Drug pipeline 2Q23 — controversy and complexity

The US Food and Drug Administration (FDA) verdict on Sarepta’s Elevidys, a gene therapy delivering microdystrophin to patients with DMD, proved to be the most controversial decision of the quarter. The company teased positive efficacy from the data for patients aged 4–5 but not for older peers, with Sarepta’s fervent promise of data by the fall from a randomized trial in that narrow patient band clinching an accelerated approval. The FDA also approved another complex therapy, Seres Therapeutics’ Wowst, the first oral microbiome therapy for recalcitrant Clostridioides difficile–associated diarrhea. At the more structurally defined end of the spectrum, Pfizer’s inhibitor of coronavirus 3C-like-proteinase, Paxlovid, became the first oral antiviral approved for the treatment of COVID-19 infections. Between the extremes of complexity falls Krystal Biotech’s Vyjuvek, a redosable gene therapy for the severe skin disorder dystrophic epidermolysis bullosa, caused by inherited deficiencies in collagen VII: Vyjuvek is the first approved topically applied gene therapy and the first based on herpes simplex virus-1 (HSV-1). The impending 2023 Northern Hemisphere respiratory disease season will see the availability of two adult vaccines for respiratory syncytial virus (RSV) infections, with the back-to-back approvals of two products based on stabilized pre-fusion F protein subunit: GSK’s Arexvy and Pfizer’s Abrysvo. As the quarter came to a close, the FDA approved the first cell therapy for type 1 diabetes, CellTrans’s Lantidra (allogeneic deceased donor pancreas–derived islets of Langerhans) and the first gene therapy for hemophilia A, Roctavian.

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