AYVAKYT (avapritinib)

Key points

Opinion in favour of reimbursement for the following indication:

AYVAKYT is indicated as 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 treatment.

Therapeutic improvement?

Therapeutic improvement in the care pathway.

Role in therapeutic strategy?

Patients with advanced systemic mastocytosis usually require antitumour therapy to reduce mastocytic infiltration and improve disease-related organ dysfunction, particularly of the bone marrow.

Currently recommended treatments for advanced systemic mastocytosis include midostaurin (approved in the US and EU for all advanced systemic mastocytosis subtypes), cladribine, interferon-alfa (both used off-label) and imatinib (approved in the US only for a subset of patients with aggressive systemic mastocytosis). Despite the marketing authorisation of midostaurin for the treatment of advanced systemic mastocytosis in 2017, cladribine is still recommended for patients requiring rapid debulking, or for patients who need to stop midostaurin due to toxicity, and interferon is recommended for patients with slow disease progression without the need for rapid cytoreduction. In addition to basic treatment, supportive care including symptomatic treatments (antihistamines, sodium cromoglycate, antacids, glucocorticoids, epinephrine, antileukotriene) is given on a long-term basis. The aim is to limit the symptoms of the disease due to the release of substances contained in the mast cells.

Role of AYVAKYT (avapritinib) in the therapeutic strategy:

AYVAKYT (avapritinib) is a second- and further-line treatment for advanced systemic mastocytosis (aggressive systemic mastocytosis, systemic mastocytosis associated with haematological neoplasm or mast cell leukaemia).