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US approval for Blueprint Medicines' gastrointestinal stromal tumour therapy

Published on 10/01/20 at 10:02am

The FDA has moved to authorise Blueprint Medicines’ kinase inhibitor Ayvakit (avapritinib) for the treatment of unresectable or metastatic gastrointestinal stromal tumour (GIST) in patients possessing a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation.

This indication includes the treatment of those with a PDGFRA D842V mutation – the most common type of exon 18 mutation.

In a study of 43 patients with PDGFRA exon 18 mutation who received either 300mg or 400mg Ayvakit once daily, 84% experienced complete or partial tumour shrinkage, breaking down to 7% for the former and 77% for the latter. In 38 patients of this total which had a PDGFRA D842V mutation, 82% reported a partial response and 7% reported a complete response.

Additionally, 61% of the total number of participants reported a duration of response lasting longer than six months, but a median duration of response was not reached throughout the trial.

The drug secured approval under the Breakthrough Therapy pathway, with the US regulator warning against its use in pregnant women due to the harm it can potentially cause to a developing foetus or newborn baby.

“GIST harbouring a PDGFRA exon 18 mutation do not respond to standard therapies for GIST. However, today’s approval provides patients with the first drug specifically approved for GIST harbouring this mutation,” said Dr Richard Pazdur, Director of the FDA’s Oncology Center of Excellence and acting Director of the Office of Oncologic Diseases in the regulator’s Center for Drug Evaluation and Research. “Clinical trials showed a high response rate with almost 85% of patients experiencing tumour shrinkage with this targeted drug.”

Matt Fellows

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