Apellis' closely watched eye drug gets new FDA goal date after changes to its filing scared investors

After some investor clamor, Apellis Pharmaceuticals’ blockbuster hopeful eye drug pegcetacoplan is back on the FDA’s review track.

The FDA has accepted a voluntary amendment to Apellis’ application for pegcetacoplan as an eye injection for geographic atrophy (GA), an advanced stage of age-related macular degeneration, the company said Friday. The agency has set a new target decision deadline of Feb. 26, 2023.

The FDA was previously expected to deliver a verdict by Nov. 26. But in a surprise development, Apellis earlier this month said it was submitting extended 24-month efficacy data—on top of the 18-month and one-year results in the original application package.

Investors were confused by the change in plans, and Apellis’ stock price tanked on the news at the time. Industry watchers feared that the company might have received some negative feedback from the FDA that prompted the change in plans.

For its part, Apellis believes that with the inclusion of the 24-month data, the company has the “potential to have the best product profile at launch for pegcetacoplan, with minimal impact to launch timing,” CEO Cedric Francois, M.D., Ph.D., said in a Friday statement.

Apellis has a lot hanging on pegcetacoplan, a C3 complement inhibitor that’s currently approved under the brand name Empaveli as an under-the-skin infusion for paroxysmal nocturnal hemoglobinuria. In that rare complement-driven disease, the drug faces competition from AstraZeneca’s C5 inhibitors Soliris and Ultomiris. In the third quarter, Empaveli only generated $17.7 million in the U.S.

By comparison, there’s no FDA-approved therapy for GA, which affects an estimated 1 million people in the U.S. alone. Analysts believe the drug could generate blockbuster-level sales in GA, with consensus forecasts collected by Evaluate Pharma recently putting the drug’s 2028 sales at $2.6 billion. Still, competition in the form of Iveric Bio's C5 agent Zimura is tailing close behind.

In its Friday announcement, Apellis again emphasized that the revision was “unsolicited,” meaning it wasn’t requested by the FDA.

During Apellis’ third-quarter earnings call last week, CEO Francois defended the decision, arguing that the revision would allow pegcetacoplan the “best possible label” at launch.

By Francois’ estimate, the extended review period would mean just a six-week delay to the commercial lunch of pegcetacoplan in GA. But submitting 24-month results after an initial approval would mean a separate filing that could take months to work its way through the FDA process, he added.

Apellis says it was emboldened by the longer-term data from its two phase 3 trials, Derby and Oaks. In the trials, pegcetacoplan showed “an acceleration in the reduction in lesion growth” after the 24-month follow-up.