Apellis wins FDA approval for first geographic atrophy drug

One of Fierce Pharma’s top three most anticipated drug launches of 2023 was also one of the year’s most uncertain. But on Friday, Apellis Pharmaceuticals bucked the industry’s losing trend in geographic atrophy (GA), scoring a second commercial nod for its C3 inhibitor pegcetacoplan.

The FDA has approved pegcetacoplan under the name Syfovre as the first treatment for GA—an advanced stage of age-related macular degeneration that is also a leading cause of blindness.

“Think of having a forest fire in your retina, where your retina slowly but certainly dies completely,” Cedric Francois, M.D., Ph.D., Apellis’ co-founder and CEO, said of the disease in an interview ahead of Syfovre’s approval.

Prior to Syfovre, there were no FDA-approved therapies for geographic atrophy. Now, Apellis hopes the story can be re-written.

It’s important to note, however, that “this is not a drug that flips the switch on day one,” the CEO pointed out.

Syfovre is no cure, with approval based on the drug’s ability to slow growth of a marker of disease progression, rather than the ability to improve or even stall central vision loss.

“It’s a drug where you make an investment over many years, and where the longer you stick to the therapy, the better this drug is going to work for you,” Francois said.

Apellis sees that as one of its drug’s core strengths. “The longer you treat, the better this drug works,” he explained.

Another potential edge for Apellis’ drug is its flexible dosing regimen, which is every 25 to 60 days. Further, Apellis has worked hard to ensure its therapy works in “any type” of GA patient, Francois said. “Regardless where the lesion in your eye is located—whether it’s centrally, whether it’s more in the periphery—our drug can slow these lesions down.”

The FDA based its approval on positive 24-month results from Apellis’ late-stage Derby and Oaks studies. The trials found Syfovre lowered the rate of GA lesion growth versus placebo and demonstrated increased treatment effects over time. The greatest benefit seen—tracking on the high-end at a 36% reduction in lesion growth—was between the 18- and 24-month mark, Apellis said in a release.

Syfovre is expected to hit the market by the beginning of March, Apellis said. The drug will be made available through specialty distributors and pharmacies around the U.S. It costs $2,190 per vial before discounts, Apellis said in a company presentation Friday.

Apellis has been working “intensively” on its GA launch for the past four years, Francois said. In 2022, the company recruited around 100 field-based representatives to chip in on the launch, including sales and medical affairs reps, reimbursement managers and more. Those representatives have now been on the road for more than two quarters building awareness with doctors and patients, Francois said.

Apellis entered the commercial realm with pegcetacoplan’s first approval in the rare blood disorder paroxysmal nocturnal hemoglobinuria in 2022. The drug made $65 million in sales in its first year on the market. In GA, meanwhile, pegcetacoplan could reach sales of around $2.6 billion by 2028, Evaluate Pharma has predicted.

Still, pegcetacoplan’s approval in GA was anything but certain. Pharma has tried and failed to push GA treatments over the FDA’s finish line in recent years, including an effort by Roche on its complement-inhibiting drug lampalizumab.

Plus, competition could be close behind, with Iveric Bio’s C5 agent Zimura hot on Syfovre’s tail. In fact, Apellis’ approval comes just one day after Iveric’s drug won priority review at the FDA, teeing up a decision date of Aug. 19.