EMA committee recommends expanded indication approval for Blueprint Medicines Ayvakyt to treat advanced systemic mastocytosis

Blueprint Medicines Corporation 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 haematological neoplasm (SM-AHN) or mast cell leukaemia (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. 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 US for the treatment of adults with unresectable or metastatic GIST harbouring 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 leukaemia (MCL). It is also approved under the brand name Ayvakit in Mainland China for the treatment of adults with unresectable or metastatic GIST harbouring 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 harbouring a PDGFRA D842V mutation. Ayvakyt/Ayvakit is not approved for the treatment of any other indication in the Europe, US, 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 US Food and Drug Administration (FDA) granted breakthrough therapy designation to Ayvakit for the treatment of moderate to severe indolent SM. 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 per cent of this patient population. Debilitating symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhoea, 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. Blueprint Medicines is a global precision therapy company that invents life-changing therapies for people with cancer and blood disorders.

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