Dermatitis, Phototoxic

Dermatologist shines a light on the latest research in photocontact dermatitis SAN DIEGO, March 8, 2024 /PRNewswire/ -- Sun sensitivity and related skin conditions are often misunderstood. Foods, medications, and skincare products can trigger symptoms like itching, redness, blistering, or burning. One of the most common conditions is photocontact dermatitis, a skin reaction occurring when certain substances come into contact with skin that is exposed to the sun. "Sun sensitivity is a common condition that can negatively impact a person's quality of life," said board-certified dermatologist Brandon Adler, MD, FAAD, an assistant professor of dermatology at Keck School of Medicine at the University of Southern California in Los Angeles, speaking at the American Academy of Dermatology's 2024 Annual Meeting. For many people, sun sensitivity is caused by the things they come into contact with, said Dr. Adler. If you develop rashes, blisters, or skin darkening after making a margarita or chopping vegetables for a salad, some of the ingredients may be to blame, said Dr. Adler. Your skin reaction could be caused by handling fruits and vegetables such as lime, figs and celery, or coming into contact with plants like hogweed and St. John's wort. Certain pain relief medications that are applied to the skin may also cause a photosensitive reaction, as can medications taken by mouth, such as some blood pressure medications like hydrochlorothiazide. While medications applied directly to the skin only cause a rash in the area of the body where it was applied, if a medication taken by mouth causes photosensitivity, it can cause a rash over any part of the body that was not protected from the sun. Reactions often appear as an itchy rash or severe sunburn on areas of the body most exposed to the sun, such as the face, neck, arms, or legs, according to Dr. Adler. To determine the cause of sun sensitivity reactions, dermatologists must consider a variety of individual factors, such as various product exposures and medical history, and less frequently bloodwork or a biopsy of the affected area of the skin. "While we will often prescribe anti-inflammatory medications to treat photocontact dermatitis, the primary treatment is identifying and avoiding the irritant or allergen," said Dr. Adler. "In many cases these are reversible reactions, so if the patient stops using the substance causing the reaction, then they will stop having symptoms and won't need ongoing treatment." Many people incorrectly assume that certain types of sun sensitivity only affect older people with lighter skin types, but recent research indicates that younger people and those with darker skin types are more susceptible than previously believed, according to Dr. Adler. New studies suggest that people with darker skin types are susceptible to two other types of photosensitivity, he said. The first is polymorphous light eruption (PMLE), which appears as tiny bumps or rashes that come and go with sun exposure. The second is chronic actinic dermatitis, which causes year-round rashes affecting the sun-exposed parts of the body due to light sensitivity. The exact causes of PMLE and chronic actinic dermatitis are not fully understood, but they are believed to involve an abnormal immune response to sunlight. While it is important for everyone to protect themselves from the sun, Dr. Adler noted that those with sun sensitivity need to be particularly mindful to seek shade, wear sun-protective clothing, and apply a broad-spectrum, water-resistant sunscreen with an SPF of 30 or higher. "If you notice a rash or blistering on your body after being in the sun, it's important to see a board-certified dermatologist, who can determine whether you have a sun-related skin disorder," said Dr. Adler. "No two patients are the same. A board-certified dermatologist can determine what is causing your sun sensitivity and provide a treatment option that works best for your condition." To find a board-certified dermatologist in your area, visit aad.org/findaderm. More Information Eczema Types: Contact Dermatitis Causes AAD B-Roll Library About the AAD Headquartered in Rosemont, Ill., the American Academy of Dermatology, founded in 1938, is the largest, most influential and most representative of all dermatologic associations. With a membership of more than 20,800 physicians worldwide, the AAD is committed to advancing the diagnosis and medical, surgical, and cosmetic treatment of the skin, hair, and nails; advocating high standards in clinical practice, education and research in dermatology; and supporting and enhancing patient care because skin, hair, and nail conditions can have a serious impact on your health and well-being. For more information, contact the AAD at (888) 462-DERM (3376) or aad.org. Follow @AADskin on Facebook, TikTok, Pinterest and YouTube and @AADskin1 on Instagram. SOURCE American Academy of Dermatology

CAMBRIDGE, England--( BUSINESS WIRE )--Napp Pharmaceuticals Limited, a member of the international network of Mundipharma independent associated companies, today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) has authorised rezafungin for the treatment of invasive candidiasis in adults in Great Britain (GB). 1,2 The authorisation was based on positive results from the pivotal ReSTORE Phase III clinical trial which demonstrated statistical non-inferiority for rezafungin, dosed once weekly, when compared to the current standard of care, caspofungin, dosed once daily. 2,3,4 These findings are supported by the STRIVE Phase II clinical trial and an extensive nonclinical development program. 3,4 ReSTORE, a multicentre, prospective, randomised and double-blind Phase III trial compared the efficacy and safety of intravenous rezafungin with intravenous caspofungin in eligible patients with invasive candidiasis. 2,3 The primary efficacy outcome was global response (confirmed by the Data Review Committee [DRC]) at day 14. 3 Global response was determined from clinical response, mycological response, and radiologic response (for qualifying subjects with invasive candidiasis). 2,3 Of the 187 patients in the modified intention-to-treat (mITT) population, 59.1% of patients in the rezafungin group (55/93) and 60.6% of patients in the caspofungin group (57/94) had a global response of cure at day 14 (weighted treatment difference −1.1% [95% CI −14.9, 12.7]. 2,3 Non-inferiority was to be concluded if the lower bound of the 95% confidence internal (CI) for the difference in Day 14 cure rates (rezafungin – caspofungin) was >-20%. 2 Rezafungin was generally well tolerated in clinical trials, to date very common adverse reactions include hypokalaemia, pyrexia, and diarrhoea. Common adverse reactions include anaemia, hypomagnesaemia, hypophosphataemia, hypotension, wheezing, vomiting, nausea, abdominal pain, constipation, erythema, and rash. Increased blood alkaline phosphatase, hepatic enzymes, alanine aminotransferase, aspartate aminotransferase and blood bilirubin were also common. Uncommon adverse events were hyperphosphataemia, hyponatraemia, phototoxicity, tremor and increased eosinophil count. There were spontaneous reports of urticaria (frequency not known). Transient infusion-related reactions have occurred with rezafungin, characterised by flushing, sensation of warmth, nausea, and chest tightness. 2 Rezafungin may cause increased risk of phototoxicity. Eligible patients should be advised to avoid sun exposure and other sources of UV radiation without adequate protection during treatment and for 7 days after the last administration of rezafungin. 2 Invasive candidiasis , is a severe, life-threatening infection of the bloodstream and/or deep/visceral tissues. 10,11 It affects seriously ill people, especially those with a weakened immune system and the mortality rate can be 40% or more. 12,13 It can place a large burden on the healthcare system, with the potential for extended treatment regimens and long hospital stays. 14 In the last 15 years there have been no new treatments and morbidity and mortality rates remain largely the same, indicating the need for alternative treatment options. 5-9,15 Professor P. Lewis White PhD, FECMM, FRCPath, Consultant Clinical Scientist and Head of the PHW Mycology Reference Laboratory, Honorary Professor, Cardiff University Centre for Trials Research said “ Invasive candidiasis remains a difficult-to-treat disease which has a significant impact on critically ill and immunocompromised patients. With limited advances in treating this condition over the past 15 years, today’s announcement is a positive step towards supporting eligible patients and could enable the healthcare professional community an alternative way to manage these patients with invasive candidiasis using a new, once-weekly intravenous treatment option .” “ This authorisation is a culmination of years of developing an additional treatment option for eligible adult patients suffering from invasive candidiasis and underscores our commitment to supporting management of infectious diseases, ” said Dr Ben David, Medical Director for Northern Europe at Mundipharma. Rezafungin has been granted Orphan Drug Designation (ODD) in GB for the treatment of invasive candidiasis in adults. 1 The MHRA grants ODD to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 5 in 10,000 people in GB where no satisfactory method of diagnosis, prevention or treatment of the condition concerned exists, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition. 16 *ENDS* About invasive candidiasis Invasive candidiasis (IC) continues to be an area of significant unmet need, especially for critically ill patients in hospitals and patients with compromised immune systems. 14 Despite a number of available treatments, the mortality rate for patients with invasive candidiasis can be 40% or more. 13,14 IC is characterised as a severe, life-threatening systemic Candida infection of the bloodstream and/or deep/visceral tissues. 17 About Rezafungin Rezafungin is a novel once-weekly echinocandin that has been developed for the treatment of invasive candidiasis in adults. 18 About Napp Napp Pharmaceuticals Limited is part of Mundipharma, a global healthcare company with a presence across Africa, Asia Pacific, Canada, Europe, Latin America, and the Middle East. Mundipharma is dedicated to bringing innovative treatments to patients in the areas of pain management, infectious disease and consumer healthcare as well as other severe and debilitating disease areas. Our guiding principles, centered around Integrity and Patient-Centricity, are at the heart of everything we do. For more information visit www.mundipharma.com Future clinical trial data A second Phase III clinical trial of rezafungin for the prevention of invasive fungal disease in patients undergoing allogeneic blood and marrow transplantation (ReSPECT trial) is currently being conducted. 18 Registered office addresses NAPP PHARMACEUTICALS LIMITED a company having its registered office at Cambridge Science Park, Milton Road, Cambridge CB4 0AB, England. MUNDIPHARMA RESEARCH LIMITED, a company having its registered office at Cambridge Science Park, Milton Road, Cambridge, CB4 0AB, England. ® REZZAYO is a Registered Trademark of Cidara Therapeutics, Inc, used under license. References 1. UK Medicines and Healthcare Products Regulatory Agency authorisation. Available at: https://www.gov.uk/government/news/rezzayo-approved-to-treat-adult-patients-with-invasive-candidiasis (last accessed February 2024) 2. REZZAYO ® Summary of Product Characteristics. 3. Thompson GR, et al. Lancet 2023;401(10370):49–59. 4. Thompson GR, et al. Lancet Infect Dis 2023; https://doi.org/10.1016/S1473-3099(23)00551-0 5. Ham YY, et al. Future Microbiology 2021;16(1):27–36. 6. CANCIDAS (formerly Caspofungin MSD) EPAR. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/cancidas-previously-caspofungin-msd (last accessed February 2024). 7. Vfend (voriconazole) EPAR. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/vfend (last accessed February 2024). 8. Ecalta (anidulafungin) EPAR. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/ecalta (last accessed February 2024). 9. Mycamine (micafungin) EPAR. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/mycamine (last accessed February 2024). 10. Pappas PG, et al. Nat Rev Dis Primers 2018;4:18026. 11. CDC. Invasive candidiasis. Available at: https://www.cdc.gov/fungal/diseases/candidiasis/invasive/definition.html (last accessed February 2024). 12. Kullberg BJ, Arendrup MC. N Engl J Med 2015;373:1445–56. 13. Bassetti M, et al. Crit Care 2019;23(1):219. 14. Cortegiani A, et al. Cochrane Database Syst Rev 2016;2016:Cd004920. 15. Bassetti M, et al. J Antimicrob Chemother 2018; 73 Suppl 1: i14–i25 doi:10.1093/jac/dkx445. 16. UK Medicines and Healthcare Products Regulatory Agency, Guidance – Orphan medicinal products. Available at: https://www.gov.uk/guidance/orphan-medicinal-products-in-great-britain (last accessed February 2024). 17. Cortes JA, Corrales IF. Invasive candidiasis: Epidemiology and Risk Factors. November 2018. Available at https://www.intechopen.com/chapters/64365 (last accessed February 2024). 18. Cidara Therapeutics and Mundipharma Announce Positive Topline Results from the Global Phase 3 Pivotal ReSTORE Trial of Rezafungin for the Treatment of invasive candidiasis. Available at https://www.mundipharma.com/Cidara-Therapeutics-and-Mundipharma-Announce-Positive-Topline-Results-from-the-Global-Phase-3-Pivotal-ReSTORE-Trial-of-Rezafungin-for-the-Treatment-of-Candidemia-and-Invasive-Candidiasis (last accessed February 2024). Job code: UK-RZF-2300053 Date of preparation: February 2024

-- First and only treatment approved by FDA for indolent SM -- CAMBRIDGE, Mass., May 23, 2023 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today announced that NEJM Evidence, a journal from The New England Journal of Medicine Group, has published detailed results from the PIONEER trial of AYVAKIT® (avapritinib) in patients with indolent systemic mastocytosis (ISM). Data reported in the publication show that AYVAKIT achieved statistically significant and clinically meaningful benefits across efficacy measures including disease symptoms and pathological mast cell burden, which continued to improve over time through 48 weeks. In addition, AYVAKIT demonstrated a favorable safety pro to placebo. On May 22, 2023, AYVAKIT became the first and only medicine approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with ISM. AYVAKIT was designed to potently and selectively inhibit KIT D816V, the primary underlying driver of the disease. ISM represents the vast majority of cases of systemic mastocytosis (SM), a rare hematologic disorder that can lead to debilitating symptoms across multiple organ symptoms and a profound impact on patients' quality of life. "Patients with indolent systemic mastocytosis can experience a range of severe, life-altering symptoms and emotional hardships. The disorder can significantly affect patients' ability to serve as active members of their families, workplaces and broader communities, and may lead individuals to 'live in a bubble' at home in an attempt to avoid unpredictable triggers," said Mariana Castells, M.D., Ph.D., Director, Mastocytosis Center, Brigham and Women's Hospital and co-first author of the paper. "Since I began specializing in mast cell disorders several decades ago, I have been waiting for a treatment that targeted the primary root cause of indolent systemic mastocytosis and enabled broad clinical benefits for patients with uncontrolled disease despite significant polypharmacy use. The impact of AYVAKIT across trial endpoints – from mast cell burden to symptoms and quality of life – offers the opportunity to redefine the standard of care in indolent systemic mastocytosis and brings a renewed sense of hope for all those living with the disorder." "This publication features the largest dataset ever reported in indolent systemic mastocytosis and the first positive registrational study for this disease, representing a landmark achievement in our long-term collaboration with clinical experts and the systemic mastocytosis community to advance patient care," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "Our deep commitment, knowledge and understanding of systemic mastocytosis has enabled us to develop a transformative targeted treatment that can significantly improve outcomes for patients. Blueprint Medicines has been pioneering research and development in the field of systemic mastocytosis for over a decade, and following the approval of AYVAKIT in indolent systemic mastocytosis, we are focused on delivering this major therapeutic advancement to a broad range of patients with the disease." Highlights from the NEJM Evidence Publication PIONEER is a randomized, double-blind, placebo-controlled trial in which 141 patients received AYVAKIT 25 mg once daily plus best supportive care (AYVAKIT) and 71 patients received placebo plus best supportive care (placebo). Disease symptoms were assessed using the ISM Symptom Assessment Form (ISM-SAF). As previously reported, AYVAKIT met the primary and all key secondary endpoints, which were evaluated from baseline to 24 weeks, and showed deepening clinical efficacy through 48 weeks of treatment. Overall, 96 percent of patients with AYVAKIT completed 24 weeks of treatment and opted to cross over to the open-label extension study. Key published results include: Patients treated with AYVAKIT achieved rapid, durable and statistically significant reductions on all measures of pathological mast cell burden, including serum tryptase, KIT D816V variant allele fraction and bone marrow mast cells. AYVAKIT achieved statistically significant and clinically meaningful benefits in overall symptoms – as measured by the ISM-SAF Total Symptom Score (TSS) – compared to placebo at 24 weeks, with improvements deepening through 48 weeks. In addition, AYVAKIT showed improvements over placebo on the most severe symptom and across all individual symptoms measured. AYVAKIT showed clinically meaningful improvements in multiple exploratory endpoints of quality of life, including a disease-specific measure – Mastocytosis Quality of Life Questionnaire (MC-QoL) – and general health status measures such as European Quality of Life Visual Analogue Scale (EQ-VAS), Patient Global Impression of Severity (PGIS) and 12-Item Short-Form Health Survey (SF-12). AYVAKIT showed a favorable safety profile, with most adverse events (AEs) mild to moderate in severity. The serious AE rate was 5.0 percent for AYVAKIT and 11.3 percent for placebo. The most common treatment-emergent AEs for AYVAKIT (incidence ≥5 percent and at least twice the rate of placebo) were flushing, edema, increased blood alkaline phosphatase and insomnia. The paper, titled "Avapritinib versus Placebo in Indolent Systemic Mastocytosis," was published in NEJM Evidence on May 23, 2023 and can be found here: . About AYVAKIT (avapritinib) AYVAKIT (avapritinib) is a precision therapy approved by the FDA for the treatment of three indications: adults with ISM, adults with advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL), and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. For more information, visit AYVAKIT.com. This medicine is approved in Europe (AYVAKYT®) for the treatment of adults with ASM, SM-AHN or MCL, after at least one systemic therapy, and adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation. Please click here to see the full U.S. Prescribing Information for AYVAKIT, and click here to see the European Summary of Product Characteristics for AYVAKYT. AYVAKIT/AYVAKYT is not approved for the treatment of any other indication in the U.S. or Europe. To learn about ongoing or planned clinical trials, contact Blueprint Medicines at medinfo@blueprintmedicines.com or 1-888-BLU-PRNT (1-888-258-7768). Additional information is available at blueprintclinicaltrials.com or clinicaltrials.gov. About Systemic Mastocytosis Systemic mastocytosis (SM) is a rare disease driven by the KIT D816V mutation in about 95 percent of cases. Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms across multiple organ systems. The vast majority of those affected have indolent systemic mastocytosis (ISM). A broad range of symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, frequently persist in patients with ISM despite treatment with multiple symptom-directed therapies. This burden of disease can lead to a profound, negative impact on quality of life. Patients often live in fear of severe, unexpected symptoms, have limited ability to work or perform daily activities, and isolate themselves to protect against unpredictable triggers. Historically, there had been no approved therapies for the treatment of ISM. A minority of patients have advanced SM, which encompasses a group of high-risk SM subtypes including ASM, SM-AHN and MCL. In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor survival. Important Safety Information Intracranial Hemorrhage—Serious intracranial hemorrhage (ICH) may occur with AYVAKIT treatment; fatal events occurred in <1% of patients. Overall, ICH (eg, subdural hematoma, ICH, and cerebral hemorrhage) occurred in 2.9% of 749 patients who received AYVAKIT in clinical trials. In Advanced SM patients who received AYVAKIT at 200 mg daily, ICH occurred in 2 of 75 patients (2.7%) who had platelet counts ≥50 x 109/L prior to initiation of therapy and in 3 of 80 patients (3.8%) regardless of platelet counts. In ISM patients, no events of ICH occurred in the 246 patients who received any dose of AYVAKIT in the PIONEER study. Monitor patients closely for risk factors of ICH which may include history of vascular aneurysm, ICH or cerebrovascular accident within the prior year, concomitant use of anticoagulant drugs, or thrombocytopenia. Symptoms of ICH may include headache, nausea, vomiting, vision changes, or altered mental status. Advise patients to seek immediate medical attention for signs or symptoms of ICH. Permanently discontinue AYVAKIT if ICH of any grade occurs. In Advanced SM patients, a platelet count must be performed prior to initiating therapy. AYVAKIT is not recommended in Advanced SM patients with platelet counts <50 x 109/L. Following treatment initiation, platelet counts must be performed every 2 weeks for the first 8 weeks. After 8 weeks of treatment, monitor platelet counts every 2 weeks or as clinically indicated based on platelet counts. Manage platelet counts of <50 x 109/L by treatment interruption or dose reduction. Cognitive Effects—Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 33% of 995 patients overall in patients who received AYVAKIT in clinical trials including: 28% of 148 Advanced SM patients (3% were Grade ≥3), and 7.8% of patients with ISM who received AYVAKIT + best supportive care (BSC) versus 7.0% of patients who received placebo + BSC (<1% were Grade 3). Depending on the severity and indication, withhold AYVAKIT and then resume at same dose or at a reduced dose upon improvement, or permanently discontinue. Photosensitivity—AYVAKIT may cause photosensitivity reactions. In all patients treated with AYVAKIT in clinical trials (n=1049), photosensitivity reactions occurred in 2.5% of patients. Advise patients to limit direct ultraviolet exposure during treatment with AYVAKIT and for one week after discontinuation of treatment. Embryo-Fetal Toxicity—AYVAKIT can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use an effective method of contraception during treatment with AYVAKIT and for 6 weeks after the final dose of AYVAKIT. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks after the final dose. Adverse Reactions—The most common adverse reactions (≥20%) in patients with Advanced SM were edema, diarrhea, nausea, and fatigue/asthenia. The most common adverse reactions (≥10%) in patients with ISM were eye edema, dizziness, peripheral edema, and flushing. Drug Interactions—Avoid coadministration of AYVAKIT with strong or moderate CYP3A inhibitors. If coadministration with a moderate CYP3A inhibitor cannot be avoided in patients with Advanced SM, reduce dose of AYVAKIT. Avoid coadministration of AYVAKIT with strong or moderate CYP3A inducers. To report suspected adverse reactions, contact Blueprint Medicines Corporation at 1-888-258-7768 or FDA at 1-800-FDA-1088 or . Please click here to see the full Prescribing Information for AYVAKIT. About the PIONEER Trial PIONEER is a randomized, double-blind, placebo-controlled, registrational trial evaluating AYVAKIT in patients with ISM. The trial includes three parts: dose-finding Part 1, registrational Part 2 and open-label extension Part 3. Key trial endpoints include the change in patient-reported disease symptoms as assessed by the ISM-SAF TSS, patient-reported quality of life, measures of mast cell burden and safety. Patients who completed Part 1 or 2 were eligible to participate in Part 3. All patients receive AYVAKIT treatment during Part 3, including those rolling over from the placebo arm. For more information about the PIONEER trial, please visit (ClinicalTrials.gov Identifier: NCT03731260). About Blueprint Medicines Blueprint Medicines is a global precision therapy company that invents life-changing therapies for people with cancer and blood disorders. Applying an approach that is both precise and agile, we create medicines that selectively target genetic drivers, with the goal of staying one step ahead across stages of disease. Since 2011, we have leveraged our research platform, including expertise in molecular targeting and world-class drug design capabilities, to rapidly and reproducibly translate science into a broad pipeline of precision therapies. Today, we are delivering our approved medicines to patients in the United States and Europe, and we are globally advancing multiple programs for systemic mastocytosis, lung cancer, breast cancer and other genomically defined cancers, and cancer immunotherapy. For more information, visit and follow us on Twitter (@BlueprintMeds) and LinkedIn. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding Blueprint Medicines' views with respect to the implications of the FDA approval of AYVAKIT/AYVAKYT for SM patients; the potential benefits of Blueprint Medicines' current and future approved drugs or drug candidates in treating patients, including expectations regarding the potential of AYVAKIT/AYVAKYT to address SM subtypes; and Blueprint Medicines' strategy, goals and anticipated milestones, business plans and focus. The words "aim," "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks and uncertainties related to our ability and plans in continuing to expand Blueprint Medicines' commercial infrastructure, and successfully launching, marketing and selling current or future approved products; Blueprint Medicines' ability to successfully expand the approved indications for AYVAKIT/AYVAKYT or obtain marketing approval for AYVAKIT/AYVAKYT in additional geographies in the future; the delay of any current or planned clinical trials or the development of our current or future drug candidates; Blueprint Medicines' advancement of multiple early-stage efforts; Blueprint Medicines' ability to successfully demonstrate the safety and efficacy of its drug candidates and gain approval of its drug candidates on a timely basis, if at all; the preclinical and clinical results for Blueprint Medicines' drug candidates, which may not support further development of such drug candidates either as monotherapies or in combination with other agents or may impact the anticipated timing of data or regulatory submissions; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; Blueprint Medicines' ability to obtain, maintain and enforce patent and other intellectual property protection for its products or any drug candidates it is developing; Blueprint Medicines' ability to develop and commercialize companion diagnostic tests for its products or any of its current and future drug candidates; Blueprint Medicines' ability to successfully expand its research platform and the costs thereof; and the success of Blueprint Medicines' current and future collaborations, partnerships or licensing arrangements. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in Blueprint Medicines' filings with the Securities and Exchange Commission (SEC), including its Annual Report on Form 10-K for the year ended December 31, 2022, as filed with the SEC on February 16, 2023, and any other filings that Blueprint Medicines has made or may make with the SEC in the future. Any forward-looking statements contained in this press release represent Blueprint Medicines' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, Blueprint Medicines assumes no obligation to update or revise these forward-looking statements for any reason, even if new information becomes available in the future. Trademarks Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are trademarks of Blueprint Medicines Corporation. Company Codes: NASDAQ-NMS:BPMC