Back to top

Image: Bigstock

Blueprint Medicines' Gist Drug Ayvakyt Gets EU Approval

Read MoreHide Full Article

Blueprint Medicines Corporation (BPMC - Free Report) announced that the European Commission (EC) has granted conditional marketing authorization to its lead drug Ayvakyt (avapritinib) as a monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumors (GIST), harboring the PDGFRA D842V mutation.

The drug is marketed under the trade name Ayvakit in the United States.

Ayvakyt is the first highly effective treatment option to get the EU nod for PDGFRA D842V mutant GIST, a rare form of cancer. In July, the CHMP rendered a positive opinion recommending conditional approval for Ayvakyt to address the given indication.

The approval in Europe was based on the combined safety results from two studies on Ayvakit and the efficacy data from the phase I NAVIGATOR study, which evaluated Ayvakit in adult patients with PDGFRA Exon 18 mutant GIST. While Ayvakyt demonstrated an overall response rate (ORR) of 95% in the given patient population, the median duration of response (DOR) was 22.1 months. The median progression-free survival (PFS) was 24 months while the median overall survival (OS) was not reached.

Moreover, Ayvakyt demonstrated a deep and durable clinical activity and was generally well-tolerated in patients with PDGFRA D842V mutant GIST, regardless of any prior therapy.

Shares of Blueprint Medicines have rallied 9.9% so far this year compared with the industry’s increase of 1.5%.


We note that in January this year, Ayvakit 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.

In the first six months of 2020, Ayvakit generated sales worth $9.1 million. The drug is also being studied for the treatment of patients with advanced systemic mastocytosis (SM), a rare, debilitating disease.

Earlier this month, Blueprint Medicines announced positive top-line data from the phase I EXPLORER study and the phase II PATHFINDER, which evaluated Ayvakit for treating advanced SM. A potential label expansion of the drug for the same indication should drive Ayvakit’s sales prospects.

Zacks Rank & Stocks to Consider

Blueprint Medicines currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the healthcare sector include Emergent BioSolutions Inc. (EBS - Free Report) , Horizon Therapeutics Public Limited Company and QIAGEN N.V. (QGEN - Free Report) , all presently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Emergent’s earnings estimates have been revised 55.5% upward for 2020 and 37.3% for 2021 over the past 60 days. The stock has surged 88.4% year to date.

Horizon Therapeutics’ earnings estimates have moved 46.7% north for 2020 and 49.1% for 2021 over the past 60 days. The stock has skyrocketed 116.2% year to date.

QIAGEN’s earnings estimates have been revised 10.6% upward for 2020 and 22.5% for 2021 over the past 60 days. The stock has soared 53.6% year to date.

More Stock News: This Is Bigger than the iPhone!

It could become the mother of all technological revolutions. Apple sold a mere 1 billion iPhones in 10 years but a new breakthrough is expected to generate more than 27 billion devices in just 3 years, creating a $1.7 trillion market.

Zacks has just released a Special Report that spotlights this fast-emerging phenomenon and 6 tickers for taking advantage of it. If you don't buy now, you may kick yourself in 2021.

Click here for the 6 trades >>


Zacks' 7 Best Strong Buy Stocks (New Research Report)


Valued at $99, click below to receive our just-released report
predicting the 7 stocks that will soar highest in the coming month.


Click Here, It's Really Free

Published in