Positive CHMP opinion for treatment of rare unpredictable disease

pharmafile | January 31, 2022 | News story | Sales and Marketing  

Blueprint Medicines has announced that the CHMP has adopted a positive opinion recommending to expand the current indication for AYVAKYT® (avapritinib) for the treatment of adults with aggressive systemic mastocytosis (ASM). ASM is a debilitating disease characterised by damage across multiple organ systems, reduced overall survival, and a poor quality of life.

The CHMP has issued a positive opinion recommending the marketing authorisation of AYVAKYT as a 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.

SM is a rare disease driven by the KIT D816V. AYVAKYT selectively targets this mutation. A final decision on the application to expand the indication for AYVAKYT is anticipated by early April 2022.

“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, 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.”

Uncontrolled proliferation and activation of mast cells result in chronic, severe, and often unpredictable symptoms for patients across the spectrum of SM. Mast cells are a type of white blood cell, found in connective tissues throughout the body, particularly under the skin, near blood vessels and lymph vessels, in nerves, and in the lungs and intestines.

“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, MD, Centre 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.”

Ana Ovey

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