The
"Ayvakyt represents an important treatment breakthrough as the first medicine approved for patients living with ISM, and the only therapy designed to selectively target the primary genetic driver of the disease," declared
A kinase inhibitor, avapritinib works by potently and selectively target KIT D816V, the main underlying driver of ISM.
EC's approval of the treatment was based on data from a Phase II double-blind, placebo-controlled clinical trial, PIONEER. It also follows a positive recommendation by
In the PIONEER study – the largest ever conducted in ISM - Ayvakyt showed clinically meaningful improvements versus placebo”
In the PIONEER study – the largest ever conducted in ISM - Ayvakyt showed clinically meaningful improvements versus placebo. It was also well-tolerated with a favourable safety profile.
"Based on these practice-changing data, Ayvakyt has the potential to advance treatment for a broad range of patients living with ISM," Panse said.
ISM is the most common form of SM and it affects an estimated 40,000 people in the EU. The disorder has a broad range of symptoms, which often persist despite treatment with symptom-directed therapies. Symptoms include anaphylaxis, maculopapular rash, pruritis, diarrhoea, brain fog, fatigue and bone pain.
Avapritinib is approved in the US under brand name Ayvakit® for the treatment of adults with ISM, advanced systemic mastocytosis and unresectable or metastatic gastrointestinal stromal tumour (GIST).
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