FDA Greenlights Blueprint Medicines’ Ayvakit for Rare Disease Treatment

Blueprint Medicines had a great start to 2020, bagging its very first FDA approval. Its kinase inhibitor, Ayvakit (avapritinib), won the agency’s nod for treating adults with an unresectable or metastatic gastrointestinal stromal tumor harboring a PDGFRA Exon 18 mutation. Since then, the Cambridge, MA-based company has had a great run with the drug regulator, bagging two more authorizations. Last week, it notched its fourth approval, a second for Ayvakit.

On June 16th, the FDA greenlit Ayvakit for treating adults with advanced systemic mastocytosis (Advanced SM), including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL).

“Today’s approval of Ayvakit for advanced systemic mastocytosis – the fourth FDA approval across our portfolio in 18 months – culminates nearly a decade of hard work, from our scientists in the laboratory and clinical team conducting trials, to our commercial organization who will now bring Ayvakit to patients,” said Jeff Albers, CEO of Blueprint Medicines.

Advanced SM – A Rare Disease

Advanced SM is a rare disease driven by the KIT D816V mutation. Most victims are affected by the non-advanced form of SM, which negatively impacts their quality of life. However, patients suffering from advanced SM experience organ damage due to mast cell infiltration and poor survival. Before Ayvakit, the only therapeutic options available were those that offered symptomatic relief.

“Advanced systemic mastocytosis is a debilitating disease characterized by extensive damage in multiple organ systems due to mast cell infiltration, and new treatment options are urgently needed to address these life-threatening complications,” said Dr. Daniel DeAngelo, from the Dana-Farber Cancer Institute.

Ayvakit is the first FDA-approved therapy for this condition. The full approval is based on results from the Phase 1 EXPLORER trial and Phase 2 PATHFINDER trial. Results showed that the drug demonstrated durable clinical efficacy in advanced SM patients across disease subtypes irrespective of their prior treatment status.

“Avapritinib will clearly establish a new standard of care for patients with advanced systemic mastocytosis. The FDA approval was based on data showing robust and durable responses, including complete remissions and a favorable safety profile. For advanced SM patients, the approval of avapritinib shifts the treatment paradigm toward precision therapy that targets the primary driver of mastocytosis.” Dr. DeAngelo added.

Earlier this year, Blueprint announced that Ayvakit raked in $6 million in net product revenue during the fourth quarter of 2020. In March, the drug had received validation from the EMA for the type II variation marketing authorization application for advanced SM. With no competitor drugs for this indication and a likely EMA approval looming, Blueprint would be anticipating a profitable year in global Ayvakit sales.

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Rajaneesh K. Gopinath