Positive top-line data from Blueprint Medicines Corp.’s phase I and II trials of Ayvakit (avapritinib) for treating advanced systemic mastocytosis (SM) are pushing the company to submit an sNDA before 2020 ends.
The FDA’s approval of Genentech Inc.’s Gavreto (pralsetinib) for treating adults with metastatic rearranged during transfection (RET) fusion-positive non-small-cell lung cancer (NSCLC) follows the FDA’s May approval of Eli Lilly and Co.’s Retevmo for patients whose tumors have a RET alteration. Gavreto will be commercialized in the U.S. by Genentech, part of the Roche Group, along with Blueprint Medicines Corp., which developed the once-daily oral therapy. Outside the U.S., Roche will handle commercialization.
DUBLIN – Blueprint Medicines Corp. is banking $775 million, including $675 million up front and another $100 million as an equity investment, from a co-commercialization deal with Roche Holding AG and its Genentech subsidiary involving RET inhibitor pralsetinib. The deal also includes up to $927 million in development, regulatory and commercial milestones, $90 million of which are described as “near-term,” plus tiered royalties on ex-U.S. sales, ranging from the high-teens to mid-twenties.
Only hours after Blueprint Medicines Corp. disclosed an FDA complete response letter for avapritinib in fourth-line gastrointestinal stromal tumor (GIST), Deciphera Pharmaceuticals Inc.’s kinase inhibitor, ripretinib, won the agency’s approval for the same indication, well ahead of its Aug. 13 PDUFA date.
Three biopharma drugs are up for FDA approval this week, including one new chemical entity, dasotraline, from Sunovion Pharmaceuticals Inc. to treat binge eating disorder, and two other candidates that are part of supplemental filings for expanded indications.
The phase III failure of Blueprint Medicines Corp.’s avapritinib to meet its primary endpoint of improving progression-free survival (PFS) in patients with locally advanced unresectable or metastatic gastrointestinal stromal tumors (GIST) pummeled the company stock on Tuesday but boosted shares and hopes at Deciphera Pharmaceuticals Inc.
Investors in small and midsized biopharma companies were certainly rewarded in 2019, with group members in the BioWorld Drug Developers index on a tear. The price-weighted index returned 40% in value thanks to a steady flow of positive regulatory and clinical trial results from the companies throughout the year. However, investors may be less impressed with the start they have made this year, with the index dipping 8.4% in January.
Cambridge, Mass.-based Blueprint Medicines Corp.’s price tag and label for Ayvakit (avapritinib) caused some chagrin on Wall Street as observers continued to weigh the drug’s odds against ripretinib, the competing drug for which Deciphera Pharmaceuticals Inc. awaits regulatory action.
