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Biopharmaceuticals Oncology Regulatory

 News Release - January 28, 2022

Blueprint Medicines' AYVAKYT(R) (avapritinib) Receives Positive CHMP Opinion for the Treatment of Adults with Advanced Systemic Mastocytosis

Positive opinion based on results from two clinical trials that comprise the largest clinical trial dataset in advanced systemic mastocytosis

AYVAKYT selectively targets KIT D816V mutation, the primary driver of systemic mastocytosis

Advanced systemic mastocytosis is a debilitating disease characterized by damage across multiple organ systems, reduced overall survival and poor quality of life

CAMBRIDGE, Mass., Jan. 28, 2022 -- (Healthcare Sales & Marketing Network) -- Blueprint Medicines Corporation (NASDAQ: BPMC) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion, recommending to expand the current indication for AYVAKYT® (avapritinib) to include monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN) or mast cell leukemia (MCL), after at least one systemic therapy.

"The positive opinion from the CHMP is a result of nearly a decade of collaboration with the systemic mastocytosis community and a dedication to bring a transformative therapy to treat and manage this rare, life-threatening disease," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "Today's announcement brings patients with advanced forms of SM in Europe one step closer to accessing AYVAKYT, the first specifically designed precision therapy to selectively target the primary driver of their disease. Blueprint Medicines remains committed to improving outcomes for patients living with systemic mastocytosis around the world."

"Patients across Europe are waiting for innovative treatment options for advanced systemic mastocytosis, which is associated with organ damage due to mast cell proliferation and poor survival outcomes," said Prof. Dr. Andreas Reiter, M.D., Center of Excellence for Myeloproliferative Neoplasms, University Medicine Mannheim. "A precision therapy associated with potent and selective targeting of the KIT D816V mutation brings the prospect of changing the course of disease and transforming treatment for patients to set a new standard of care in Europe."

The CHMP opinion will now be reviewed by the European Commission, which has the authority to grant and expand marketing authorizations for medicinal products in the European Union. A final decision on the application to expand the indication for AYVAKYT is anticipated by early April 2022.

The CHMP based its opinion on results from the Phase 1 EXPLORER trial and Phase 2 PATHFINDER trial, in which AYVAKYT showed durable clinical efficacy in advanced SM patients across disease subtypes and a generally well-tolerated safety profile. These two studies, which enrolled approximately 150 patients with up to four years of follow-up and comprise the largest ever reported dataset in advanced systemic mastocytosis, were recently published in Nature Medicine.

About AYVAKYT (avapritinib)

AYVAKYT (avapritinib) is a kinase inhibitor approved by the European Commission for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring the PDGFRA D842V mutation. Under the brand name AYVAKIT, the medicine is approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and for the treatment of adults with Advanced SM, including aggressive SM (ASM), SM-AHN and mast cell leukemia (MCL).1 It is also approved under the brand name AYVAKIT in Mainland China for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and in Hong Kong and Taiwan for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA D842V mutation.2-4

AYVAKYT/AYVAKIT is not approved for the treatment of any other indication in the Europe, U.S., or Greater China, or for any indication in any other jurisdiction by any other health authority.

Blueprint Medicines is developing AYVAKYT/AYVAKIT globally for the treatment of advanced and non-advanced SM. The European Commission granted orphan medicinal product designation for AYVAKYT for the treatment of GIST and mastocytosis. The U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to AYVAKIT for the treatment of moderate to severe indolent SM.

To learn about ongoing or planned clinical trials, contact Blueprint Medicines at and +31 85 064 4001. Additional information is available at and

Please see the Summary of Product Characteristics for AYVAKYT.

About Systemic Mastocytosis

Systemic mastocytosis (SM) is a rare disease driven by the KIT D816V mutation. Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms for patients across the spectrum of SM. The vast majority of those affected have non-advanced (indolent or smoldering) SM, with debilitating symptoms that lead to a profound, negative impact on quality of life. 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. In Europe, there are about 40,000 patients with SM, and advanced SM represents about 5 to 10 percent of this patient population.5

Debilitating symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, often persist across all forms of SM despite treatment with a number of symptomatic therapies. 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 SM that selectively inhibit D816V mutant KIT.6,7

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 approved medicines directly to patients in the United States and Europe, and we are globally advancing multiple programs for systemic mastocytosis, lung 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 approval of AYVAKYT and the implications of such approval for patients, caregivers and healthcare professionals; expectations regarding the potential benefits of AYVAKYT in treating patients with advanced SM; expectations concerning when AYVAKYT will be commercially available in Europe; 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 the impact of the COVID-19 pandemic to Blueprint Medicines' business, operations, strategy, goals and anticipated milestones, including Blueprint Medicines' ongoing and planned research and discovery activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Blueprint Medicines' ability and plans in establishing a commercial infrastructure, and successfully launching, marketing and selling current or future approved products, including AYVAKIT and GAVRETO® (pralsetinib); Blueprint Medicines' ability to successfully expand the approved indications for AYVAKIT and GAVRETO or obtain marketing approval for AYVAKIT and GAVRETO 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; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; Blueprint Medicines' ability to develop and commercialize companion diagnostic tests for its current and future drug candidates; and the success of Blueprint Medicines' current and future acquisitions, 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 Blueprint Medicines' most recent Annual Report on Form 10-K, as supplemented by its most recent Quarterly Report on Form 10-Q 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 explicitly disclaims any obligation to update any forward-looking statements.


1 Blueprint Medicines. AYVAKIT (avapritinib) Prescribing Information. Accessed at January 18, 2022.

2 CStone Announces China NMPA New Drug Approval of Precision Therapy AYVAKIT® (avapritinib) for the Treatment of Adults with Unresectable or Metastatic PDGFRA Exon 18 Mutant Gastrointestinal Stromal Tumor. CStone Pharmaceuticals. Accessed at January 26, 2022.

3 "CStone announced new drug approval of precision therapy AYVAKIT® (avapritinib) in Hong Kong, China for the treatment of PDGFRA D842V mutant gastrointestinal stromal tumors (GIST)." CStone Pharmaceuticals. Accessed at January 26, 2022.

4 " CStone Announces Acceptance of New Drug Application in Hong Kong for Avapritinib for the Treatment of Adults with Unresectable or Metastatic PDGFRA D842V Mutant Gastrointestinal Stromal Tumor." Accessed at: January 26, 2022.

5 Estimated SM prevalence and patient subtypes based on internal claims analysis and epidemiology reported in Orphanet ( and Cohen SS, Skovbo S, Vestergaard H, et al. Epidemiology of systemic mastocytosis in Denmark. Br J Haemotol. 2014;166(4):521-528.

6 Jennings SV, Slee VM, Zack RM, et al. Patient perceptions in mast cell disorders. Immunol Allergy Clin North Am. 2018;38(3):505-525.

7 Mesa, RA, Sullivan, EM, Dubinski, D, et al. Patient reported outcomes among systemic mastocytosis (SM) patients in routine clinical practice: results from the TouchStone Survey. Blood. 2020;136(1):37.


Blueprint Medicines AYVAKYT, AYVAKIT, GAVRETO and associated logos are trademarks of Blueprint Medicines Corporation.

Source: Blueprint Medicines

Issuer of this News Release is solely responsible for its content.
Please address inquiries directly to the issuing company.

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