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First drug approved for the treatment of indolent systemic mastocytosis

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Patients with indolent systemic mastocytosis can breathe a sigh of relief. Thanks to a recent clinical trial, a drug for the treatment of the disease is now available for the first time to patients in the USA and soon also in Germany.

Systemic mastocytosis is a rare disease caused in almost all cases by the KIT-D816V mutation. It is characterized by uncontrolled proliferation and activation of mast cells, which in turn lead to chronic, severe and often unpredictable symptoms. The majority of patients suffer from indolent systemic mastocytosis in which, despite symptom-oriented therapies, there is no improvement in symptoms. Typical symptoms include anaphylaxis, maculopapular rash, pruritus, diarrhea, drowsiness, fatigue, and bone pain. Thus, the quality of life is severely affected.

Since the end of May, there is now for the first time in the USA and soon also in Germany an approved drug that treats the primary trigger of the disease and provides comprehensive and lasting symptom relief. The approval is based on the positive results of a recent multicenter study in which the active ingredient avapritinib achieved significant improvements in disease symptoms and mast cell burden when administered once daily. More than 200 pateints participated in the nearly one-year clinical trial. "The PIONEER study showed that the drug significantly reduced serum tryptase levels, a critical marker of the extent of mast cell proliferation. Already after half of the study duration, the good tolerability and efficacy could be seen," says, PD Dr. med. Frank Siebenhaar, study leader and physician at the Institute of Allergology, Charité - Universitätsmedizin Berlin and adds "with this new treatment it will be possible in the future to improve the quality of life of severely affected patients."



Press release


Prof. Dr. Marcus Maurer
Director of the Institute of Allergology
Charité – Universitätsmedizin Berlin
tel: +49 30 450 518043 
email: marcus.maurer(at)

PD Dr. Frank Siebenhaar
Head of University outpatient clinic for Allergology
Charité – Universitätsmedizin Berlin
tel: +49 30 450 518318
email: frank.siebenhaar(at)

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