opportunity. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the ability to raise additional capital to finance operations; the ability to advance product candidates through preclinical and clinical development; the ability to obtain regulatory approval for, and ultimately commercialize, Palvella’s product candidates, including QTORIN™ rapamycin; the outcome of early clinical trials for Palvella’s product candidates, including the ability of those trials to satisfy relevant governmental or regulatory requirements; the fact that data and results from clinical studies may not necessarily be indicative of future results; Palvella’s limited experience in designing clinical trials and lack of experience in conducting clinical trials; the ability to identify and pivot to other programs, product candidates, or indications that may be more profitable or successful than Palvella’s current product candidates; the substantial competition Palvella faces in discovering, developing, or commercializing products; the negative impacts of global events on operations, including ongoing and planned clinical trials and ongoing and planned preclinical studies; the ability to attract, hire, and retain skilled executive officers and employees; the ability of Palvella to protect its intellectual property and proprietary technologies; reliance on third parties, contract manufacturers, and contract research organizations; and the risks and uncertainties described in the filings made by Palvella with the Securities and Exchange Commission (SEC), including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at www.sec.gov. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties that Palvella may face. Except as required by applicable law, Palvella does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release. Contact Information Investors Wesley H. Kaupinen Founder and CEO, Palvella Therapeutics wes.kaupinen@palvellatx.com Media Marcy Nanus Managing Partner, Trilon Advisors LLC mnanus@trilonadvisors.com Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Orphan DrugPhase 3Fast TrackBreakthrough Therapy

04 Apr 2025

·www.prnewswire.com

UPSHER-SMITH TO SHOWCASE RARE DISEASE PORTFOLIO AT 2025 AAN ANNUAL MEETING

Company to Feature First and Only Ready-to-Use Liquid Vigabatrin at Booth #846 MAPLE GROVE, Minn., April 4, 2025 /PRNewswire/ -- Upsher-Smith Laboratories, LLC (Upsher-Smith) today announced its participation at the 2025 American Academy of Neurology (AAN) Annual Meeting, from April 5-9, 2025 in San Diego, California. Representatives from Upsher-Smith will be available at Booth #846 to share information with clinicians about its expanding portfolio of products for rare diseases, including the first and only ready-to-use liquid vigabatrin, VIGAFYDE™ (vigabatrin) Oral Solution. VIGAFYDE™ is indicated for infantile spasms (IS) as monotherapy in infants 1 month to 2 years of age for whom the potential benefits outweigh the risk of vision loss. "Since the successful launch of VIGADRONE® (vigabatrin) for Oral Solution to the specialty pharmacy channel in 2018, we have broadened our offerings to include VIGADRONE® (vigabatrin) Tablets, TORPENZ™ (everolimus) Tablets, and VIGAFYDE™ ready-to-use solution," said Jim Maahs, Head of Commercial, Upsher-Smith. "As part of our commitment to supporting the rare disease community, Upsher-Smith is dedicated to not only maintaining, but enhancing the support and resources available to patients, caregivers, and healthcare practitioners. We encourage attendees to stop by our booth during this year's AAN meeting to learn more." About Upsher-Smith's Rare Disease Portfolio Upsher-Smith's rare disease portfolio continues to grow with the addition of high-quality medications that match or exceed the level of service provided by innovator brands. The Company offers three vigabatrin formulations including VIGAFYDE™ Oral Solution, VIGADRONE® Powder for Oral Solution and VIGADRONE® Tablets. VIGAFYDE™, the only ready-to-use vigabatrin oral solution, is the first new drug application (NDA) approved for infantile spasms in 15 years. As the only premixed, ready-to-use vigabatrin formulation, VIGAFYDE™ represents a welcome expansion of vigabatrin treatment options, offering patients and caregivers a simpler dose preparation process which may improve dosing accuracy. Upsher-Smith representatives will be available throughout the conference at exhibit booth #846 to engage with clinicians about their products including VIGAFYDE™, and VIGADRONE® and the Promise of Support™ Program, which has been providing comprehensive support to patients prescribed VIGADRONE® since 2018. Representatives will also be available to discuss Upsher-Smith's TORPENZ™ Tablets, an oral mTOR inhibitor used to treat certain manifestations of Tuberous Sclerosis Complex (TSC). TORPENZ™, is the only everolimus backed by the Upsher-Smith Promise of Support™ Program to deliver reliable and consistent support to patients, caregivers, and healthcare providers in the TSC community. TORPENZ™ is indicated for the treatment of adult patients with renal angiomyolipoma and TSC, not requiring immediate surgery and is also indicated in adult and pediatric patients aged 1 year and older with TSC for the treatment of subependymal giant cell astrocytoma (SEGA) that requires therapeutic intervention but cannot be curatively resected. About Upsher-Smith's Promise of Support™ Program Central to Upsher-Smith's commitment to rare disease communities is its Promise of Support™ Program, ensuring comprehensive assistance for patients, caregivers, and healthcare providers throughout the treatment journey. For nearly a decade, the program has offered resources for uninterrupted medication supply, prior authorization support, patient education materials, collaboration with a leading specialty pharmacy partner, and streamlined prescribing and enrollment procedures. It also includes copay assistance for eligible patients to help lessen the financial burden on patients and their families. To learn more about Upsher-Smith, its products, and the Promise of Support™ Program visit: . Vigabatrin REMS All vigabatrin products are governed by a Risk Evaluation and Mitigation Strategy (REMS) mandated by the FDA. The Vigabatrin REMS ensures that patients and healthcare providers make informed risk-benefit decisions about its use. Healthcare providers must be enrolled in the Vigabatrin REMS to prescribe these products, and patients must be enrolled to receive them. WHAT IS VIGAFYDE? VIGAFYDE™ (vigabatrin) Oral Solution is a prescription medicine used to treat: Infantile Spasms (IS) in babies 1 month to 2 years of age, if you and your baby's healthcare provider decide the possible benefits of taking VIGAFYDE are more important than the possible risk of vision loss. WHAT IMPORTANT SAFETY INFORMATION SHOULD I KNOW ABOUT VIGAFYDE™ (vigabatrin)? Because VIGAFYDE might cause permanent vision loss, it is available to healthcare providers and patients only under a special program called the Vigabatrin Risk Evaluation and Mitigation Strategy (REMS) Program. Your baby's healthcare provider will explain the details of this Program to you. VIGAFYDE can damage the vision of anyone who takes it. Some people can have severe loss, particularly to their ability to see to the side when looking straight ahead (peripheral vision). With severe vision loss, your baby may only be able to see things straight in front of them (sometimes called "tunnel vision"). Your baby may also have blurry vision. If this happens, it will not get better. Tell your healthcare provider right away if your baby might not be seeing as well as before starting VIGAFYDE or is acting differently than normal. These changes can mean that vision damage has occurred. Regular vision testing is recommended. It is recommended that your healthcare provider test your baby's vision before or within 4 weeks after starting VIGAFYDE, and at least every 3 months during treatment until VIGAFYDE is stopped. It is also recommended that vision be tested about 3 to 6 months after VIGAFYDE is stopped. It is difficult to test vision in babies, but to the extent possible, all patients should have their vision tested. Your baby's healthcare provider will determine if testing can be done. Regular vision testing is important because damage can happen before any changes are noticed. Vision tests cannot prevent the vision damage that can happen with VIGAFYDE, but they do allow VIGAFYDE to be stopped if vision has gotten worse, which usually will lessen further damage. Even these regular vision tests may not show vision damage before it is serious and permanent. Parents, caregivers, and healthcare providers may not recognize the symptoms, or find vision loss in babies, until it is severe. If you do not have these vision tests regularly, your baby's healthcare provider may stop prescribing VIGAFYDE. Your baby may not be able to complete vision testing. If vision testing cannot be done, the healthcare provider may continue prescribing VIGAFYDE but will not be able to watch for any vision loss. Magnetic resonance imaging (MRI) changes in patients with infantile spasms (IS). Brain pictures taken by MRI show changes in some patients after they are given VIGAFYDE. It is not known if these changes are harmful. A type of swelling in the brain called intramyelinic edema (IME) has been seen in postmortem examination of infants treated with vigabatrin. Risk of suicidal thoughts or actions. VIGAFYDE is approved for use in patients 1 month to 2 years of age [see WHAT IS VIGAFYDE?]. VIGAFYDE is not approved for use in adolescents or adults. Like other antiepileptic drugs, vigabatrin may cause suicidal thoughts and actions in some adolescents and adults (about 1 in 500 people). A healthcare provider should be called right away if a patient taking vigabatrin experiences any symptoms, especially sudden changes in mood, behaviors, thoughts, or feelings, and especially if they are new, worse, or worrying. Do not stop VIGAFYDE without first talking to a healthcare provider. Stopping VIGAFYDE suddenly can cause seizures that will not stop. VIGAFYDE is more concentrated than vigabatrin solutions prepared from powder. The volume of VIGAFYDE solution prescribed by your baby's healthcare provider may be less than the volume prescribed for other vigabatrin solutions prepared from powder. Always double check the volume (mL) that you are to give your baby when you get a new prescription filled. VIGAFYDE can cause serious side effects such as low red blood cell counts (anemia), sleepiness and tiredness, and weight gain. VIGAFYDE may make certain types of seizures worse. Tell your healthcare provider right away if seizures get worse. Before starting VIGAFYDE, tell your baby's doctor about all of their medical conditions including any allergic reaction to vigabatrin, vision problems, kidney problems, or low red blood cell counts (anemia). Tell your doctor about all the medicines you baby is receiving. The most common side effects of VIGAFYDE in babies include: sleepiness (sleepy babies may have a harder time suckling and feeding or may be irritable), swelling in the bronchial tubes (bronchitis), ear infection and irritability. Tell your healthcare provider if your baby has any side effect that bothers them or that does not go away. This is the most important information to know about VIGAFYDE, but not all of the safety information. For more information, ask your healthcare provider or pharmacist, or please see the VIGAFYDE Medication Guide, full Prescribing Information including Boxed Warning for risk of permanent vision loss, and Instructions for Use. You can also visit VIGAFYDE.com, upsher-smith.com or call 1-888-650-3789. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-332-1088. WHAT IS VIGADRONE? VIGADRONE® (vigabatrin) is a prescription medicine used to treat: Infantile Spasms (IS) in patients 1 month to 2 years of age, if you and your healthcare provider decide the possible benefits of taking VIGADRONE are more important than the possible risk of vision loss. Refractory Complex Partial Seizures (CPS) in adults and children 2 years and older with refractory complex partial seizures (CPS) along with other treatments if: The CPS do not respond well enough to several other treatments, and You and your healthcare provider decide the possible benefit of taking VIGADRONE is more important than the risk of vision loss. VIGADRONE should not be the first medicine used to treat CPS. WHAT IMPORTANT SAFETY INFORMATION SHOULD I KNOW ABOUT VIGADRONE® (vigabatrin)? Because VIGADRONE might cause permanent vision loss, it is available to healthcare providers and patients only under a special program called the Vigabatrin Risk Evaluation and Mitigation Strategy (REMS) Program. Your healthcare provider will explain the details of this Program to you. VIGADRONE can damage the vision of anyone who takes it. Some people can have severe loss, particularly to their ability to see to the side when looking straight ahead (peripheral vision). With severe vision loss, you may only be able to see things straight in front of you (sometimes called "tunnel vision"). You may also have blurry vision. If this happens, it will not get better. Tell your healthcare provider right away if you (or your child): might not be seeing as well as before starting VIGADRONE; start to trip, bump into things, or are more clumsy than usual; are surprised by people or things coming in front of you that seem to come out of nowhere; or if your baby is acting differently than normal. These changes can mean that vision damage has occurred. Regular vision testing is recommended. It is recommended that your healthcare provider test your (or your child's) vision before or within 4 weeks after starting VIGADRONE, and at least every 3 months during treatment until VIGADRONE is stopped. It is also recommended that vision be tested about 3 to 6 months after VIGADRONE is stopped. It is difficult to test vision in babies, but to the extent possible, all patients should have their vision tested. Your healthcare provider will determine if testing can be done. Regular vision testing is important because damage can happen before any changes are noticed. Vision tests cannot prevent the vision damage that can happen with VIGADRONE, but they do allow VIGADRONE to be stopped if vision has gotten worse, which usually will lessen further damage. Even these regular vision tests may not show vision damage before it is serious and permanent. Parents, caregivers, and healthcare providers may not recognize the symptoms, or find vision loss in patients, until it is severe. If you do not have these vision tests regularly, your healthcare provider may stop prescribing VIGADRONE for you (or your child). Some people are not able to complete vision testing. If vision testing cannot be done, your healthcare provider may continue prescribing VIGADRONE, but will not be able to watch for any vision loss. Magnetic resonance imaging (MRI) changes in patients with infantile spasms (IS). Brain pictures taken by MRI show changes in some patients after they are given VIGADRONE. It is not known if these changes are harmful. A type of swelling in the brain called intramyelinic edema (IME) has been seen in autopsy examination of patients treated with vigabatrin. Risk of suicidal thoughts or actions. Like other antiepileptic drugs, VIGADRONE may cause suicidal thoughts and actions in some people (about 1 in 500 people). Call a healthcare provider right away if you (or your child) have any symptoms, especially sudden changes in mood, behaviors, thoughts or feelings, and especially if they are new, worse, or worry you. Do not stop VIGADRONE without first talking to a healthcare provider. Stopping VIGADRONE suddenly can cause seizures that will not stop. VIGADRONE can cause serious side effects such as low red blood cell counts (anemia), sleepiness and tiredness, nerve problems, weight gain, and swelling. Because VIGADRONE causes sleepiness and tiredness, do not drive, operate machinery, or perform any hazardous task, unless it is decided that these things can be done safely. VIGADRONE may make certain types of seizures worse. Tell your healthcare provider right away if seizures get worse. Before starting VIGADRONE, tell your doctor about all of your (or your child's) medical conditions including depression, mood problems, suicidal thoughts or behavior, any allergic reaction to VIGADRONE, vision problems, kidney problems, low red blood cell counts (anemia), and any nervous or mental illnesses. Tell your doctor about all the medicines you (or your child) take. If you are breastfeeding or plan to breastfeed, VIGADRONE can pass into breast milk and may harm your baby. Breastfeeding is not recommended. If you are pregnant or plan to become pregnant, VIGADRONE can cause harm to your unborn baby. You and your healthcare provider will have to decide if you should take VIGADRONE while you are pregnant. The most common side effects of VIGADRONE in adults include: blurred vision, sleepiness, dizziness, problems walking or feeling uncoordinated, shaking (tremor) and tiredness. The most common side effect of VIGADRONE in children 3 to 16 years of age is weight gain. Also expect side effects like those seen in adults. The most common side effects of VIGADRONE in babies include: sleepiness (sleepy babies may have a harder time suckling and feeding or may be irritable), swelling in the bronchial tubes (bronchitis), ear infection and irritability. Tell your healthcare provider if you or your child have any side effect that bothers you or that does not go away. This is the most important information to know about VIGADRONE, but it is not all the safety information. For more information, ask your healthcare provider or pharmacist, or please see the VIGADRONE Medication Guide, full Prescribing Information including Boxed Warning for risk of permanent vision loss, and Instructions for Use. You can also visit VIGADRONE.com, upsher-smith.com or call 1-888-650-3789. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-332-1088. WHAT ARE TORPENZ (everolimus) TABLETS? TORPENZ is a prescription medicine used to treat the following types of benign (non-cancerous) tumors that are seen with a genetic condition called tuberous sclerosis complex (TSC): Brain tumors called subependymal giant cell astrocytoma (SEGA), when the tumor cannot be removed completely by surgery, in adults and children 1 year and older. Kidney tumors called angiomyolipoma, when the tumor does not require surgery right away, in adults. For additional information, including safety information, for non-TSC indications, see the full Prescribing Information, including Patient Information. WHAT IMPORTANT SAFETY INFORMATION SHOULD I KNOW ABOUT TORPENZ™ ( everolimus) TABLETS in Tuberous Sclerosis Complex? Do not take TORPENZ if you have had an allergic reaction to everolimus. Talk to your healthcare provider before taking TORPENZ if you are allergic to sirolimus or temsirolimus. Ask your provider if you do not know. TORPENZ can cause serious side effects, including: Lung or breathing problems. In some people, these may be severe and can be life threatening. Tell your healthcare provider right away if you have new or worsening cough, shortness of breath, chest pain, difficulty breathing or wheezing. Higher likelihood of infection such as pneumonia, or a bacterial, fungal, or viral infection. Viral infections may include active hepatitis B in people who have had hepatitis B in the past (reactivation). In some adults and children, these infections may be severe and can be life threatening. You may need to be treated as soon as possible. Tell your healthcare provider right away if you have a temperature of 100.5˚F or above, chills, or do not feel well. Symptoms of infection may include: Severe allergic reactions. Get medical help right away if you have signs of an allergic reaction, including rash, itching, hives, flushing, trouble breathing or swallowing, chest pain or dizziness. Possible increased risk for a type of allergic reaction called angioedema in people who take an Angiotensin-Converting Enzyme (ACE) inhibitor with TORPENZ. Talk with your healthcare provider before taking TORPENZ if you are not sure if you take an ACE inhibitor. Get medical help right away if you have trouble breathing or develop swelling of your tongue, mouth, or throat during treatment with TORPENZ. Mouth ulcers and sores. Mouth ulcers and sores are common during treatment with TORPENZ but can also be severe. When you start TORPENZ, your healthcare provider may tell you to also start a prescription mouthwash to reduce the likelihood of getting mouth ulcers or sores and to reduce their severity. Follow your provider's instructions on how to use this mouthwash. If you develop pain, discomfort, or open sores in your mouth, tell your provider. Kidney failure. In some people, this may be severe and can be life threatening. Your healthcare provider should check your kidney function before and during treatment. Wound healing problems. Wounds may not heal properly during TORPENZ treatment. Tell your healthcare provider if you plan to have any surgery before starting or during treatment. You should stop TORPENZ at least 1 week before planned surgery. Your provider should tell you when to start taking TORPENZ again after surgery. Increased blood sugar and fat (cholesterol and triglyceride) levels in the blood. Your healthcare provider should check your fasting blood sugar, cholesterol, and triglyceride levels in the blood before you start and during treatment. Decreased blood cell counts. TORPENZ can cause you to have decreased red blood cells, white blood cells and platelets. Your healthcare provider should check your blood cell counts before you start and during treatment. Worsening side effects from radiation treatment that can sometimes be severe. Tell your healthcare provider if you have had or are planning to receive radiation therapy. Before taking TORPENZ, tell your healthcare provider about all of your medical conditions, including if you: Have or have had kidney or liver problems, have diabetes or high blood sugar, have high blood cholesterol, have any infections, or previously had hepatitis B. Are scheduled to receive any vaccinations. You should not receive a "live vaccine" or be around people who have recently received a "live vaccine" during your treatment with TORPENZ. If you are not sure about the type of immunization or vaccine, ask your provider. For children, work with your provider to complete the recommended childhood series of vaccines before your child starts TORPENZ. Are pregnant, can become pregnant, or have a partner who can become pregnant. TORPENZ can cause harm to your unborn baby. For females who are able to become pregnant, your provider will give you a pregnancy test before you start TORPENZ. Use effective birth control during treatment and for 8 weeks after your last dose of TORPENZ. Males with a female partner should use effective birth control during treatment and for 4 weeks after your last dose of TORPENZ. Are breastfeeding or plan to breastfeed. Do not breastfeed during treatment and for 2 weeks after your last dose of TORPENZ. Are planning to have or have had a recent surgery. You should stop taking TORPENZ at least 1 week before planned surgery. Have received or are planning to receive radiation therapy. Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking TORPENZ with some medicines can cause serious side effects. Keep a list of medications you take, and show it to your healthcare provider and pharmacist when you get a new medicine. Especially tell your provider if you take: St. John's Wort (Hypericum perforatum) Medicine for fungal or bacterial infections, tuberculosis, seizures, HIV-AIDS, heart conditions or high blood pressure, including ACE inhibitors Medicines that weaken your immune system (your body's ability to fight infections and other problems) Ask your provider or pharmacist if you are not sure if your medicine is one of those taken for the conditions listed above. If you are taking any medicines for the conditions listed above, your provider might need to prescribe a different medicine or your dose of TORPENZ may need to be changed. Tell your provider before you start any new medicine. You should not drink grapefruit juice or eat grapefruit during your treatment with TORPENZ. It may make the amount of TORPENZ in your blood increase to a harmful level. The most common side effect of TORPENZ in people who have SEGA or renal angiomyolipoma include respiratory tract infection. Other side effects that may occur with TORPENZ: Absence of menstrual periods (menstruation). Tell your healthcare provider if this happens. TORPENZ may affect fertility and may affect your ability to become pregnant if you are female or your ability to father a child if you are male. Talk to your provider if this is a concern. Tell your healthcare provider if you have any side effect that bothers you or does not go away. These are not all the possible side effects of TORPENZ. For more information, ask your healthcare provider or pharmacist. Call your doctor for medical advice about side effects. You can also visit TORPENZ.com, upsher-smith.com or call 1-888-650-3789. You are encouraged to report negative side effects of prescription drugs to FDA. Visit or call 1-800-332-1088. About Upsher-Smith Upsher-Smith Laboratories, LLC, now a member of Bora Group, is a trusted U.S. pharmaceutical company that strives to improve the health and lives of patients through an unwavering commitment to high-quality products and sustainable growth. We bring generics and brands to a wide array of customers, always backed by our attentive level of service, strong industry relationships, and dedication to uninterrupted supply. For more information, visit . About Bora Founded in 2007, Bora Pharmaceutical Co., Ltd. ("Bora" or "the Company", 6472.TW) now is the largest pharmaceutical manufacturer in Taiwan with well-connected global distribution to supply more than 100 countries around the world. Bora is dedicated to becoming a global leader in pharmaceutical manufacturing by offering its clients the best quality, efficiency and reliability. For more information, visit . VIGADRONE, TORPENZ and Promise of Support are trademarks of Upsher-Smith Laboratories, LLC. VIGAFYDE is a trademark of Pyros Pharmaceuticals, Inc. SOURCE Upsher-Smith Laboratories, LLC WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug Approval

11 Nov 2024

·www.prnewswire.com

UPSHER-SMITH PRESENTS EXPANDED RARE DISEASE PORTFOLIO AT 2024 CHILD NEUROLOGY SOCIETY ANNUAL MEETING

Visit Our Booths to Learn More about the Company's Latest Product Offerings and Promise of Support™ Program MAPLE GROVE, Minn., Nov. 11, 2024 /PRNewswire/ -- Upsher-Smith Laboratories, LLC (Upsher-Smith) today announced that it will attend the 2024 Child Neurology Society (CNS) Annual Meeting in San Diego, CA from November 11-14, 2024. Representatives from Upsher-Smith will be at our booths to speak with clinicians about its expanding portfolio of medications including newly acquired VIGAFYDE™ (vigabatrin) Oral Solution and recently launched TORPENZ™ (everolimus) Tablets, which became available in August 2024. VIGAFYDE™ is the first and only ready-to-use vigabatrin oral solution and represents a welcome expansion of options for the treatment of infantile spasms as the first new drug application approved for this condition in 15 years. As the only premixed, ready-to-use vigabatrin formulation, VIGAFYDE™ offers patients and caregivers a new option in liquid vigabatrin that does not require reconstitution by the caregiver. VIGAFYDE™, which became available to patients in September 2024, is indicated as monotherapy for the treatment of infantile spasms in pediatric patients 1 month to 2 years, where the potential benefits outweigh the potential risk of vision loss. TORPENZ™, an oral mTOR inhibitor used to treat certain manifestations of Tuberous Sclerosis Complex (TSC), is the only everolimus backed by the Upsher-Smith Promise of Support™ Program to deliver reliable and consistent support to patients, caregivers, and healthcare providers in the TSC community. TORPENZ™ is indicated for the treatment of adult patients with renal angiomyolipoma and TSC, not requiring immediate surgery and is also indicated in adult and pediatric patients aged 1 year and older with TSC for the treatment of subependymal giant cell astrocytoma (SEGA) that requires therapeutic intervention but cannot be curatively resected. "Since the successful launch of VIGADRONE® (vigabatrin) for Oral Solution to the specialty pharmacy channel in 2018, we have broadened our offerings to include VIGADRONE® (vigabatrin) Tablets, TORPENZ™ Tablets, and now VIGAFYDE™ ready-to-use solution," said Jim Maahs, Head of Commercial, Upsher-Smith. "As part of our commitment to supporting the rare disease community, Upsher-Smith is dedicated to not only maintaining, but enhancing the support and resources available to patients, caregivers, and healthcare practitioners. We encourage attendees to stop by our booths during this year's CNS meeting to learn more." About Upsher-Smith's Promise of Support™ Program Central to Upsher-Smith's commitment to rare disease communities is its Promise of Support™ Program, ensuring comprehensive assistance for patients, caregivers, and healthcare providers throughout the treatment journey. For nearly a decade, the program has offered resources for uninterrupted medication supply, prior authorization support, patient education materials, collaboration with a leading specialty pharmacy partner, and streamlined prescribing and enrollment procedures. It also includes copay assistance for eligible patients to help lessen the financial burden on patients and their families. To learn more about Upsher-Smith, its products, and the Promise of Support™ Program visit: . Vigabatrin REMS All vigabatrin products are governed by a Risk Evaluation and Mitigation Strategy (REMS) mandated by the FDA. The Vigabatrin REMS ensures that patients and healthcare providers make informed risk-benefit decisions about its use. Healthcare providers must be enrolled in the Vigabatrin REMS to prescribe these products, and patients must be enrolled to receive them. IMPORTANT SAFETY INFORMATION FOR VIGAFYDE™ (vigabatrin) What is the most important information I should know about VIGAFYDE? VIGAFYDE can cause serious side effects, including: Permanent vision loss: VIGAFYDE can damage the vision of anyone who takes it. Some babies can have severe loss, particularly to their ability to see to the side when they look straight ahead (peripheral vision). With severe vision loss, your baby may only be able to see things straight in front of them (sometimes called "tunnel vision"). Your baby also may have blurry vision. If this happens, it will not get better. Tell your healthcare provider right away if your baby might not be seeing as well as before starting VIGAFYDE or is acting differently than normal. Vision loss in babies: Because of the risk of vision loss, VIGAFYDE is used in babies 1 month to 2 years of age with infantile spasms only when you and your healthcare provider decide that the possible benefits of VIGAFYDE are more important than the risks. Parents or caregivers are not likely to recognize the symptoms of vision loss in babies until it is severe. Healthcare providers may not find vision loss in babies until it is severe. It is difficult to test vision in babies, but, to the extent possible, all babies should have their vision tested before starting VIGAFYDE or within 4 weeks after starting VIGAFYDE, and every 3 months after that until VIGAFYDE is stopped. Your baby also should have a vision test about 3 to 6 months after VIGAFYDE is stopped. Your healthcare provider will determine if your baby can be tested. If your baby cannot be tested, your healthcare provider may continue prescribing VIGAFYDE, but they will not be able to watch for any vision loss. Even if your baby's vision seems fine, it is important to get regular vision tests because damage can happen before your baby acts differently. Even these regular vision exams may not show the damage to your baby's vision before it is severe and permanent. All babies who take VIGAFYDE: Are at risk for permanent vision loss with any amount of VIGAFYDE. The risk of vision loss may increase with higher doses of VIGAFYDE, and the longer it is taken. It is not possible for your healthcare provider to know when vision loss will happen. It could happen soon after starting VIGAFYDE or any time during treatment. It may even happen after treatment has stopped. Because VIGAFYDE might cause permanent vision loss, it is available to healthcare providers and patients only under a special program called Vigabatrin Risk Evaluation and Mitigation Strategy (REMS). Your healthcare provider will explain the details of the Vigabatrin REMS to you. For more information, go to or call 1-866-244-8175. Magnetic resonance imaging (MRI) changes in babies with infantile spasms (IS): brain pictures taken by MRI show changes in some babies after they are given VIGAFYDE. It is not known if these changes are harmful. What are the possible side effects of VIGAFYDE? VIGAFYDE can cause serious side effects, including sleepiness (sleepy babies may have a harder time suckling and feeding or may be irritable) and weight gain without swelling. Serious side effects that happen in adults who take vigabatrin and are not known if they will happen in babies are low red blood cell counts, nerve problems, and swelling. Tell your baby's healthcare provider right away if your baby's seizures get worse. The most common side effects of VIGAFYDE in babies include sleepiness, ear infection, swelling of the bronchial tubes, and irritability. Tell your healthcare provider if your baby has any side effect that bothers them or that does not go away. These are not all the possible side effects of VIGAFYDE. Do not stop taking VIGAFYDE suddenly. This can cause serious problems. Talk to your healthcare provider for directions on how to stop treatment. What should I tell my healthcare provider before starting VIGAFYDE? Before giving VIGAFYDE to your baby for IS, inform your healthcare provider about all your baby's medical conditions. This includes whether your baby has ever had an allergic reaction to vigabatrin, such as hives, itching, or trouble breathing, as well as any history of vision problems, or kidney issues. Tell your healthcare provider about all the medicines your baby takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Interactions between VIGAFYDE and other medicines could lead to side effects. You may report side effects to the FDA at or call 1-800-FDA-1088 or Pyros at 1-855-406-1010. Please see full Prescribing Information, including BOXED WARNING, Medication Guide, and Instructions for Use for additional Important Safety Information. WHAT IMPORTANT SAFETY INFORMATION SHOULD I KNOW ABOUT TORPENZ™ ( everolimus) TABLETS in Tuberous Sclerosis Complex? Do not take TORPENZ if you have had an allergic reaction to everolimus. Talk to your healthcare provider before taking TORPENZ if you are allergic to sirolimus or temsirolimus. Ask your provider if you do not know. TORPENZ can cause serious side effects, including: Lung or breathing problems. In some people, these may be severe and can be life threatening. Tell your healthcare provider right away if you have new or worsening cough, shortness of breath, chest pain, difficulty breathing or wheezing. Higher likelihood of infection such as pneumonia, or a bacterial, fungal, or viral infection. Viral infections may include active hepatitis B in people who have had hepatitis B in the past (reactivation). In some adults and children, these infections may be severe and can be life threatening. You may need to be treated as soon as possible. Tell your healthcare provider right away if you have a temperature of 100.5˚F or above, chills, or do not feel well. Symptoms of infection may include: Severe allergic reactions. Get medical help right away if you have signs of an allergic reaction, including rash, itching, hives, flushing, trouble breathing or swallowing, chest pain or dizziness. Possible increased risk for a type of allergic reaction called angioedema in people who take an Angiotensin-Converting Enzyme (ACE) inhibitor with TORPENZ. Talk with your healthcare provider before taking TORPENZ if you are not sure if you take an ACE inhibitor. Get medical help right away if you have trouble breathing or develop swelling of your tongue, mouth, or throat during treatment with TORPENZ. Mouth ulcers and sores. Mouth ulcers and sores are common during treatment with TORPENZ but can also be severe. When you start TORPENZ, your healthcare provider may tell you to also start a prescription mouthwash to reduce the likelihood of getting mouth ulcers or sores and to reduce their severity. Follow your provider's instructions on how to use this mouthwash. If you develop pain, discomfort, or open sores in your mouth, tell your provider. Kidney failure. In some people, this may be severe and can be life threatening. Your healthcare provider should check your kidney function before and during treatment. Wound healing problems. Wounds may not heal properly during TORPENZ treatment. Tell your healthcare provider if you plan to have any surgery before starting or during treatment. You should stop TORPENZ at least 1 week before planned surgery. Your provider should tell you when to start taking TORPENZ again after surgery. Increased blood sugar and fat (cholesterol and triglyceride) levels in the blood. Your healthcare provider should check your fasting blood sugar, cholesterol, and triglyceride levels in the blood before you start and during treatment. Decreased blood cell counts. TORPENZ can cause you to have decreased red blood cells, white blood cells and platelets. Your healthcare provider should check your blood cell counts before you start and during treatment. Worsening side effects from radiation treatment that can sometimes be severe. Tell your healthcare provider if you have had or are planning to receive radiation therapy. Before taking TORPENZ, tell your healthcare provider about all of your medical conditions, including if you: Have or have had kidney or liver problems, have diabetes or high blood sugar, have high blood cholesterol, have any infections, or previously had hepatitis B. Are scheduled to receive any vaccinations. You should not receive a "live vaccine" or be around people who have recently received a "live vaccine" during your treatment with TORPENZ. If you are not sure about the type of immunization or vaccine, ask your provider. For children, work with your provider to complete the recommended childhood series of vaccines before your child starts TORPENZ. Are pregnant, can become pregnant, or have a partner who can become pregnant. TORPENZ can cause harm to your unborn baby. For females who are able to become pregnant, your provider will give you a pregnancy test before you start TORPENZ. Use effective birth control during treatment and for 8 weeks after your last dose of TORPENZ. Males with a female partner should use effective birth control during treatment and for 4 weeks after your last dose of TORPENZ. Are breastfeeding or plan to breastfeed. Do not breastfeed during treatment and for 2 weeks after your last dose of TORPENZ. Are planning to have or have had a recent surgery. You should stop taking TORPENZ at least 1 week before planned surgery. Have received or are planning to receive radiation therapy. Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking TORPENZ with some medicines can cause serious side effects. Keep a list of medications you take, and show it to your healthcare provider and pharmacist when you get a new medicine. Especially tell your provider if you take: St. John's Wort (Hypericum perforatum) Medicine for fungal or bacterial infections, tuberculosis, seizures, HIV-AIDS, heart conditions or high blood pressure, including ACE inhibitors Medicines that weaken your immune system (your body's ability to fight infections and other problems) Ask your provider or pharmacist if you are not sure if your medicine is one of those taken for the conditions listed above. If you are taking any medicines for the conditions listed above, your provider might need to prescribe a different medicine or your dose of TORPENZ may need to be changed. Tell your provider before you start any new medicine. You should not drink grapefruit juice or eat grapefruit during your treatment with TORPENZ. It may make the amount of TORPENZ in your blood increase to a harmful level. The most common side effect of TORPENZ in people who have SEGA or renal angiomyolipoma include respiratory tract infection. Other side effects that may occur with TORPENZ: Absence of menstrual periods (menstruation). Tell your healthcare provider if this happens. TORPENZ may affect fertility and may affect your ability to become pregnant if you are female or your ability to father a child if you are male. Talk to your provider if this is a concern. Tell your healthcare provider if you have any side effect that bothers you or does not go away. These are not all the possible side effects of TORPENZ. For more information, ask your healthcare provider or pharmacist. Call your doctor for medical advice about side effects. You can also visit TORPENZ.com, upsher-smith.com or call 1-888-650-3789. You are encouraged to report negative side effects of prescription drugs to FDA. Visit or call 1-800-332-1088. WHAT ARE TORPENZ (everolimus) TABLETS? TORPENZ is a prescription medicine used to treat the following types of benign (non-cancerous) tumors that are seen with a genetic condition called tuberous sclerosis complex (TSC): Brain tumors called subependymal giant cell astrocytoma (SEGA), when the tumor cannot be removed completely by surgery, in adults and children 1 year and older. Kidney tumors called angiomyolipoma, when the tumor does not require surgery right away, in adults. For additional information, including safety information, for non-TSC indications, see the full Prescribing Information, including Patient Information. WHAT IMPORTANT SAFETY INFORMATION SHOULD I KNOW ABOUT VIGADRONE® (vigabatrin)? Because VIGADRONE might cause permanent vision loss, it is available to healthcare providers and patients only under a special program called the Vigabatrin Risk Evaluation and Mitigation Strategy (REMS) Program. Your healthcare provider will explain the details of this Program to you. VIGADRONE can damage the vision of anyone who takes it. Some people can have severe loss, particularly to their ability to see to the side when looking straight ahead (peripheral vision). With severe vision loss, you may only be able to see things straight in front of you (sometimes called "tunnel vision"). You may also have blurry vision. If this happens, it will not get better. Tell your healthcare provider right away if you (or your child): might not be seeing as well as before starting VIGADRONE; start to trip, bump into things, or are more clumsy than usual; are surprised by people or things coming in front of you that seem to come out of nowhere; or if your baby is acting differently than normal. These changes can mean that vision damage has occurred. Regular vision testing is recommended. It is recommended that your healthcare provider test your (or your child's) vision before or within 4 weeks after starting VIGADRONE, and at least every 3 months during treatment until VIGADRONE is stopped. It is also recommended that vision be tested about 3 to 6 months after VIGADRONE is stopped. It is difficult to test vision in babies, but to the extent possible, all patients should have their vision tested. Your healthcare provider will determine if testing can be done. Regular vision testing is important because damage can happen before any changes are noticed. Vision tests cannot prevent the vision damage that can happen with VIGADRONE, but they do allow VIGADRONE to be stopped if vision has gotten worse, which usually will lessen further damage. Even these regular vision tests may not show vision damage before it is serious and permanent. Parents, caregivers, and healthcare providers may not recognize the symptoms, or find vision loss in patients, until it is severe. If you do not have these vision tests regularly, your healthcare provider may stop prescribing VIGADRONE for you (or your child). Some people are not able to complete vision testing. If vision testing cannot be done, your healthcare provider may continue prescribing VIGADRONE, but will not be able to watch for any vision loss. Magnetic resonance imaging (MRI) changes in patients with infantile spasms (IS). Brain pictures taken by MRI show changes in some patients after they are given VIGADRONE. It is not known if these changes are harmful. A type of swelling in the brain called intramyelinic edema (IME) has been seen in autopsy examination of patients treated with vigabatrin. Risk of suicidal thoughts or actions. Like other antiepileptic drugs, VIGADRONE may cause suicidal thoughts and actions in some people (about 1 in 500 people). Call a healthcare provider right away if you (or your child) have any symptoms, especially sudden changes in mood, behaviors, thoughts or feelings, and especially if they are new, worse, or worry you. Do not stop VIGADRONE without first talking to a healthcare provider. Stopping VIGADRONE suddenly can cause seizures that will not stop. VIGADRONE can cause serious side effects such as low red blood cell counts (anemia), sleepiness and tiredness, nerve problems, weight gain, and swelling. Because VIGADRONE causes sleepiness and tiredness, do not drive, operate machinery, or perform any hazardous task, unless it is decided that these things can be done safely. VIGADRONE may make certain types of seizures worse. Tell your healthcare provider right away if seizures get worse. Before starting VIGADRONE, tell your doctor about all of your (or your child's) medical conditions including depression, mood problems, suicidal thoughts or behavior, any allergic reaction to VIGADRONE, vision problems, kidney problems, low red blood cell counts (anemia), and any nervous or mental illnesses. Tell your doctor about all the medicines you (or your child) take. If you are breastfeeding or plan to breastfeed, VIGADRONE can pass into breast milk and may harm your baby. Breastfeeding is not recommended. If you are pregnant or plan to become pregnant, VIGADRONE can cause harm to your unborn baby. You and your healthcare provider will have to decide if you should take VIGADRONE while you are pregnant. The most common side effects of VIGADRONE in adults include: blurred vision, sleepiness, dizziness, problems walking or feeling uncoordinated, shaking (tremor) and tiredness. The most common side effect of VIGADRONE in children 3 to 16 years of age is weight gain. Also expect side effects like those seen in adults. The most common side effects of VIGADRONE in babies include: sleepiness (sleepy babies may have a harder time suckling and feeding or may be irritable), swelling in the bronchial tubes (bronchitis), ear infection and irritability. Tell your healthcare provider if you or your child have any side effect that bothers you or that does not go away. This is the most important information to know about VIGADRONE, but it is not all the safety information. For more information, ask your healthcare provider or pharmacist, or please see the VIGADRONE Medication Guide, full Prescribing Information including Boxed Warning for risk of permanent vision loss, and Instructions for Use. You can also visit VIGADRONE.com, upsher-smith.com or call 1-888-650-3789. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-332-1088. WHAT IS VIGADRONE? VIGADRONE® (vigabatrin) is a prescription medicine used to treat: Infantile Spasms (IS) in patients 1 month to 2 years of age, if you and your healthcare provider decide the possible benefits of taking VIGADRONE are more important than the possible risk of vision loss. Refractory Complex Partial Seizures (CPS) in adults and children 2 years and older with refractory complex partial seizures (CPS) along with other treatments if: The CPS do not respond well enough to several other treatments, and You and your healthcare provider decide the possible benefit of taking VIGADRONE is more important than the risk of vision loss. VIGADRONE should not be the first medicine used to treat CPS. About Upsher-Smith Upsher-Smith Laboratories, LLC, now a member of Bora Group, is a trusted U.S. pharmaceutical company that strives to improve the health and lives of patients through an unwavering commitment to high-quality products and sustainable growth. We bring generics and brands to a wide array of customers, always backed by our attentive level of service, strong industry relationships, and dedication to uninterrupted supply. For more information, visit . About Bora Founded in 2007, Bora Pharmaceutical Co., Ltd. ("Bora" or "the Company", 6472.TW) now is the largest pharmaceutical manufacturer in Taiwan with well-connected global distribution to supply more than 100 countries around the world. Bora is dedicated to becoming a global leader in pharmaceutical manufacturing by offering its clients the best quality, efficiency and reliability. For more information, visit . TORPENZ, VIGADRONE, and Promise of Support are trademarks of Upsher-Smith Laboratories, LLC. VIGAFYDE is a trademark of Pyros Pharmaceuticals, Inc. SOURCE Upsher-Smith Laboratories, LLC WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug Approval

100 Deals associated with Temsirolimus

External Link

KEGG	Wiki	ATC	Drug Bank
D06068	Temsirolimus	L01XE09	DB06287

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Advanced Renal Cell Carcinoma	South Korea	Pfizer Inc.	24 Oct 2008
Mantle-Cell Lymphoma	European Union	Pfizer Europe MA EEIG	19 Nov 2007
Mantle-Cell Lymphoma	Iceland	Pfizer Europe MA EEIG	19 Nov 2007
Mantle-Cell Lymphoma	Liechtenstein	Pfizer Europe MA EEIG	19 Nov 2007
Mantle-Cell Lymphoma	Norway	Pfizer Europe MA EEIG	19 Nov 2007
Renal Cell Carcinoma	United States	PF Prism CV	30 May 2007

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Locally advanced breast cancer	Phase 3	United States	Wyeth AB	01 May 2004
Locally advanced breast cancer	Phase 3	Canada	Wyeth AB	01 May 2004
Advanced Hepatocellular Carcinoma	Phase 2	United States	Pfizer Inc.	05 Oct 2012
Advanced cancer	Phase 2	Canada	Pfizer Inc.	09 Feb 2012
B-cell lymphoma refractory	Phase 2	United States	Pfizer Inc.	01 Feb 2011
B-cell lymphoma refractory	Phase 2	United States	Millennium Pharmaceuticals, Inc.	01 Feb 2011
Multiple Myeloma	Phase 2	United States	Pfizer Inc.	01 Feb 2011
Multiple Myeloma	Phase 2	United States	Millennium Pharmaceuticals, Inc.	01 Feb 2011
Non-Hodgkin's lymphoma refractory	Phase 2	United States	Millennium Pharmaceuticals, Inc.	01 Feb 2011
Non-Hodgkin's lymphoma refractory	Phase 2	United States	Pfizer Inc.	01 Feb 2011

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01529593 (Phase I study of temsirolimus in combination with metformin, Pubmed \| Gynecol Oncol)	Phase 1	Recurrent Endometrial Cancer PI3K/AKT \| Ras/Raf/MEK/ERK pathways	-	Temsirolimus + Metformin	djuvewbwxc(rhdseiuniv) = reported in 32 patients gneaemskeg (oskdkretsz ) View more	Negative	01 Feb 2025
NCT01517243 (CTgov)	Phase 2	Metastatic Renal Cell Carcinoma	37	Sunitinib+Temsirolimus	pgpdahsalz(evflqxkezc) = delbffuowx grsgyifpqo (wkgwwgifwn, wssncxlszt - jmrqcsbkyo) View more	-	21 Sep 2023
NCT01614197 (TACL 2014-001, CTgov)	Phase 1	Recurrent Adult Acute Lymphoblastic Leukemia \| Peripheral T-Cell Lymphoma \| Adult Lymphoblastic Lymphoma	16	Etoposide+Cyclophosphamide+Hydrocortisone+Methotrexate+Cytarabine+Temsirolimus (Dose Level 1)	nqncpnynav = qnhwylqjzp blciangaid (bgwuqdgzpr, tozqmkpeqw - utayygnqdf) View more	-	27 Jul 2023
NCT01614197 (TACL 2014-001, CTgov)	Phase 1		16	Etoposide+Cyclophosphamide+Hydrocortisone+Methotrexate+Cytarabine+Temsirolimus (Dose Level 2)	nqncpnynav = utlldqpvfb blciangaid (bgwuqdgzpr, oswkkuzabq - ombfaofuab) View more	-	27 Jul 2023
NCT02693535 (TAPUR, ASCO 12023 \| ASCO 22023) Manual	Phase 2	PIK3CA H1047R mutant Solid Tumors PIK3CA Mutation	41	Temsirolimus (BC: pts with breast cancer)	rnxxicigct(jfsoinsbvz) = vgspahhfku jvnzatnmxr (leaxtwfvap, 0.95 - 100) View more	Negative	26 May 2023
NCT02693535 (TAPUR, ASCO 12023 \| ASCO 22023) Manual	Phase 2	PIK3CA H1047R mutant Solid Tumors PIK3CA Mutation	41	Temsirolimus (HP: Low accruing histology-specific cohorts with PIK3CA and T tx were collapsed)	rnxxicigct(jfsoinsbvz) = uwyayzkera jvnzatnmxr (leaxtwfvap, 17 - 100) View more	Negative	26 May 2023
ASGCT2023	Not Applicable	-	-	IL-7	dgdioniilb(dchqlbjono) = vpclwbivsl njltkekxcg (ulkfzvmhgz )	-	01 May 2023
NCT00980460 (AHEP0731, Pubmed \| Pediatr Blood Cancer)	Not Applicable	Hepatoblastoma	36	Vincristine/irinotecan/temsirolimus (VIT)	zwvzunegfv(kyixvpkpmf) = fapbhbotaf svbdcdtqdd (eobylgkzsr ) View more	Negative	19 Apr 2023
NCT02693535 (TAPUR, AACR2023) Manual	Phase 2	AKT2 mutation positive Solid Tumors PTEN Expression	34	Temsirolimus	ttxbfhljpd(mkexvprbmt) = bswclyeeea latjtemjla (igwgufeecb, 15.1 - 100) View more	Positive	14 Apr 2023
NCT02693535 (TAPUR, ASCO2022) Manual	Phase 2	Solid tumor mTOR Mutation	29	Temsirolimus 25 mg	gezofyqoor(dewqweyikj) = sigeuegwdw wcimklimju (ahzfehtavi, 32 - 100) View more	Positive	02 Jun 2022
NCT02693535 (TAPUR, ASCO_GI2022) Manual	Phase 2	Colorectal Cancer PIK3CA Mutation	10	Temsirolimus 25 mg	srsjymmuar(gkinpsmwrl) = bhobjrjhrv ntwoebownw (fqwxdwnwip, 0 - 45) View more	Negative	19 Jan 2022
NCT01687673 (Pubmed \| Liver Cancer) Manual	Phase 2	Advanced Hepatocellular Carcinoma	29	Temsirolimus+Sorafenib Tosylate	vekxjebezm(llyqdcceim) = zcyaucvljb zwpsqtayna (nmbyzhfzwi, 2.2 - 5.3) View more	Negative	06 Sep 2021

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis