EC approves treatment for rare haematological disorder

The European Commission has approved Blueprint Medicines’ Ayvakyt® (avapritinib) for treatment of adult patients with the haematological disorder indolent systemic mastocytosis (ISM).

“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 Jens Panse, Deputy Director of the Department of Haematology/Oncology of the University Hospital RWTH Aachen in Germany.

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 EMA’s Committee for Medicinal Products for Human Use (CHMP) in November.

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.

The EC approval “represents an important step toward delivering a new global standard of care for patients with ISM and builds on years of collaboration with the SM community,” explained Georg Pirmin Meyer, Senior Vice President, International at Blueprint Medicines.

Blueprint Medicines plans first commercial launch of Ayvakyt in Germany. Additional markets will follow dependent on local healthcare technology assessment and reimbursement process timelines. 

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).