Multiple Sclerosis Market to Grow Rapidly at a CAGR of 3.8% During the Forecast Period (2025-2034)

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The multiple sclerosis market is growing due to rising prevalence, earlier diagnosis, and wider access to care. Demand is fueled by advancements in DMTs and emerging BTK inhibitors, which offer targeted, high-efficacy options, especially for progressive forms with limited existing therapies.
LAS VEGAS, June 11, 2025 /PRNewswire/ -- Multiple sclerosis is a long-term autoimmune disorder in which the immune system mistakenly attacks the protective sheath surrounding nerves in the brain and spinal cord. This interference disrupts the flow of signals between the brain and the rest of the body, leading to symptoms such as fatigue, muscle weakness, vision disturbances, and difficulty with mobility.
The severity and progression of multiple sclerosis can vary greatly among individuals. As of 2024, there were an estimated 1.6 million diagnosed cases of multiple sclerosis across the 7MM. This number is expected to increase at a CAGR of 1.4% over the forecast period from 2025 to 2034.
The therapeutic landscape for multiple sclerosis has undergone a significant transformation, spurred by progress in both drug-based and supportive treatment approaches. Although MS remains without a cure, the market is seeing an expanding array of disease-modifying therapies (DMTs), greater personalization of care, and innovations in how treatments are delivered—all aimed at enhancing patient outcomes and quality of life.
Pharmacologically, multiple sclerosis treatment involves both immediate and long-term strategies. For managing acute relapses, high-dose intravenous corticosteroids, most commonly methylprednisolone, continue to be the primary treatment, usually given over 3 to 5 days and followed by a course of tapering oral steroids.
Learn more about the multiple sclerosis therapeutics market @ Multiple Sclerosis Drugs Market
Intravenous corticosteroids, such as methylprednisolone, are effective for treating acute multiple sclerosis relapses but do not impact the long-term course of the disease. Ongoing disease management relies on disease-modifying therapies (DMTs), selected based on the severity of MS and individual patient requirements, to reduce relapse frequency and delay disability progression.
While older injectable multiple sclerosis treatments like PLEGRIDY are still used, newer oral options like MAYZENT, VUMERITY, and MAVENCLAD provide greater convenience and may improve patient adherence. High-efficacy monoclonal antibodies (mAbs), including OCREVUS (now also available in a subcutaneous form), KESIMPTA, and BRIUMVI, are increasingly preferred for home-based therapy. Although LEMTRADA is a potent option, its use is restricted due to safety risks and its inclusion in the REMS program.
As key multiple sclerosis drugs such as TYSABRI, TECFIDERA, AUBAGIO, and GILENYA face generic competition, pricing and access are being reshaped. In response, originator companies are shifting focus to next-generation treatments and improved formulations, such as subcutaneous versions and enhanced safety features, to retain market share.
Supportive therapies also play a critical role; rehabilitation via exercise and physiotherapy can improve mobility and reduce fatigue, especially in progressive multiple sclerosis. Cognitive behavioral therapy (CBT) and mindfulness strategies help manage mood disorders like depression and anxiety, which in turn support treatment adherence.
Additionally, lifestyle modifications involving diet, sleep hygiene, and vitamin D intake aid in symptom control. Assistive devices and occupational therapy further promote independence and enhance quality of life.
Find out more on FDA-approved multiple sclerosis medication @ Multiple Sclerosis Treatments
Multiple sclerosis treatment lacks neuroprotective and remyelinating therapies, with limited options for progressive multiple sclerosis. Current drugs focus on immune modulation but fail to reverse damage or halt progression. Diagnostic delays remain common due to nonspecific symptoms and limited biomarkers. There is a critical need for early detection tools, personalized therapies, and agents targeting neurodegeneration to improve outcomes in multiple sclerosis.
The multiple sclerosis treatment pipeline remains robust, with promising candidates like Remibrutinib, Tolebrutinib, Fenebrutinib, and CNM-Au8 targeting novel mechanisms such as BTK inhibition and neuroprotection.
Other multiple sclerosis drugs in the pipeline include Frexalimab (SAR441344) (Sanofi), Vidofludimus calcium (IMU-838) (Immunic Therapeutics), Masitinib (AB Science), PIPE-307 (Contineum Therapeutics), and others.
These multiple sclerosis pipeline therapies reflect an increasing emphasis on progressive multiple sclerosis and remyelination to improve long-term outcomes.
Discover which therapies are expected to grab major multiple sclerosis market share @ Multiple Sclerosis Treatment Market
Tolebrutinib is an oral BTK inhibitor designed to penetrate the blood–brain barrier, targeting both B cells and microglia to mitigate smoldering neuroinflammation in multiple sclerosis (MS). By focusing on central nervous system (CNS) processes that drive neurodegeneration, it aims to address disease progression beyond peripheral inflammation. In March 2025, the US FDA accepted tolebrutinib for priority review as a treatment for non-relapsing secondary progressive multiple sclerosis, to slow relapse-independent disability progression. A decision is anticipated by September 2025, with a parallel evaluation ongoing in the EU.
Vidofludimus calcium is an investigational oral therapy currently in Phase III trials for relapsing multiple sclerosis and Phase II for progressive multiple sclerosis. It combines DHODH inhibition for immune regulation with Nurr1 activation for potential neuroprotective effects, aiming to target key mechanisms of neurodegeneration. In April 2025, Immunic reported positive outcomes from the Phase II CALLIPER trial, supporting vidofludimus calcium's role as a Nurr1 activator in progressive multiple sclerosis patients.
Fenebrutinib is an innovative, oral, reversible, non-covalent BTK inhibitor in Phase III development for MS. By modulating both adaptive and innate immune responses via B cells and microglia, it offers a dual approach to controlling disease activity and slowing disability. It is currently the only reversible BTK inhibitor in late-stage multiple sclerosis trials.
The Phase III program includes FENhance 1 and 2 studies in relapsing multiple sclerosis (vs. teriflunomide) and the FENtrepid trial in primary progressive multiple sclerosis (vs. ocrelizumab), marking the first head-to-head comparison of a BTK inhibitor with an approved PPMS therapy. Regulatory submissions for both RMS and PPMS are planned for 2026. In September 2024, Roche presented 48-week results from the FENopta Phase II open-label extension at the 40th ECTRIMS Congress.
Remibrutinib is an oral covalent BTK inhibitor under investigation for multiple sclerosis and chronic spontaneous urticaria (CSU), where it blocks BTK signaling to prevent histamine release. While no Phase I or II trials have yet been conducted for multiple sclerosis, remibrutinib is in Phase III development for CSU through the REMODEL-1 and REMODEL-2 trials. Primary completion is expected in 2026, with regulatory submission anticipated in 2027. Earlier Phase IIb CSU data showed favorable safety and tolerability.
CNM-Au8 is an oral gold nanocrystal therapy designed to address mitochondrial dysfunction in multiple sclerosis by boosting NAD+ production, enhancing antioxidant capacity, and activating the heat shock factor-1 pathway to support protein homeostasis. Although the VISIONARY-MS Phase II trial was halted early due to COVID-19, CNM-Au8 is being evaluated in the ongoing Phase II REPAIR-MS trial. Results from REPAIR-MS, along with a planned FDA End-of-Phase II meeting in 2025, will inform the design of a global Phase III trial. In April 2025, Clene Nanomedicine presented promising new data at the AAN Annual Meeting, showing meaningful improvements in cognition and visual function from the VISIONARY-MS open-label extension.
Masitinib is being developed for progressive multiple sclerosis with a focus on inhibiting mast cells and microglia, key players in chronic neuroinflammation. This approach aims to slow progression in patients with primary progressive multiple sclerosis (PPMS) and non-active secondary progressive multiple sclerosis (SPMS). Following positive results from a Phase IIb/III study, a confirmatory Phase III trial is ongoing. In September 2024, AB Science provided an update on masitinib's development at the ECTRIMS 2024 conference.
Discover more about the multiple sclerosis pipeline @ Multiple Sclerosis Clinical Trials
The anticipated launch of these emerging therapies for multiple sclerosis are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the multiple sclerosis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.
DelveInsight estimates that the market size for Multiple Sclerosis is expected to grow from USD 20 billion in the 7MM in 2024 at a 3.8% CAGR by 2034. This expansion across the 7MM will be driven by the introduction of innovative therapies, remibrutinib, tolebrutinib, fenebrutinib, frexalimab, vidofludimus calcium, CNM-Au8, and masitinib, among others. Furthermore, multiple sclerosis prevalence is rising due to modifiable environmental and lifestyle factors like Epstein-Barr virus, low vitamin D, smoking, obesity, and night shifts, with genetics and geography also contributing.
DelveInsight's latest published market report, titled Multiple Sclerosis – Market Insight, Epidemiology, and Market Forecast – 2034, will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the multiple sclerosis country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The multiple sclerosis market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into:
Total Diagnosed Prevalent Cases of Multiple Sclerosis
Gender-specific Diagnosed Prevalent Cases of Multiple Sclerosis
Phenotype-specific Diagnosed Prevalent Cases of Multiple Sclerosis
EDSS-specific Diagnosed Prevalent Cases of Multiple Sclerosis
The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM multiple sclerosis market. Highlights include:
10-year Forecast
7MM Analysis
Epidemiology-based Market Forecasting
Historical and Forecasted Market Analysis upto 2034
Emerging Drug Market Uptake
Peak Sales Analysis
Key Cross Competition Analysis
Industry Expert's Opinion
Access and Reimbursement
Download this multiple sclerosis market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the multiple sclerosis market. Also, stay abreast of the mitigating factors to improve your market position in the multiple sclerosis therapeutic space.
Related Reports
Multiple Sclerosis Epidemiology Forecast
Multiple Sclerosis Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted multiple sclerosis epidemiology in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, Spain), the United Kingdom, and Japan.
Multiple Sclerosis Pipeline
Multiple Sclerosis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key multiple sclerosis companies, including Sanofi, Immunic, InnoCare Pharma, Tiziana Life Sciences, Biogen, Bristol-Myers Squibb, ImStem Biotechnology, Idorsia Pharmaceuticals, among others.
Relapsing-Remitting Multiple Sclerosis Market
Relapsing-Remitting Multiple Sclerosis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key RRMS companies including Mapi Pharma, Apimeds, Genentech (Roche), Merck, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, among others
Relapsing-Remitting Multiple Sclerosis Pipeline
Relapsing-Remitting Multiple Sclerosis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key RRMS companies, including TG Therapeutics, Novartis, Sanofi, CinnaGen, Immunic, Mapi Pharma, Apimeds, Genentech (Roche), Merck, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, GlaxoSmithKline, among others.
About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve.
Contact UsShruti Thakur info@delveinsight.com +14699457679
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Cancer Pain Market Set to Transform by 2034 Due to Launch of Breakthrough Therapies

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Cancer Pain Market Set to Transform by 2034 Due to Launch of Breakthrough Therapies

According to DelveInsight's analysis, the growth of the cancer pain market is expected to be mainly driven by increasing incidence and advances in disease mechanisms have yielded new diagnostic and therapeutic approaches, opening the way to more drug development. The market is expected to show positive growth, mainly attributed to the increasing cases and also, the launch of upcoming therapies during the forecast period. LAS VEGAS, June 12, 2025 /PRNewswire/ -- DelveInsight's Cancer Pain Market Insights report includes a comprehensive understanding of current treatment practices, cancer pain emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Cancer Pain Market Report According to DelveInsight's analysis, the market size for cancer pain is expected to rise at a significant CAGR by 2034. The United States is expected to account for the highest market size of cancer pain, of the total market size in 7MM in comparison to the other major markets, i.e., EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan. According to DelveInsight's analysis, the total cancer pain cases in 2023 for the US were 2.4 million. In 2023, based on the pathophysiology of cancer pain, nociceptive pain accounted for the highest number of cases in the US. Leading cancer pain companies developing emerging therapies, such as InhaleRX, Dogwood Therapeutics, WinSanTor, AlgoTherapeutix, Artelo Biosciences, Asahi Kasei Pharma, and others, are developing novel cancer pain drugs that can be available in the cancer pain market in the coming years. The promising cancer pain therapies in the pipeline include IRX211, HALNEURON (tetrodotoxin), WST-057, ATX01, ART26.12, ART-123, and others. Discover which therapies are expected to grab the major cancer pain market share @ Cancer Pain Market Report Cancer Pain Overview Cancer pain is a common and often debilitating symptom experienced by individuals with cancer, resulting from a variety of causes such as tumor invasion into surrounding tissues, inflammation, nerve compression, or the side effects of treatments like chemotherapy, radiation, and surgery. The nature of cancer pain can vary significantly, it may be acute or chronic, localized or widespread, and described as dull, sharp, burning, or throbbing. Pain not only affects a patient's physical functioning but also takes a toll on emotional well-being, often leading to anxiety, depression, and sleep disturbances. Effective pain management is crucial to maintaining quality of life and supporting overall treatment goals. Cancer Pain Epidemiology Segmentation The cancer pain epidemiology section provides insights into the historical and current cancer pain patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The cancer pain market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Cases of Cancer Pain Etiology-specific Cases of Cancer Pain Incident Cases of Chemotherapy-Induced Peripheral Neuropathy Severity-specific Cases of Cancer Pain Pathophysiology-specific Cases of Cancer Pain Temporal Variation-specific Cases of Cancer Pain Incident Cases of Breakthrough Cancer Pain (BTcP) Cancer Pain Treatment Market Managing chronic cancer-related pain should be personalized, balancing the benefits and drawbacks within the broader context of the patient's overall care goals. When planning pain management, it's important to consider whether disease-modifying treatments are feasible, appropriate, and how they might impact pain. If the pain is localized and caused by tumor pressure or destruction, such as bone lesions, radiotherapy can be very effective. Regardless of whether primary treatments are possible, many patients with active disease-related pain will still require symptom-focused therapies. A thorough pain evaluation is essential to develop a comprehensive, multimodal treatment plan. The first step is to set realistic expectations for the patient, since the cause of the pain affects the likely improvement in pain levels and daily functioning. Patients can be offered various treatment options, including over-the-counter pain relievers, non-opioid prescriptions, procedural interventions, complementary therapies, and systemic opioids. For moderate to severe cancer pain, opioids remain the cornerstone of treatment. Long-term opioid use aims to achieve consistent, meaningful pain relief with manageable side effects, ultimately improving quality of life. Pure μ-opioid receptor agonists are generally preferred for cancer pain, except for drugs like pethidine and dextropropoxyphene, which are discouraged due to their risk of adverse effects. While partial agonists like buprenorphine and mixed-mechanism drugs such as tramadol and tapentadol are options, pure μ-agonists are more commonly used and allow for more flexible dosing. Patients have access to various non-opioid treatment options, which include both medication-based and non-medication approaches. Acetaminophen is often used as a first-choice therapy for patients experiencing mild cancer pain who either do not need opioids or prefer to avoid them. It can also be combined with opioids, and some prescribed medications include both acetaminophen and an opioid in a single tablet for convenience. However, its use in cancer patients is limited due to the risk of liver toxicity, especially in those with liver conditions, and requires careful monitoring for fever in patients with neutropenia. To know more about cancer pain treatment guidelines, visit @ Cancer Pain Management Cancer Pain Pipeline Therapies and Key Companies IRX211: InhaleRX HALNEURON (tetrodotoxin): Dogwood Therapeutics WST-057: WinSanTor ATX01: AlgoTherapeutix ART26.12: Artelo Biosciences ART-123: Asahi Kasei Pharma Discover more about cancer pain drugs in development @ Cancer Pain Clinical Trials Cancer Pain Market Dynamics The cancer pain market dynamics are expected to change in the coming years. The increasing global prevalence of cancer and rising awareness of cancer-associated pain are expected to drive growth in the cancer pain therapeutics market. With no recent drug approvals in this space, pharmaceutical companies have a significant opportunity to penetrate the market, particularly by developing nonopioid-based treatments. As opioids continue to dominate cancer pain management, the anticipated launch of drugs like tetrodotoxin signals a major shift toward novel, nonopioid mechanisms, highlighting the vast potential for innovation in this therapeutic area. Furthermore, potential therapies are being investigated for the treatment of cancer pain, and it is safe to predict that the treatment space will significantly impact the cancer pain market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the cancer pain market in the 7MM. However, several factors may impede the growth of the cancer pain market. Poor assessment of pain, inadequate clinician knowledge, lack of coverage and uneven healthcare reimbursement policies, and the limited effectiveness of standard pain medications such as opioids in cases of refractory pain have all been identified as major barriers to effective cancer pain management, underscoring the need to improve understanding of pain and leverage that knowledge to develop new and more effective treatments. Moreover, cancer pain treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the cancer pain market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the cancer pain market growth. Cancer Pain Market Report Metrics Details Study Period 2020–2034 Coverage 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Cancer Pain Companies InhaleRX, Dogwood Therapeutics, WinSanTor, AlgoTherapeutix, Artelo Biosciences, Asahi Kasei Pharma, and others Key Pipeline Cancer Pain Therapies IRX211, HALNEURON (tetrodotoxin), WST-057, ATX01, ART26.12, ART-123, and others Scope of the Cancer Pain Market Report Therapeutic Assessment: Cancer Pain current marketed and emerging therapies Cancer Pain Market Dynamics: Key Market Forecast Assumptions of Emerging Cancer Pain Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Cancer Pain Market Access and Reimbursement Download the report to understand which factors are driving cancer pain market trends @ Cancer Pain Market Trends Table of Contents 1. Cancer Pain Market Key Insights 2. Cancer Pain Market Report Introduction 3. Cancer Pain Market Overview at a Glance 4. Cancer Pain Market Executive Summary 5. Disease Background and Overview 6. Cancer Pain Treatment and Management 7. Cancer Pain Epidemiology and Patient Population 8. Patient Journey 9. Cancer Pain Marketed Drugs 10. Cancer Pain Emerging Drugs 11. Seven Major Cancer Pain Market Analysis 12. Cancer Pain Market Outlook 13. Potential of Current and Emerging Therapies 14. KOL Views 15. Unmet Needs 16. SWOT Analysis 17. Appendix 18. DelveInsight Capabilities 19. Disclaimer 20. About DelveInsight Related Reports Cancer Pain Epidemiology Forecast Cancer Pain Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology as well as the cancer pain epidemiology trends. Advanced Cancer Pain Management Pipeline Advanced Cancer Pain Management Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key advanced cancer pain management companies, including Tetra Bio-Pharma, ZYUS Life Sciences, among others. Advanced Cancer Pain Management Market Advanced Cancer Pain Management Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key advanced cancer pain management companies, including Tetra Bio-Pharma, ZYUS Life Sciences, among others. Cancer Supportive Care Drugs Market Cancer Supportive Care Drugs Market Insights, Competitive Landscape, and Market Forecast – 2032 report deliver an in-depth understanding of the market trends, market drivers, market barriers, and key cancer supportive care drugs companies, including Helsinn Healthcare SA, Merck & Co., Inc., Sanofi, Pfizer Inc., GSK plc, F. Hoffmann-La Roche Ltd., Amgen Inc., Novartis AG, Viatris Inc., Nimbus Therapeutics, Gilead Sciences, Inc., Eli Lilly and Company, Moderna, Inc., AbbVie Inc., Amgen Inc., Regeneron Pharmaceuticals Inc., Takeda Pharmaceutical Company Limited, Otsuka Holdings Co., Ltd., Affimed GmbH, Bristol-Myers Squibb Company, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact Us Shruti Thakur info@ + Logo: View original content: SOURCE DelveInsight Business Research, LLP Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data

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Blueprint Medicines Announces Data Reinforcing Sustained Clinical Efficacy and Well-Tolerated Safety Profile of Long-Term AYVAKIT®/AYVAKYT® (avapritinib) Treatment at 2025 EHA and EAACI Congresses

-- Breadth of presentations, including one oral and two flash talks, showcase Blueprint Medicines' leadership role in advancing care for patients with systemic mastocytosis -- CAMBRIDGE, Mass., June 12, 2025 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today announced data presentations reflecting over a decade of collaboration with clinical experts and patient advocates to transform the treatment of systemic mastocytosis (SM). Key results continue to position AYVAKIT®/AYVAKYT® (avapritinib) as the durable standard of care across indolent and advanced SM, and highlight the real-world burden of the disease, reinforcing the importance of treating with a therapy that addresses the root cause of SM. These data will be reported at the 2025 European Hematology Association (EHA2025) Hybrid Congress, being held June 12 to 15 in Milan, Italy, and the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, being held June 13 to 16 in Glasgow, United Kingdom. "Our presentations feature large patient populations from the PIONEER, PATHFINDER and EXPLORER trials, with follow-up reaching up to five years in ISM and up to 6.5 years in advanced SM, reflecting both the favorable long-term benefits of AYVAKIT and the unprecedented datasets we have amassed over time," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "AYVAKIT has shown transformative clinical outcomes for patients across the spectrum of SM, including sustained disease control in ISM and prolonged survival in advanced SM. These compelling results have translated into real-world practice, with clinicians expanding their view of who is an appropriate candidate for disease-modifying therapy after positive AYVAKIT experiences, and treatment durations trending toward multiple years." PIONEER Three-Year Data: Durable Clinical Benefits and Consistent Safety Profile with Long-Term AYVAKIT Use in ISM As previously presented,1 AYVAKIT demonstrated robust improvements through 144 weeks in overall symptom and symptom domain measures (skin, gastrointestinal, neurocognitive) representative of real-world patient impacts. AYVAKIT showed a well-tolerated safety profile and a low discontinuation rate due to treatment-related adverse events (TRAEs; 3 percent) with a median of three years of exposure, and some patients out to five years on therapy. Common TRAEs included low-grade edemas, headache and nausea. In newly reported data, AYVAKIT showed sustained clinical benefits across quality-of-life measures that reflect general health status and are broadly recognized by allergists/immunologists, validating previously presented results from the disease-specific, Mastocytosis Quality of Life (MC-QoL) questionnaire. This data presentation follows the May 2025 online publication of PIONEER two-year efficacy and safety data in The Journal of Allergy and Clinical Immunology: In Practice. PATHFINDER/EXPLORER Multi-Year Data: Long-Term Survival Benefits of AYVAKIT in Advanced SM AYVAKIT showed prolonged overall survival (OS) in PATHFINDER and EXPLORER, when indirectly compared to real-world data for midostaurin from the German Registry on Disorders of Eosinophils and Mast Cells (GREM). AYVAKIT led to meaningful survival benefits in patients across all prognostic categories (low, intermediate and high risk), per the Revised Mutation-Adjusted Risk Score (MARS-R) – a new OS risk assessment tool for advanced SM. Conducted in collaboration with University Hospital Mannheim, the analyses validate the MARS-R tool's ability to assess OS risks in advanced SM patients treated with AYVAKIT or midostaurin, using clinical and genetic parameters. The MARS-R was developed to inform physician care decisions based on individual patient needs. PRISM Data: Substantial Disease Burden Across Broad Population of Patients with ISM PRISM is one of the largest studies characterizing the impact of SM from both patient and clinical perspectives. Across the spectrum of disease severity, patients with ISM experienced physical, social and emotional challenges that caused meaningful disruption to their daily lives. Patients reported a broad constellation of disease-related impacts, including limitations to physical activities, work/college and relationships; problems with pain/discomfort and anxiety/depression; and adjustments in their daily lives to avoid certain foods, extended sun exposure and smells. Data Presentations EHA2025 Congress Oral Presentation: The Revised Mutation-Adjusted Risk Score (MARS-R) for Predicting Overall Survival in Patients with Advanced Systemic Mastocytosis Treated with Midostaurin or Avapritinib (Abstract S216) Poster Presentation: Blood-Based Proteomics for Deeper Insights Into Indolent Systemic Mastocytosis: The PIONEER Trial Experience (Abstract PS1838) Poster Presentation: High Accuracy of Peripheral Blood Testing Using Machine Learning–Derived Predictive Models to Distinguish Advanced from Indolent Systemic Mastocytosis: Analysis of Avapritinib and Elenestinib Trial Data (Abstract PF1310) Publication-Only Abstract: Phase 2/3 HARBOR Study of Elenestinib in ISM: A Trial-in-Progress Update of Novel Endpoints and Biomarkers Aimed at Evaluating Disease Modification (Abstract PB3108) EAACI Congress 2025 Flash Talk Presentation: Favorable Benefit-Risk Profile of Avapritinib in Indolent Systemic Mastocytosis Is Maintained After 3 Years of Therapy: Longer-Term Analysis of the PIONEER Study (Abstract 000621) Flash Talk Presentation: The Socio-Emotional Impact of Indolent Systemic Mastocytosis: Insights from the PRISM Survey (Abstract 000488) Poster Presentation: The Phase 2/3 Study of Elenestinib, a Highly Potent and Selective Tyrosine Kinase Inhibitor, in Patients with Indolent Systemic Mastocytosis (Abstract 001121) Data presentations are being made available in the "Science―Publications and Presentations" section of the company's website at 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, pruritus, 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. Until 2023, there were 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 aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL). In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor survival. About AYVAKIT AYVAKIT (avapritinib) is the first and only medicine approved by the U.S. Food and Drug Administration (FDA) to treat the root cause of SM. It was FDA approved for the treatment of advanced SM in June 2021 and ISM in May 2023. It now is indicated in adults with ISM, adults with advanced SM, including ASM, SM-AHN and MCL, and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. The medicine is approved in the EU as AYVAKYT for the treatment of adults with ISM with moderate to severe symptoms inadequately controlled on symptomatic treatment, 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. The therapy is not recommended for the treatment of patients with low platelet counts (less than 50,000/µL). Globally, the medicine is approved for one or more indications in 16 countries, including China where it is marketed by CStone Pharmaceuticals, paying tiered percentage royalties on sales. 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. 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. If contraception requires estrogen, limit ethinyl estradiol to ≤20 mcg unless a higher dose is necessary. To report suspected adverse reactions, contact Blueprint Medicines Corporation at 1-888-258-7768 or the FDA at 1-800-FDA-1088 or AYVAKIT is available in 25-mg, 50-mg, 100-mg and 200-mg tablets. Please click here to see the full U.S. Prescribing Information for AYVAKIT. About Blueprint Medicines Blueprint Medicines is a global, fully integrated biopharmaceutical company that invents life-changing medicines. We seek to alleviate human suffering by solving important medical problems in two core focus areas: allergy/inflammation and oncology/hematology. Our approach begins by targeting the root causes of disease, using deep scientific knowledge in our core focus areas and drug discovery expertise across multiple therapeutic modalities. We have a track record of success with two approved medicines, including AYVAKIT/AYVAKYT (avapritinib) which we are bringing to patients with SM in the U.S. and Europe. Leveraging our established research, development, and commercial capability and infrastructure, we aim to significantly scale our impact by advancing a broad pipeline of programs ranging from early science to advanced clinical trials in mast cell diseases and solid tumors. For more information, visit and follow us on X (formerly 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' leadership role and its ability to transform treatment across the spectrum of SM, including sustained disease control in ISM and prolonged survival in advanced SM; AYVAKIT/AYVAKYT's position as the durable standard of care and clinicians' view of appropriate candidates and treatment duration; plans and expectations for Blueprint Medicines' current or future approved drugs and drug candidates; the potential benefits of any of Blueprint Medicines' current or future approved drugs or drug candidates in treating patients; and Blueprint Medicines' strategy, goals, 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 Blueprint Medicines' ability and plans in continuing to build out and expand a 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 Blueprint Medicines' 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; the timing of the initiation of clinical trials and trial cohorts at clinical trial sites and patient enrollment rates; 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 AYVAKIT/AYVAKYT or any drug candidates it is developing; Blueprint Medicines' ability to successfully expand its operations, research platform and portfolio of therapeutic candidates, and the timing and costs thereof; the success of Blueprint Medicines' current and future collaborations, financing arrangements, partnerships or licensing arrangements; and the ability of the parties to consummate the proposed merger between Blueprint Medicines and Sanofi on the timeline anticipated or at all, including the occurrence of any event, change or other circumstance that could give rise to the termination of the merger. 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 Blueprint Medicines' most recent Annual Report on Form 10-K, as supplemented by 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 explicitly disclaims any obligation to update any forward-looking statements. Footnote 1 Reported at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI) / World Allergy Organization (WAO) Joint Congress Trademarks Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are trademarks of Blueprint Medicines Corporation. 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