Our preclinical study confirmed that copper accumulation can lead to radioresistance in vitro and in vivo, and reducing the concentration of copper with copper chelator help to overcome radioresistance. Therefore, the investigators plan to carry out a prospective interventional phase II clinical trial to explore the safety and efficacy of penicillamine (a common copper chelator) as a radiosensitizer in the treatment of recurrent head and neck cancer.

Start Date15 Nov 2023

Sponsor / Collaborator

Southern Hospital

IRCT20200407046981N49

/ SuspendedNot ApplicableIIT

Comparative bioequivalence study of the Penicillamine 250-mg Capsules manufactured by Vana Darou Gostar Company with Penicillamine brand capsules (Cupripen®) by Laboratorios Rubio company

Start Date11 Dec 2022

Sponsor / Collaborator-

NCT03957720

/ RecruitingEarly Phase 1IIT

The Individual Therapy for Patients With Wilson's Disease

Based on the genotype characteristics and genotype-phenotype-treatment prognosis data of Chinese WD patients, this study intends to further optimize the treatment regimen of Chinese WD patients and formulate individualized treatment regimens for each genotype, so as to further improve the prognosis of patients.

Start Date15 Mar 2019

Sponsor / Collaborator

Second Affiliated Hospital Zhejiang University College of

100 Clinical Results associated with Penicillamine

100 Translational Medicine associated with Penicillamine

100 Patents (Medical) associated with Penicillamine

5,835

Literatures (Medical) associated with Penicillamine

01 Nov 2025·Neural Regeneration Research

Copper homeostasis and neurodegenerative diseases

Article

Author: Wang, Yuanyuan ; Li, Daidi ; Xu, Kaifei ; Zhang, Feng ; Wang, Guoqing

Copper, one of the most prolific transition metals in the body, is required for normal brain physiological activity and allows various functions to work normally through its range of concentrations. Copper homeostasis is meticulously maintained through a complex network of copper-dependent proteins, including copper transporters (CTR1 and CTR2), the two copper ion transporters the Cu -transporting ATPase 1 (ATP7A) and Cu-transporting beta (ATP7B), and the three copper chaperones ATOX1, CCS, and COX17. Disruptions in copper homeostasis can lead to either the deficiency or accumulation of copper in brain tissue. Emerging evidence suggests that abnormal copper metabolism or copper binding to various proteins, including ceruloplasmin and metallothionein, is involved in the pathogenesis of neurodegenerative disorders. However, the exact mechanisms underlying these processes are not known. Copper is a potent oxidant that increases reactive oxygen species production and promotes oxidative stress. Elevated reactive oxygen species levels may further compromise mitochondrial integrity and cause mitochondrial dysfunction. Reactive oxygen species serve as key signaling molecules in copper-induced neuroinflammation, with elevated levels activating several critical inflammatory pathways. Additionally, copper can bind aberrantly to several neuronal proteins, including alpha-synuclein, tau, superoxide dismutase 1, and huntingtin, thereby inducing neurotoxicity and ultimately cell death. This study focuses on the latest literature evaluating the role of copper in neurodegenerative diseases, with a particular focus on copper-containing metalloenzymes and copper-binding proteins in the regulation of copper homeostasis and their involvement in neurodegenerative disease pathogenesis. By synthesizing the current findings on the functions of copper in oxidative stress, neuroinflammation, mitochondrial dysfunction, and protein misfolding, we aim to elucidate the mechanisms by which copper contributes to a wide range of hereditary and neuronal disorders, such as Wilson’s disease, Menkes’ disease, Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis, Huntington’s disease, and multiple sclerosis. Potential clinically significant therapeutic targets, including superoxide dismutase 1, D-penicillamine, and 5,7-dichloro-2-[(dimethylamino)methyl]-8-hydroxyquinoline, along with their associated therapeutic agents, are further discussed. Ultimately, we collate evidence that copper homeostasis may function in the underlying etiology of several neurodegenerative diseases and offer novel insights into the potential prevention and treatment of these diseases based on copper homeostasis.

01 Mar 2025·Biosensors and Bioelectronics

Multi-metal MOF-on-MOF metal-organic gel-based visual sensing platform for ultrasensitive detection of chlortetracycline and D-penicillamine

Article

Author: Ding, Ping ; Qiu, Yuzhe ; Qu, Xiaolong ; Wu, Pian ; Wang, Bin ; Zhang, Zerun ; Zhang, Tao ; Meng, Zongwu

The widespread presence of antibiotic residues in the environment and food represents a significant threat to human health. In this study, a novel MOF-on-MOF (FCZE) gel with excellent peroxidase activity and strong fluorescence properties successfully synthesized by using Polyvinyl pyrrolidone (PVP) as a cross-linker and H₄BTEC as a ligand. Based on the energy transfer from the FCZE ligand to Eu luminescence, an ultrasensitive fluorescence method was developed for the detection of chlortetracycline (CTC), achieving a limit of detection (LOD) as low as 0.43 nM. Furthermore, the combination of FCZE with the 3,3',5,5'-tetramethylbenzidine (TMB)-hydrogen peroxide (H₂O₂) system enabled sensitive colorimetric detection of D-penicillamine (D-PA) with an LOD of 8.66 nM. Theoretical calculations reveal that the fluorescence quenching of FCZE by CTC is attributed to the inner filter effect of CTC and its suppression of the H₄BTEC excited-state return to the ground state. Furthermore, Fukui function was employed for the first time to clarify the role of D-PA in scavenging free radicals and reducing oxidized TMB (oxTMB), providing a comprehensive understanding of the colorimetric detection mechanism. Additionally, a smartphone-assisted visual detection platform based on FCZE was developed for the detection of CTC and D-PA, with LODs of 0.71 μM and 0.13 μM, respectively. This study highlights the potential of nanomaterials for the simultaneous detection of multiple antibiotics, offering a novel strategy for the rapid and efficient monitoring of antibiotic residues in environmental samples.

01 Feb 2025·Talanta

Cu-NC single-atom nanozymes with peroxidase-like activity for colorimetric detection of d-penicillamine

Article

Author: Yang, Xiupei ; Jiang, Ling ; Xie, Xiaoyi ; Fan, Yuxiu ; Wang, Ya ; Zhang, Chenglin

Most conventional nanozymes have poor specificity and low activity, and designing high-performance nanozymes remains a challenge. In contrast, single-atom nanozymes have high atom utilization and high reactivity. Here, we prepared Cu single-atom nanozymes (Cu-NC) with excellent peroxidase-like activity by high-temperature pyrolysis using Cu as a transition metal source. The introduction of Cu formed the Cu-N_x active site, which accelerated charge transfer between the reactants and the active site and was the key for improving the activity. With Cu-NC as a catalyst, H₂O₂ rapidly oxidized 3,3',5,5'-tetramethylbenzidine (TMB) to oxTMB, and the solution turned blue with strong absorption at 652 nm. Because d-penicillamine (D-PA) can reduce oxTMB or react with reactive oxygen species radicals to inhibit the color reaction, we built a colorimetric sensing platform around Cu-NC for the determination of D-PA and successfully used it for the determination of D-PA in urine samples. This work provides new ideas for the design of high-performance nanozymes and the detection of D-PA in real environments.

News (Medical) associated with Penicillamine

23 Dec 2024

·www.accesswire.com

Telomir Pharmaceuticals Confirms Copper Binding Capabilities of Telomir-1 and Expands Pipeline Into Wilson's Disease

MIAMI, FL / ACCESSWIRE / December 23, 2024 / Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), or the "Company", an emerging leader in age-reversal science, today announced significant findings from its copper binding studies with Telomir-1. This is a promising development as the company explores Telomir-1 for the treatment of Wilson's disease and other copper related disorders. The announcement marks an important step forward in Telomir's mission to address critical unmet medical needs while continuing to explore transformative therapies.Key FindingsRecent preclinical in vitro studies conducted by Recipharm and Smart Assays have demonstrated Telomir-1's exceptional binding affinity for copper ions, highlighting its ability to selectively interact with and regulate copper metabolism. Additionally, Telomir-1's capability to bind and exchange key ions, including copper, iron, and zinc, emphasizes its unique profile and positions it as a promising candidate for further research. These findings form a strong foundation for investigating Telomir-1's potential in addressing Wilson's disease, a rare genetic disorder characterized by excessive copper accumulation that can result in significant damage to the liver, brain, and other vital organs.Wilson's Disease: A Critical Unmet NeedWilson's disease is a rare genetic disorder that affects approximately 1 in 30,000 individuals worldwide. The condition is caused by mutations in the ATP7B gene, which impair the body's ability to properly regulate copper metabolism. As a result, copper accumulates to toxic levels, particularly in the liver and brain, leading to severe and potentially life-threatening complications, including liver failure, neurological damage, and psychiatric disorders. Left untreated, Wilson's disease is fatal.Current treatments for Wilson's disease, such as chelating agents (e.g., penicillamine and trientine) and zinc therapy, are primarily aimed at reducing copper levels in the body. While these treatments can be effective, they come with significant drawbacks, including:Chelating agents often cause severe side effects, including kidney damage, bone marrow suppression, and gastrointestinal issues. Additionally, these drugs require lifelong adherence, which can be burdensome for patients.Zinc therapy works by preventing copper absorption in the gut but can result in side effects like anemia, gastrointestinal discomfort, and reduced effectiveness over time.The limitations of current therapies underscore the urgent need for new treatment options that address the root cause of Wilson's disease while minimizing side effects. Telomir-1's copper binding properties hold the potential to offer a safer, more effective alternative that directly targets the underlying mechanisms of the disease. The designation of Wilson's disease as a rare and life-threatening condition also positions it as a candidate for orphan drug designation, which could accelerate development timelines and provide additional regulatory and financial incentives."These recent groundbreaking results on copper binding and interaction showcase Telomir-1's potential to address a significant unmet need in Wilson's disease by enabling beneficial regulations at the underlying causes rather than just managing the symptoms of the disease," said Dr. Itzchak Angel, Chief Scientific Advisor at Telomir. "This is a pivotal moment as we work to bring transformative therapies to patients who need them most."Update on Previous Osteoarthritis StudyEarlier this year, Telomir partnered with Midwest Veterinary Services to launch a planned large-scale study on osteoarthritis in dogs. While only the safety phase of the study was completed-showing no adverse effects-the Company chose to discontinue the study. This decision was based on the requirement to induce osteoarthritis in the dogs, a practice that conflicted with the new management's commitment to the Company's mission and values. Instead, the Company is now in discussions with a well-known veterinary institution to design a new study that will recruit elderly dogs who naturally suffer from osteoarthritis. As such, Telomir is committed to obtaining an INAD (Investigational New Animal Drug) application for pets while progressing toward an IND (Investigational New Drug) application for humans.Expanding Research and DevelopmentBuilding on these results, Telomir is planning and advancing studies to expand the therapeutic potential of Telomir-1. Key ongoing initiatives include:Wilson's Disease Study: Investigating Telomir-1's effects on copper regulation in preclinical models.Type 2 Diabetes Studies: After the success achieved with the zebrafish model, Telomir is currently conducting a rat diabetic model to further strengthen the data on Telomir-1's efficacy in reversing key metabolic parameters.Progeria Research: Examining Telomir-1's impact on accelerated aging and telomere function.Cancer Models: Exploring anti-cancer applications using xenograft studies.Alzheimer's Disease: Investigating Telomir-1 for its potential to mitigate cognitive decline and neurodegeneration associated with Alzheimer's.Metal Toxicity: Exploring Telomir-1's role in addressing metal toxicity, which occurs when metals like copper, iron, or lead accumulate to harmful levels in the body. Metal toxicity can disrupt cellular function, lead to oxidative stress, and contribute to chronic and age-related diseases. By regulating metal levels, Telomir-1 aims to restore cellular balance and reduce associated health risks.DNA Methylation Analysis: Investigating Telomir-1's role in modulating DNA methylation rates, which influence gene expression and play a key role in aging and disease progression.DNA methylation, the addition of chemical groups to DNA, can disrupt normal gene function when dysregulated, contributing to various chronic and age-related diseases. By addressing these epigenetic changes, Telomir-1 aims to restore healthy gene expression patterns.Clinical Progress and Future MilestonesBuilding on our preclinical success demonstrating the potential for age reversal, improved longevity, and health span, as well as the reversal of key Type 2 diabetes parameters, the Company is committed to unlocking the full potential of Telomir-1 to revolutionize the treatment of age-related diseases by addressing their root causes. The possibilities are vast, and we believe this drug holds extraordinary promise to potentially transform the pharmaceutical industry.As we continue to gather scientific data and delve deeper into the molecular mechanisms of action of Telomir-1, we are positioning ourselves to identify the most impactful indication for our initial IND application. The Company is currently working on the optimization of manufacturing processes to enable an affordable and scalable approach. This will support the production of GMP-grade quantities necessary for IND-enabling studies and clinical trials. The Company plans to carry out the IND-enabling safety studies in Q2 2025, targeting IND submission by Q4 2025, with first-in-human clinical trials anticipated in the first half of 2026. The company also plans to secure an INAD for pets, paving the way for Telomir-1's dual application in both human and veterinary medicine.Market PotentialThe global anti-aging drugs market is experiencing significant growth, driven by increasing demand for treatments that address age-related diseases and promote longevity. In 2024, the market was valued at approximately USD 91.05 billion and is projected to reach USD 160.24 billion by 2031, growing at a compound annual growth rate (CAGR) of 7.32% during this period. This expansion reflects a rising consumer focus on health and wellness, advancements in medical research, and a growing aging population seeking solutions to enhance quality of life. (Verified Market Research)Telomir-1 represents a novel approach by targeting the root causes of age-related diseases rather than simply managing symptoms. By developing therapies that extend health span and enhance quality of life, the Company seeks to redefine patient care and make a meaningful impact in this rapidly growing multi-billion-dollar market.Leadership Perspective"As we look to 2025, Telomir remains dedicated to its mission," said Erez Aminov, Chairman and CEO of the Company. "We are pleased to have secured additional financing at a 20% premium with no warrants, providing the resources needed to continue advancing Telomir-1's development while focusing on creating value for our shareholders."About Telomir Pharmaceuticals, Inc.Telomir Pharmaceuticals, Inc. (Nasdaq:TELO) is a pre-clinical stage pharmaceutical company seeking to lead development in several areas, including age-reversal science. Telomir is focused on the development of Telomir-1, a novel small molecule designed to lengthen the DNA's protective telomere caps, which are crucial in the aging process. Telomir's goal is to explore the potential of Telomir-1 starting with ongoing research in animals and then in humans.Telomeres are the protective end caps of a chromosome made up of DNA sequences and proteins. As humans age, telomeres shorten, with metal reactivity accelerating the process, which presents humans and pet animals with an increased chance of contracting a number of degenerative and age-related diseases. Telomir's goal is to develop and gain regulatory approval for Telomir-1, proposed to be dosed orally, with the broader aim of promoting longevity and enhancing overall quality of life.The Nobel Assembly at Karolinska Institute (Sweden) awarded the Nobel Prize in Physiology or Medicine in 2009 for the discovery of how chromosomes are protected by telomeres and the enzyme telomerase.Telomir-1 is in preclinical development and has not yet been tested in humans. There is no assurance that Telomir-1 will proceed through development or will ultimately receive FDA approval for marketing.Cautionary Note Regarding Forward-Looking StatementsThis press release, statements of Telomir Pharmaceuticals' management or advisors related thereto, and the statements contained in the news story linked in this release contain "forward-looking statements," which are statements other than historical facts made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements may be identified by words such as "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding (i) the anticipated benefits of the preclinical testing results described herein, (ii) anticipated timelines and subject matter for additional preclinical and clinical testing of Telomir-1 and (iii) the potential therapeutic benefits of Telomir-1 generally.Any forward-looking statements in this press release are based on Telomir's current expectations, estimates and projections only as of the date of this release and are subject to a number of significant risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the potential use of the data from our studies, our ability to develop and commercialize Telomir-1 for specific indications and safety of Telomir-1. These and other risks concerning Telomir's programs and operations are described in additional detail in its Annual Report on Form 10-K for the fiscal year ended December 31, 2023, which is on file with the SEC. Telomir explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.Telomir's lead compound, Telomir-1, is being investigated for its potential to address a range of conditions, including Type 2 diabetes, Wilson's disease, progeria, Alzheimer's disease, and cancer. The company's innovative approach aims to treat the root causes of disease, offering a promising path forward in improving patient care.For more information, visit www.telomirpharma.com.Contact InformationHelga Moya [email protected](786) 396-6723SOURCE: Telomir Pharmaceuticals, Inc.

Orphan Drug

28 Oct 2024

·www.globenewswire.com

Monopar Announces Agreement with Alexion, AstraZeneca Rare Disease For Late-Stage Wilson Disease Drug Candidate

Oct. 24, 2024 -- Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage biotechnology company focused on developing innovative treatments for patients with unmet medical needs, today announced that it has entered into an agreement with Alexion, AstraZeneca Rare Disease for an exclusive worldwide license to ALXN-1840 (bis-choline tetrathiomolybdate), a drug candidate for Wilson disease that Alexion has progressed through a Phase 3 clinical trial that met its primary endpoint. Monopar will be responsible for all future global development and commercialization activities. Chandler D. Robinson, MD, Co-Founder and Chief Executive Officer of Monopar previously researched tetrathiomolybdate at the laboratory bench, published his results in Science, and helped launch a company around what became known as ALXN-1840. The focus was on Wilson disease. Dr. Robinson came to know the Wilson disease community well through his efforts. At the Wilson Disease Association’s request, he delivered the keynote address at their 2013 Annual Conference celebrating their 30th anniversary, sharing his experience helping advance ALXN-1840 from the laboratory bench to patients. In 2023, Alexion terminated the ALXN-1840 program in Wilson disease based on review of results from Phase II mechanistic trials and discussions with regulatory authorities. Chris Starr, PhD, Co-Founder and Executive Chair of Monopar said, “Upon the 2023 announcement, Chandler, due to his long history with the program and the continued high level of unmet medical need, was contacted by Wilson disease patients, executives and board members of the Wilson Disease Association, as well as physicians regarding the potential for Monopar to obtain rights to ALXN-1840. Due in no small measure to the testimonials Chandler received from clinical trial patients who reported benefit while on the drug for years, we decided that this was an opportunity Monopar needed to pursue, and it fits well with my rare disease drug development and commercialization background as well as Chandler’s background.” Dr. Starr also previously co-founded the orphan drug companies BioMarin and Raptor Pharma (acquired by Horizon Pharma, now a part of Amgen). Dr. Robinson commented, “Alexion has generated a substantial clinical data package on ALXN-1840, including a completed Pivotal Phase 3 clinical trial. The medical data gathered from Alexion’s clinical trials furthers our understanding of Wilson disease and stands to benefit this community.” Under the terms of the license agreement, Monopar will pay Alexion an upfront in the form of a cash payment and equity in Monopar. Future payments are based on tiered royalties on net sales and pre-determined regulatory and sales milestones. “We are excited to have Alexion and AstraZeneca as partners of Monopar as, in addition to Alexion’s work in Wilson disease, AstraZeneca maintains a significant presence in the radiopharma field, in which Monopar is committed to continue growing as Monopar recently announced positive human clinical data with our novel radiopharma program (link),” stated Andrew Cittadine, Chief Operating Officer of Monopar. Wilson disease is a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised. 1 It affects one in 30,000 live births in the US.1 Over time this results in the build-up of toxic copper levels in the liver, brain, and other organs, leading to damage that greatly impacts a patient’s life. 1 Patients can develop a wide range of symptoms, including liver disease and/or psychiatric or neurological symptoms, such as personality changes, tremors and difficulty walking, swallowing or talking. 1 In some cases, the damage and loss of function may be irreversible. 1,2,3 ALXN-1840 (bis-choline tetrathiomolybdate) is an investigational once-daily, oral medicine in development for the treatment of Wilson disease. This novel molecule is designed to selectively and tightly bind and remove copper from the body’s tissues and blood. ALXN-1840 has been granted Orphan Drug Designation in the United States and orphan designation in the European Union for Wilson disease. The FoCus trial was a pivotal Phase 3, randomized, rater-blinded, multi-center clinical trial designed to evaluate the efficacy and safety of ALXN-1840 versus standard-of-care (SoC) in patients with Wilson disease aged 12 years and older. The primary endpoint assessed copper mobilization over 48 weeks, defined as daily mean AUEC (Area Under the Effect Curve) for dNCC (directly measured non-ceruloplasmin-bound copper). In the trial, 214 patients were enrolled in one of two cohorts on a 3:1 basis (treatment-experienced:treatment-naïve). Each cohort was then randomized 2:1 (ALXN1840:SoC). The first cohort enrolled 161 patients who received SoC (chelation therapy with penicillamine or trientine, zinc therapy or a combination of both chelation and zinc therapy) for more than 28 days and the second cohort enrolled 53 patients who were treatment-naïve or had received SoC for 28 days or less. The FoCus trial met its primary endpoint demonstrating three-times greater copper mobilization from tissues compared to the SoC arm (Least Square Mean Difference [LSM Diff] 2.18 µmol/L; p Monopar Therapeutics is a clinical-stage biotechnology company with late-stage ALXN-1840 for Wilson disease, and radiopharma programs including Phase 1-stage MNPR-101-Zr for imaging advanced cancers, and Phase 1a-stage MNPR-101-Lu and late preclinical-stage MNPR-101-Ac225 for the treatment of advanced cancers. For more information, visit: www.monopartx.com. References: 1. Patil, M., et al. (2013) J Clin Exp Hepatol, 3, 321-336. 2. Roberts, E.A., Schilsky, M.L. American Association for the Study of Liver D. (2008). Diagnosis and treatment of Wilson disease: An update. Hepatology, 47(6), 2089-2111. 3. European Association for the Study of the Liver. (2012). EASL clinical practice guidelines: Wilson’s disease. J Hepatol, 56(3), 671-685. The content above comes from the network. if any infringement, please contact us to modify.

Clinical ResultLicense out/inOrphan Drug

19 Aug 2024

·www.prnewswire.com

Behind The Mystery Unveils Treatment in Wilson Disease with Cuviror® by Orphalan

Wilson Disease, a rare, genetic disorder, hampers the body's ability to eliminate copper, leading to serious health issues. Cuvrior® (trientine tetrahydrochloride) is the only FDA-approved treatment in nearly 30 years. FORT LAUDERDALE, Fla., Aug. 19, 2024 /PRNewswire/ -- This special episode of Behind the Mystery features insightful discussions with a dedicated physician, an advocacy leader, and a CEO whose company has pioneered the first FDA approved treatment for Wilson Disease in nearly 30 years. Mandy, diagnosed with Wilson Disease at a young age, shares her journey living with the condition on the show, addressing the challenges faced by patients in managing their treatment regimens and the need for more convenient options. In addition, Hepatologist Dr. Cheung delves into the complexities of Wilson Disease, including its genetic origins, symptoms, and the critical importance of early diagnosis, management, and adherence to treatment. "I'm proud to be part of the 'Behind the Mystery' team, where we use our platform to raise awareness for the rare disease community. Our series underscored the immense challenges faced by patients, as well as the dedication of physicians, the pharmaceutical industry, and advocacy leaders to provide meaningful solutions to the community," said Senior Writer and Producer of Behind the Mystery, Brittany Cocilova. Our latest episode highlights Orphalan's commitment to developing the only FDA-approved therapy for Wilson Disease in nearly 30 years, showcasing the continued efforts to help patients living with and managing their rare disease. Hosts Montel Williams and Olga Villaverde spotlight the Wilson Disease Association's (WDA) efforts to support patients, improve awareness, and facilitate access to developments. Rhonda Rowland, VP of the WDA, discusses the evolution of treatment options and the organization's role in fostering partnerships between patients, physicians, and the pharmaceutical industry. "Orphalan has a tremendous commitment to educating not only the Wilson Disease community and the general public, but also physicians," says Rhonda Rowland, Vice President of Wilson Disease Association. "We've seen a partnership evolve between advocacy and the pharmaceutical industry which enables an open line of communication addressing the unmet needs in our community." Dr. Naseem Amin, CEO of Orphalan, details how Orphalan is addressing unmet needs in the patient community and emphasizes the importance of empathy in the pharmaceutical industry. CUVRIOR®, a treatment marketed by Orphalan, offers a twice-daily dosing and is a non-refrigerated option for long-term maintenance therapy for Wilson Disease. Behind the Mystery - Wilson Disease will air on Lifetime on August 19th and August 27th at 7:30 AM ET/PT and will remain on the website. By sharing these intimate narratives, the series aims to educate the public, inspire empathy, and advocate for better research, treatments, and support systems. INDICATION CUVRIOR is a copper chelator indicated for the treatment of adult patients with stable Wilson Disease who are de-coppered and tolerant to penicillamine. IMPORTANT SAFETY INFORMATION Do not use CUVRIOR if you are allergic to trientine or to any of the ingredients in CUVRIOR. Your Wilson's Disease symptoms could get worse when you start treatment. This could happen because too much copper is removed from the body in a short period of time. Your doctor may need to reduce your dose or stop CUVRIOR treatment. Copper deficiency may develop following treatment with CUVRIOR. Your doctor will do tests to monitor your urine and blood for copper. Iron deficiency may develop while taking CUVRIOR. If this happens, your doctor may tell you to take iron supplements for a limited time. Allergic reactions, such as a rash, have been reported with trientine, the active ingredient in CUVRIOR. If a rash or other allergic reaction occurs, stop taking CUVRIOR and get emergency medical help. The most common side effects are pain in the abdomen, change in bowel habits, rash, hair loss (alopecia), and mood swings. Take CUVRIOR at least 1 hour apart from any other oral medicine. Tell your doctor about all the medicines you're taking, including over-the-counter medicines, vitamins, and herbal supplements. Avoid taking supplements when taking CUVRIOR. Taking CUVRIOR with mineral supplements (e.g., iron, zinc, calcium, magnesium) can reduce the effectiveness of CUVRIOR. If iron supplementation is necessary, take CUVRIOR at least 2 hours before or 2 hours after taking iron; for other mineral supplements (e.g., zinc, calcium, magnesium), take CUVRIOR at least 1 hour before or 2 hours after. Do not start or stop any medicines while taking CUVRIOR without talking to your doctor. These are not all the possible side effects of CUVRIOR. You are encouraged to report negative side effects of prescription drugs. Contact Orphalan at 1-800-961-8320 or [email protected] or FDA at 1-800-FDA-1088 or medwatch. Please see full Prescribing Information at: About Behind the Mystery: Behind the Mystery™ is the first and only television series dedicated to raising awareness about rare and genetic diseases through powerful patient storytelling. Each episode delves into the personal journeys of individuals and families affected by these conditions, highlighting their struggles, triumphs, and daily realities. By sharing these intimate narratives, the series aims to educate the public, inspire empathy, and advocate for better research, treatments, and support systems. Behind the Mystery not only sheds light on the medical aspects of rare diseases but also celebrates the resilience and strength of the human spirit. Join us weekday mornings on Lifetime®. About Orphalan Orphalan is an international orphan drug development and commercialization company headquartered in Paris. Founded in 2011, the company develops innovative therapies for people living with rare and debilitating diseases. Our trientine tetrahydrochloride product for the treatment of Wilson disease is approved and available in more than 20 countries, branded as CUPRIOR® in EU, the UK, Saudi Arabia, Switzerland, Columbia and China, and as CUVRIOR® in the United States. For more information visit and follow us on LinkedIn. SOURCE Behind The Mystery

Orphan DrugDrug Approval

100 Deals associated with Penicillamine

External Link

KEGG	Wiki	ATC	Drug Bank
D00496	Penicillamine	M01CC01	DB00859

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Heavy Metal Poisoning	CN	Sichuan Pharmaceutical Co., Ltd.	01 Jan 1981
Lead Poisoning	CN	Sichuan Pharmaceutical Co., Ltd.	01 Jan 1981
Cystinuria	US	Bausch Health Cos., Inc.	04 Dec 1970
Hepatolenticular Degeneration	US	Bausch Health Cos., Inc.	04 Dec 1970
Rheumatoid Arthritis	US	Bausch Health Cos., Inc.	04 Dec 1970

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03539952 (EASL2021)	Phase 3	Hepatolenticular Degeneration Maintenance non-ceruloplasmin copper;(NCC)	53	D-penicillamine	qceknmlyko(ikhntwcokg) = osvhahlnhh stnhuuetww (mbqnayguoa )	Positive	23 Jun 2021
				TETA4	qceknmlyko(ikhntwcokg) = darrpradyy stnhuuetww (mbqnayguoa )
ASN2020	Not Applicable	Hepatolenticular Degeneration antimyeloperoxidase(MPO)-ANCA \| antinuclear factors \| anti- proteinase(PR3) antibody ... View more	-	D-Penicillamine	zadwxuedib(ugpuxdpakt) = Oral zinc acetate was once truncated due to gastroenterological side effect scddtgenbl (ehrkuyosup )	-	19 Oct 2020
				Methyl-prednisolone
MDS2020	Not Applicable	-	-	D-penicillamine	eszntnwvgu(pkxetvawmq) = vhfwcctlvj uiihrqgycb (kdhvbincyu ) View more	Negative	12 Sep 2020
EAN2015	Not Applicable	Hepatolenticular Degeneration	33	d-penicillamine (Compliant patients)	hohioxdsyp(dkdjfdoacf) = yylykhusal qsrklbvutz (nitbcsciuv )	Positive	29 Jun 2015
				d-penicillamine (Non-compliant patients)	hohioxdsyp(dkdjfdoacf) = onjzwxasir qsrklbvutz (nitbcsciuv )
EULAR2014	Not Applicable	Eosinophilic Fasciitis	16	D-Penicillamine plus Corticosteroids	(oqkjgnsmsf) = proteinuria was unexpectedly common in both groups although it was more frequent in the D-Pen plus CS group amlhezotcm (fwzcawfptw ) View more	-	11 Jun 2014
				Corticosteroids alone
ASN2011	Not Applicable	Toxicity \| Renal cyst	-	D-Penicillamine	aqcndxdmha(flhypfphfv) = including loosening of the skin, keratoconus, popliteal aneurysms, cutix laxa, cardiomyopathy, restrictive lung disease, thickening of the walls of the esophagus and duodenum rajipbctyt (vbraninsez ) View more	-	08 Nov 2011

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Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis

Penicillamine

Overview

Basic Info

Structure/Sequence

Related

External Link

R&D Status

Clinical Result

Translational Medicine

Deal

Core Patent

Clinical Trial

Approval

Regulation