Current Edition

Broadening horizons: positive CHMP opinion for expanding Ayvakyt

Ayvakyt receives a positive opinion from the CHMP for the treatment of adults with advanced systemic mastocytosis

The Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the expansion ofAyvakytto include monotherapy for the treatment of adult patients with advanced systemic mastocytosis (ASM)–systemic mastocytosis (SM) with an associated haematological neoplasm or mast cell leukaemia.

ASM is a rare and debilitating disease, characterised by damage across multiple organ systems, reduced overall survival and poor quality of life.

Patients with ASM often face issues with being diagnosed and many patients cycle between healthcare professionals without reaching a definitive diagnosis for two to over ten years. Systemic mastocytosis (SM) is driven by the KIT D816V mutation, which the treatment selectively targets.

“The positive opinion from the CHMP is a result of nearly a decade of collaboration with the systemic mastocytosis community and a dedication to bringing 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 accessingAyvakyt, 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,” she reflected.

The CHMP opinion will be reviewed by the European Commission and a final decision on the application is expected by April 2022.

“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 Professor Dr Andreas Reiter, Centre of Excellence for Myeloproliferative Neoplasms, University Medicine Mannheim.

He added: “A precision therapy associated with potent and selective targeting of the KIT D816V mutation brings the prospect of changing the course of the disease and transforming treatment for patients to set a new standard of care in Europe.”