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

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Blueprint Medicines Corporation (BPMC - Free Report) announced that the European Commission has approved its marketed drug, Ayvakyt (avapritinib), for a new indication. The drug is now approved as a monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (“SM”), including SM with an associated hematological neoplasm as well as mast cell leukemia, after at least one systemic therapy.

Following the latest nod from the EU, Ayvakyt became the first approved therapy designed to selectively target the KIT D816V mutation, the primary driver of disease in the region. The company plans to launch Ayvakyt in Germany immediately following the approval in Europe.

The EC’s approval for Ayvakyt was expected, as in January 2022, the European Medicines Agency’s Committee for Medicinal Products for Human Use rendered a positive opinion on and recommended the approval for Ayvakyt as a monotherapy for treating adult patients with advanced SM.

Shares of Blueprint Medicines have dipped 39.8% so far this year compared with the industry’s decline of 13.6%.

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Last June, the FDA approved Ayvakit for the treatment of adult patients with advanced SM, a rare, debilitating disease.

The FDA approved Ayvakit 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 drug is marketed under the trade name Ayvakyt in Europe.

The European Commission has also granted 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 is also being evaluated in the phase II PIONEER study for treating non-advanced SM. Data from the same is expected in mid-2022. Pending results from the study, Blueprint Medicines plans to submit a supplemental new drug application for Ayvakit in non-advanced SM to the FDA by the end of 2022.

Ayvakit/Ayvakyt recorded sales of $53 million in 2021, reflecting a significant increase year over year. Label expansion of the drug in additional indications should drive sales in the days ahead.

Zacks Rank & Stocks to Consider

Blueprint Medicines currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector are Athersys, Inc. , Kaleido Biosciences, Inc. and Vertex Pharmaceuticals Incorporated (VRTX - 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.

Athersys’ earnings estimates have been revised 21.7% upward for 2022 over the past 60 days.

Earnings of ATHX surpassed estimates in one of the trailing four quarters and missed the same on the other three occasions.

Kaleido Biosciences’ loss per share estimates have narrowed 23% for 2022 over the past 60 days.

Earnings of KLDO surpassed estimates in three of the trailing four quarters and missed the same on the remaining occasion.

Vertex’s earnings estimates have been revised 8.7% upward for 2022 over the past 60 days. The VRTX stock has increased 15.6% year to date.

Earnings of Vertex surpassed estimates in each of the trailing four quarters.


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