RARE Daily

FDA Grants Blueprint Medicines Priority Review for Ayvakit in Expanded Indication

January 23, 2023

The U.S. Food and Drug Administration granted priority review to Blueprint Medicines’ Ayvakit for the treatment of adults with indolent systemic mastocytosis, a rare form of cancer.

Photo: Becker Hewes, chief medical officer at Blueprint Medicines

The agency is expected to act on the company’s application by May 22, 2023.

Systemic mastocytosis (SM) is a rare disease driven by the KIT D816V mutation in about 95 percent of cases. Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms across multiple organ systems. Most of those affected have non-advanced (indolent or smoldering) SM, and among these patients, the vast majority have indolent SM. A broad range of symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, frequently persist in patients with non-advanced SM despite treatment with multiple symptom-directed therapies. Patients often live in fear of severe, unexpected symptoms, have limited ability to work or perform daily activities, and isolate themselves to protect against unpredictable triggers. Currently, there are no approved therapies for the treatment of non-advanced SM.

A minority of patients have advanced SM, which encompasses a group of high-risk SM subtypes including ASM, SM-AHN and MCL. In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor survival.

Ayvakit is a kinase inhibitor approved by the FDA for the treatment of adults with advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL), and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Ayvakit is approved in Europe for the treatment of adults with ASM, SM-AHN or MCL, after at least one systemic therapy, and adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.

The FDA previously granted breakthrough therapy designation to Ayvakit for the treatment of moderate to severe indolent SM. AYVAKIT was designed to potently and selectively inhibit D816V mutant KIT, the primary underlying cause of SM.

“People with indolent systemic mastocytosis experience debilitating symptoms and poor quality of life, and we have the potential to transform clinical outcomes for these patients by targeting the genetic driver of disease with Ayvakit,” said Becker Hewes, chief medical officer at Blueprint Medicines. ” Ayvakit achieved the primary and all key secondary endpoints in the PIONEER trial, with highly meaningful reductions in patient-reported symptoms and all measures of mast cell burden studied, and a well-tolerated safety profile supporting chronic treatment.”

Author: Rare Daily Staff

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