Recent & Upcoming FDA Approvals: A Gamma Secretase Inhibitor Outside of Alzheimer’s, a PI3Ki Outside of Oncology, and More!

Pfizer spinout, SpringWorks Therapeutics, was granted priority review by the FDA for its oral gamma secretase inhibitor for the treatment of soft-tissue desmoid tumors, which has no FDA-approved therapies. Meanwhile, Pharming Groups’ oral PI3Kδ inhibitor (licensed from Novartis) received the first and only FDA-approval for the treatment of the rare, activated phosphoinositide 3-kinase delta syndrome, APDS.

In this update of small molecules in approval news for March 2023, we will cover potential upcoming FDA-approvals, as well as provide briefs on selected recent FDA-approvals, with the deep-dives for those to come later.

Want updates like this straight to your inbox? Sign up for our weekly newsletter here.

Potential Upcoming Approvals

nirogacestat

Nirogacestat has been granted FDA priority review for rare, aggressive, locally invasive, soft-tissue desmoid tumors (DTs), a condition with no FDA-approved treatments. This reversible, non-competitive gamma secretase inhibitor, which draws its anticancer activity from its ability to block Notch signaling, is the lead drug from Pfizer spinout SpringWorks Therapeutics, and has been granted Fast Track, Breakthrough Therapy and Orphan Drug designations from the FDA. The Ph. III candidate reduced the risk of disease progression by 71% and improved pain, other tumor-specific symptoms and physical functioning (150 mg BID, n = 142, NCT03785964). The PDUFA date is August 27th 2023. The small molecule is also in Ph. II for ovarian granulosa cell tumors (NCT05348356).