FDA to split Blueprint's cancer drug application in two

The Food and Drug Administration will review a biotech company’s approval application for a cancer drug in two parts.

Cambridge, Massachusetts-based Blueprint Medicines said Monday that the FDA would split its application for the drug avapritinib in gastrointestinal stromal tumor into two separate applications – one for GIST with a PDGFRa Exon 18 mutation and another for fourth-line GIST. The agency indicated to the company that data from its Phase III VOYAGER study – which has been accelerated, with data expected in the second quarter of 2020 – would be informative in its review of the fourth-line GIST indication. The company said an extension of the review period for the fourth-line GIST application will likely be required to enable Blueprint to provide top-line data from VOYAGER.

With the Rise of AI, What IP Disputes in Healthcare Are Likely to Emerge?

Munck Wilson Mandala Partner Greg Howison shared his perspective on some of the legal ramifications around AI, IP, connected devices and the data they generate, in response to emailed questions.

Shares of Blueprint were down about 5 percent on the NASDAQ Monday morning following the news. GIST is a sarcoma, meaning a cancer of the connective tissue. Avapritinib is an inhibitor of KIT and PDGFRa that is also in development for advanced systemic mastocytosis, a type of blood cancer. Novartis’ Gleevec (imatinib), now available generically, is also a KIT inhibitor used to treat GIST. Another company, Deciphera Pharmaceuticals, is also developing a drug targeting KIT and PDGFRa for fourth-line GIST, ripretinib.

“Throughout the development of avapritinib, we have had a productive and collaborative dialogue with the FDA about the potential for avapritinib to address important medical needs in subsets of patients with advanced GIST,” Blueprint CEO Jeff Albers said in a statement. “We plan to continue to work closely with the FDA during its review of the separate [New Drug Applications] for PDGFRa Exon 18-mutant GIST and fourth-line GIST, and we plan to submit the requested VOYAGER trial data as expeditiously as possible.”

VOYAGER is enrolling patients with third- and fourth-line GIST and is expected to complete enrollment by the end of next month, with the ClinicalTrials.gov page stating an enrollment target of 460 patients and an April 2021 primary completion date.

In a note to investors following the announcement, Cowen analyst Marc Frahm wrote that the news was “slightly disappointing” as it means there will likely be a delay in avapritinib’s approval for fourth-line GIST. The best-case and most likely scenario, he wrote, is that the agency would request a three-month extension of the original target date, pushing it to May 14, 2020 and then approving avapritinib at that time. However, a complete response letter from the FDA rejecting the drug would make timelines less clear, but a target date at the end of 2020 would still be possible. Frahm noted that Deciphera plans to file for approval of ripretinib in the first quarter of 2020, with the possibility of winning approval by the end of the year.