EC approves Blueprint’s Ayvakyt as first treatment for rare haematological disorder
14 Dec 2023
Drug ApprovalClinical Result
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Source: PMLiVE
The European Commission (EC) has approved Blueprint Medicines’ Ayvakyt (avapritinib) as the first treatment for indolent systemic mastocytosis (ISM).
Systemic mastocytosis (SM) is a rare haematological disorder that can lead to a range of debilitating symptoms across multiple organ systems.
ISM accounts for the vast majority of SM cases and, despite the availability of multiple symptom-directed therapies, a number of ISM patients continue to experience a substantial disease burden.
Blueprint’s Ayvakyt works by targeting KIT D816V, the primary underlying cause of the disease.
“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,” said Jens Panse, deputy director of the department of haematology/oncology at the University Hospital RWTH Aachen in Germany.
The EC’s decision on the drug follows a recent recommendation from the European Medicines Agency’s human medicines committee and was supported by positive results from the PIONEER trial, the largest study ever conducted in ISM.
Results showed that Ayvakyt was associated with significant improvements versus placebo in the primary and all key secondary endpoints, including overall symptoms and measures of mast cell burden.
Ayvakyt was also found to be well-tolerated with a favourable safety profile, and most adverse reactions were reported as mild.
“Based on this practice-changing data, Ayvakyt has the potential to advance treatment for a broad range of patients living with ISM,” Panse said.
Georg Pirmin Meyer, senior vice president, international at Blueprint Medicines, said the approval "represents an important step toward delivering a new global standard of care for patients with ISM”.
"For the first time in Europe, ISM patients have an approved therapy, marking a new era in the treatment of this disease. Ayvakyt is the first approved medicine for both ISM and advanced SM, and our team is committed to bringing this transformative therapy to patients across the spectrum of disease,” he said.
Ayvakit has already been approved by the EC to treat certain patients with aggressive systemic mastocytosis, SM with an associated haematological neoplasm, mast cell leukaemia and unresectable or metastatic gastrointestinal stromal tumours.
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