Blueprint Medicines Corporation (BPMC - Free Report) announced that the FDA has extended the review period of a new drug application (“NDA”) for its lead pipeline candidate, avapritinib, by three months. The NDA is seeking accelerated approval for avapritinib, an inhibitor of KIT and PDGFRA proteins, as a treatment for fourth-line gastrointestinal stromal tumors (“GIST”).
As a result, the regulatory body will now announce its decision by May 14, 2020 instead of the previously anticipated date in February 2019. The extension of the review period is due to requested submission of additional data on the candidate by the FDA.
Shares of Blueprint Medicines fell 5.4% on Feb 6, following the announcement. The company’s shares have declined 15.5% in the past year compared with the industry’s decrease of 0.1%.
The company submitted the NDA in June 2019 to the FDA, seeking accelerated approval for avapritinib for treating PDGFRA Exon 18 mutant GIST and fourth-line GIST. The NDA was accepted and granted priority review by the regulatory authority in August. The NDA included data from the registration-enabling NAVIGATOR study, which showed that patients with PDGFRA Exon 18 mutant GIST achieved an objective response rate ("ORR") of 86% while the median duration of response ("DOR") was not reached. Meanwhile, patients with fourth-line GIST had an ORR of 22% while the median DOR was 10.2 months.
In October 2019, the FDA informed Blueprint Medicines that it intends to split the NDA into two separate NDAs, each for PDGFRA Exon 18 mutant GIST and fourth-line GIST.
The candidate was approved for treating PDGFRA Exon 18 mutant GIST last month under the tradename of Ayvakit. However, for approval in fourth-line GIST the regulatory body requested the company to submit data from ongoing phase III VOYAGER study, evaluating avapritinib in third and fourth-line GIST patients. The company expects to report top-line data from the study early in the second quarter of 2020..
Avapritinib is also under review in Europe for both PDGFRA Exon 18 mutant GIST and fourth-line GIST indications.
The phase III VOYAGER program is evaluating the safety and efficacy of avapritinib compared with Bayer’s (BAYRY - Free Report) Stivarga (regorafenib) in patients with the third or fourth-line GIST. The company also plans to initiate a global phase III precision medicine study, COMPASS-2L, to evaluate the safety and efficacy of avapritinib compared to Pfizer’s (PFE - Free Report) Sutent (sunitinib) for treating second-line GIST patients with pre-specified disease genotypes in 2020. It plans to submit a label expansion application to seek approval for avapritinib as a treatment for third-line GIST in the second half of 2020.
Other than GIST, Blueprint Medicines is pursuing a broad clinical development program for avapritinib across the advanced, indolent and smoldering forms of systemic mastocytosis ("SM"). Avapritinib is currently being evaluated in two studies, namely the phase I EXPLORER clinical study and the registration-enabling phase II PATHFINDER study for advanced SM. The company plans to submit a regulatory application seeking label expansion of avapritinib to include patients with advanced SM indication in the first quarter of 2020.
Apart from avapritinib, Blueprint Medicines has several pipeline candidates in early developmental stages. Notably, Blueprint Medicines presently has no approved product in its portfolio. Thus, approval of avapritinib will lower the company’s heavy dependence on its partners for revenue generation. The company is currently in partnership with Roche (RHHBY - Free Report) and CStone Pharmaceuticals for the development and commercialization of its pipeline candidates.
Blueprint Medicines currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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