Blueprint Medicines Submits New Supplementary Drug Application to FDA for AYVAKIT ™ (avapritinib) for the Treatment of Advanced Systemic Mastocytosis


CAMBRIDGE, Mass., December 17, 2020 / PRNewswire / – Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced the submission of a new drug application Supplement (sNDA) to the United States Food and Drug Administration. (FDA) for AYVAKIT ™ (avapritinib) for the treatment of adult patients with advanced systemic mastocytosis (MS). AYVAKIT is a potent and selective inhibitor of the D816V KIT mutant, the primary driver of MS, and is under development to treat advanced and non-advanced forms of the disease.

Blueprint Medicines has requested a priority review for this application, which, if granted, could result in a six-month review process. The FDA has a 60-day filing review period to determine if the sNDA is complete and acceptable for filing. The FDA has granted AYVAKIT Breakthrough Therapy designation for the treatment of advanced MS, including aggressive MS subtypes, MS with associated hematologic neoplasm and mast cell leukemia.

“Today’s submission is an important step towards our goal of bringing AYVAKIT to patients with advanced systemic mastocytosis, a rare, debilitating and potentially fatal disease,” said Fouad Namouni, MD, president, Research and Development. “Our application is based on an unprecedented clinical data set in this disease, which showed that patients receiving AYVAKIT had high overall response and complete remission rates, with prolonged survival, and the treatment was generally well tolerated. We look forward to working closely with the FDA during the review, as we seek to introduce the first precision therapy targeting the underlying cause of systemic mastocytosis. “

About SM

MS is a rare disease caused by the KIT D816V mutation. The uncontrolled proliferation and activation of mast cells results in chronic, severe and often unpredictable symptoms in patients across the spectrum of MS. The vast majority of those affected have non-advanced (indolent or smoldering) MS, with debilitating symptoms that have a profound negative impact on quality of life. A minority of patients have advanced MS, which encompasses a group of high-risk MS subtypes including aggressive MS, MS with associated hematologic neoplasia, and mast cell leukemia. In addition to symptoms of mast cell activation, advanced MS is associated with organ damage due to mast cell infiltration and poor overall survival.

Debilitating symptoms, including anaphylaxis, maculopapular rashes, pruritus, diarrhea, brain fog, fatigue, and bone pain, often persist in all forms of MS despite treatment with a number of symptomatic therapies. Patients often live in fear of severe and unexpected symptoms, have a limited ability to work or perform daily activities, or self-isolate to protect themselves from unpredictable triggers. Currently, there are no approved therapies for the treatment of MS that selectively inhibit the mutant KIT D816V.

About AYVAKIT (avapritinib)

AYVAKIT (avapritinib) is an FDA-approved kinase inhibitor for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) carrying a mutation in PDGFRA exon 18, including mutations of PDGFRA D842V. For more information visit This medicine is approved in Europe under the brand name AYVAKYT® for the treatment of adults with unresectable or metastatic GIST carrying the PDGFRA D842V mutation.

AYVAKIT / AYVAKYT is not approved for the treatment of any other indication, including SM, in the United States by the FDA or in Europe by the European Commission, or for any indication in any other jurisdiction by any other health authority.

Blueprint Medicines is developing AYVAKIT globally for the treatment of advanced and indolent MS.

Blueprint Medicines has entered into an exclusive collaboration and licensing agreement with CStone Pharmaceuticals for the development and commercialization of AYVAKIT in Mainland China, Hong Kong, Macao and Taiwan. Blueprint Medicines retains the development and commercial rights of AYVAKIT in the rest of the world.

About Blueprint Medicines

Blueprint Medicines is a precision therapy company dedicated to improving human health. With a focus on genomically defined cancers, rare diseases and cancer immunotherapy, we are developing transformational drugs rooted in our cutting edge expertise in protein kinases, which are proven drivers of disease. Our uniquely scalable and targeted approach enables the rapid design and development of new treatments and increases the chances of clinical success. We have two approved precision therapies and are currently advancing several investigational drugs in clinical and preclinical development, as well as a number of early stage research programs. For more information visit and follow us on Twitter (@BlueprintMeds) and LinkedIn.

Caution 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 AYVAKIT’s development plans and timelines for SM advanced and non-advanced; plans and timelines for the commercialization of AYVAKIT for Advanced MS, if approved; the potential benefits of Blueprint Medicines’ current and future approved drugs or drug candidates in the treatment of patients, including expectations regarding the potential of AYVAKIT for the treatment of patients with MS; and the strategy, goals and anticipated milestones, business plans and direction of Blueprint Medicines. The words “aim”, “be able”, “will fly”, “could”, “would”, “should”, “expect”, “plan”, “anticipate”, “intend to”, ” believe “,” estimate “,” plan “,” project “,” potential “,” continue “,” target “and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these words. ‘identification. All forward-looking statements contained in this press release are based on the current expectations and beliefs of management and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to occur. differ materially from those expressed or implied by any forward-looking statement. the statements contained in this press release, including, without limitation, the risks and uncertainties relating to the impact of the COVID-19 pandemic on the activities, operations, strategy, objectives and planned milestones Blueprint Medicines, including ongoing and planned research and discovery of Blueprint Medicines 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 launch, marketing and sale of current or future approved products; the ability and plans of Blueprint Medicines to continue to build and maintain a business infrastructure and to successfully launch, market and sell current or future approved products; Blueprint Medicines’ ability to successfully expand the approved indications for AYVAKIT / AYVAKYT and GAVRETO ™ (pralsetinib) or obtain marketing authorization for AYVAKIT / AYVAKYT and GAVRETO in other geographies in the future; the delay of any ongoing or planned clinical trials or the development of current or future Blueprint Medicines drug candidates; Advancement by Blueprint Medicines of multiple early stage efforts; Blueprint Medicines’ ability to successfully demonstrate the safety and efficacy of its drug candidates and to obtain approval of its drug candidates on a timely basis, if at all; the preclinical and clinical results of Blueprint Medicines drug candidates, which may not support the further development of these drug candidates; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; Blueprint Medicines’ ability to obtain, maintain and enforce patents and other intellectual property protections for AYVAKIT / AYVAKYT, GAVRETO or any drug candidate it develops; Blueprint Medicines’ ability to develop and market companion diagnostic tests for AYVAKIT / AYVAKYT, GAVRETO or any of its current and future drug candidates; and the success of Blueprint Medicines’ current and future collaborations, partnerships or licensing agreements. These and other risks and uncertainties are further described in the section entitled “Risk Factors” in the documents filed by Blueprint Medicines with the Securities and Exchange Commission (SEC), including the most recent annual report of Blueprint Medicines on Form 10-K, as completed by its most recent Quarterly Report on Form 10-Q and any other filings Blueprint Medicines has made or may make with the SEC in the future. All forward-looking statements contained in this press release represent the views of Blueprint Medicines only as of the date hereof and should not be construed as representing its views as of any later date. Except as required by law, Blueprint Medicines expressly disclaims any obligation to update any forward-looking statements.


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

SOURCE Blueprint Medicines Corporation

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