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Blueprint Medicines (BPMC) Gets FDA Nod for Ayvakyt in SM

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Blueprint Medicines Corporation (BPMC - Free Report) announced that the FDA has approved its lead drug, Ayvakit (avapritinib), for a new indication. The drug is now approved for the treatment of adult patients with advanced systemic mastocytosis (“SM”), including aggressive SM, SM with an associated hematological neoplasm as well as mast cell leukemia.

Following the FDA nod, Ayvakit became the precision therapy that specifically targets the primary driver of the disease. Thus, patients with advanced SM can now receive a targeted therapy designed to potently and selectively inhibit D816V mutant KIT.

The FDA approval was based on data from the phase I EXPLORER study and the phase II PATHFINDER study – which evaluated Ayvakit for advanced SM, a rare, debilitating disease. In the study, treatment with Ayvakit led to durable clinical responses, including complete remissions, in patients with or without prior treatment.

Please note that the FDA accepted Blueprint Medicines’ supplemental new drug application (sNDA) seeking approval of Ayvakit for treating advanced SM in February 2021.

Ayvakyt is also under review in Europe for the treatment of advanced SM. The drug is marketed under the trade name Ayvakit in the United States.

Shares of Blueprint Medicines have plunged 23.7% so far this year compared with the industry’s decrease of 0.7%.

Zacks Investment ResearchImage Source: Zacks Investment Research


We remind investors that Ayvakit was approved by the FDA for treating unresectable or metastatic gastrointestinal stromal tumor (“GIST”), harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations in adults, in January 2020.

The European Commission too has granted a conditional marketing authorization to Ayvakyt as a monotherapy for the treatment of adult patients with unresectable or metastatic GIST, harboring the PDGFRA D842V mutation.

Ayvakit/Ayvakyt recorded sales of $7.1 million in the first quarter of 2021, reflecting an increase of 18.3% on a sequential basis. Label expansion of the drug in additional indications should drive sales in the days ahead.

Ayvakit is also being evaluated in the PIONEER study for treating non-advanced SM.

Zacks Rank & Stocks to Consider

Blueprint Medicines currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector are Repligen Corporation (RGEN - Free Report) , Kamada Ltd. (KMDA - Free Report) and Bio-Techne Corporation (TECH - Free Report) , all carrying a Zacks Rank #2 (Buy) at present. You can see  the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Repligen’s earnings estimates have been revised 18.3% upward for 2021 and 14.7% upward for 2022 over the past 60 days.

Kamada’s earnings estimates have been revised 21.4% upward for 2021 and 18.1% upward for 2022 over the past 60 days.

Bio-Techne’s earnings estimates have been revised 8.8% upward for 2021 and 9.1% upward for 2022 over the past 60 days. The stock has rallied 39.4% year to date.

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