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Blueprint Medicines Gets CRL for Ayvakit in Fourth-Line Gist

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Blueprint Medicines Corporation (BPMC - Free Report) announced that the FDA has issued a complete response letter (CRL) to its new drug application (NDA) seeking approval for Ayvakit (avapritinib), an inhibitor of KIT and PDGFRA proteins, to treat adult patients with unresectable or metastatic fourth-line gastrointestinal stromal tumor (GIST). The CRL confirmed that the FDA cannot pass the above-mentioned application.

Notably, Ayvakit is already marketed for treating unresectable or metastatic GIST, harboring a PDGFRA exon 18 mutation in adult patients.

Shares of Blueprint Medicines have lost 19.2% so far this year versus the industry’s increase of 7.8%.


In April 2020, Blueprint Medicines announced that the phase III VOYAGER study evaluating the safety and efficacy of Ayvakit compared to Bayer’s (BAYRY - Free Report) Stivarga (regorafenib) in patients with third- and fourth-line GIST did not meet its primary endpoint of improvement in progression-free survival (PFS) for Ayvakit versus Stivarga. Based on the top-line data from the VOYAGER study, the company previously announced plans to discontinue the further development of Ayvakit for GIST indications other than PDGFRA exon 18 mutant GIST.

We remind investors that in January 2020, Blueprint Medicines’ lead product Ayvakit (avapritinib) was approved by the FDA for treating unresectable or metastatic GIST, harboring a PDGFRA exon 18 mutation including PDGFRA D842V mutations in adults.

Following this nod, Ayvakit became the first precision therapy to have won the FDA nod for treating genomically-defined patients with GIST, a rare form of cancer.

In the first quarter of 2020, Ayvakit generated sales worth $3.5 million, which marks its initial partial quarter since its launch in January. The drug is also under review in Europe with a decision pending in the third quarter 2020. A potential approval should boost Ayvakit’s sales in the quarters ahead.

Notably, the medicine is also being studied for the advanced, indolent and smoldering forms of systemic mastocytosis (SM).

Ayvakit is currently being evaluated in the two ongoing studies, namely the phase I EXPLORER clinical study and the registration-enabling phase II PATHFINDER program for advanced SM. The company plans to submit a supplemental NDA for Ayvakit in the second half of 2020 to address the advanced SM indication.

Zacks Rank & Other Stocks to Consider

Blueprint Medicines currently carries a Zacks Rank #2 (Buy). Other top-ranked stocks in the healthcare sector include Monopar Therapeutics Inc. (MNPR - Free Report) and Abeona Therapeutics Inc (ABEO - Free Report) , both flaunting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Monopar Therapeutics’ loss per share estimates have narrowed 51.6% for 2020 and 6.3% for 2021 over the past 60 days.

Abeona Therapeutics’ loss per share estimates have narrowed 4.5% for 2020 and 24.5% for 2021 over the past 60 days.

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