Blueprint Medicines Corporation (BPMC - Free Report) announced that it has received the FDA approval for Ayvakit (avapritinib) as a treatment of unresectable or metastatic gastrointestinal stromal tumor (GIST), a rare form of cancer, harboring a PDGFRA exon 18 mutation including PDGFRA D842V mutations in adults. This marks the first FDA nod to the company for any of its pipeline candidates.
The FDA approval was based on the combined safety results from multiple 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. In the study, Ayvakit demonstrated an overall response rate (ORR) of 84% in the given patient population while the median duration of response (DOR) was not reached.
Following the nod, Ayvakit became the first precision therapy to be approved by the FDA for treating genomically defined patients with GIST, a population that previously had very limited treatment options. Blueprint Medicines plans to launch Ayvakit in the United States in a week’s time.
Shares of Blueprint Medicines have rallied 25.4% in the past year against the industry’s decline of 3.4%.
We remind investors that in August 2019, the FDA accepted the new drug application (NDA) for Ayvakit to treat patients with PDGFRA Exon 18 mutant GIST, regardless of any prior therapy and the fourth-line GIST. Later in October, Blueprint Medicines announced that the FDA intends to administratively split the proposed above-mentioned indications for Ayvakit into two separate NDAs. Back then, the regulatory agency had set an action date of Feb 14, 2020 for both indications.
Therefore, this approval for Ayvakit to treat patients with PDGFRA Exon 18 mutant GIST comes before the scheduled date. However, along with press release, the company did provide an update on the fourth-line GIST indication. The action date for the same remains Feb 14, 2020.
Per the company, regarding the NDA for the fourth-line GIST indication, an extension of up to three months for the action date will likely be required for Blueprint Medicines to report top-line data to the FDA from the phase III VOYAGER study, which is evaluating Ayvakit compared to Bayer’s (BAYRY - Free Report) Stivarga (regorafenib) in patients with third- and fourth-line GIST.
We note that until now, Blueprint Medicines had no approved product in its portfolio. Revenues generated by the company were drawn from its partnerships with Roche (RHHBY - Free Report) and CStone Pharmaceuticals. Hence, the approval of Ayvakit should lower the company’s heavy dependence on its partners for revenues in the future quarters.
Meanwhile, other than GIST, Blueprint Medicines is pursuing a broad clinical development program for Ayvakit across the advanced, indolent and smoldering forms of systemic mastocytosis (SM).
Last month, the company announced encouraging initial data from the dose-finding part 1 of phase II PIONEER study on Ayvakit for treating patients with indolent SM. The company plans to submit an NDA for Ayvakit in the first quarter of 2020 to treat advanced SM indication.
Zacks Rank & Key Pick
Blueprint Medicines currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the biotech sector is Emergent Biosolutions Inc. (EBS - Free Report) , which sports 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 moved 5.6% north for 2020 over the past 60 days.
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