The Food and Drug Administration on Friday approved the first drug for a common and irreversible form of vision loss. Whether the medicine, developed by Apellis Pharmaceuticals, can safely help patients maintain their sight for longer isn’t yet clear, though, setting up a potential debate among eye doctors about its use.

The regulator cleared the drug, which will be sold as Syfovre, for people with the condition, called geographic atrophy. An eye injection administered once every 25 to 60 days, the drug is meant to slow the progression of the disease, a condition Apellis believes to affect at least 1 million people in the U.S. alone.

To some experts, the agency’s decision is a milestone, marking at long last the availability of a medicine for a progressive and potentially debilitating condition.

“This is the beginning of a new therapeutic era” for geographic atrophy, said Eleonora Lad, an associate professor of ophthalmology at Duke University, and the lead investigator for the studies underlying the drug’s approval. “My patients are desperate for treatments that can slow the disease,” she said in a recent interview.

The FDA’s decision also boosts Apellis, a biotechnology company that aims to become profitable after years of losses. The company’s only product, Empaveli for a rare blood disease, generated an estimated $65 million in net sales in 2022. Wall Street analysts believe approval of Syfovre could translate into $3 billion in peak yearly sales for the company.

“We have built this company to be a large commercial organization,” said CEO Cedric Francois, in a recent interview.

Apellis priced the drug at $2,190 per vial, according to a company presentation Friday.

The company could have a difficult time making its case to doctors and patients. In testing, Syfovre slowed the growth of a marker of disease progression, but not the degradation of patients’ vision. It missed its main goal in one of the two key studies supporting its application, forcing Apellis to ask the FDA to delay its review to incorporate more data. And because the drug doesn’t restore lost vision, as medicines for other types of age-related vision loss can, patients may not easily see the treatment’s impact.

Treatment was also associated with a small, but increased risk of infections and inflammation, as well as the formation of new blood vessels, which can cause further vision loss and require additional therapy. The FDA placed warnings on the drug’s labeling for those side effects. 

The risks of their occurrence appear to grow with time, which has led some retinal specialists to question Syfovre’s potential.

“In my opinion, this drug is all pain and no gain,” said Demetrios Vavvas, an ophthalmology professor at Harvard Medical School and the director of the Retina Service at Massachusetts Eye and Ear, in an interview ahead of the FDA’s decision.

“We shouldn't base our decisions on emotions, or [an idea that] ‘we have to do something because we have nothing else.’ That ‘something’ may be worse than nothing,” he added. Vavvas isn’t associated with Apellis.

Geographic atrophy is triggered by age-related macular degeneration, or AMD, a leading cause of vision loss in the elderly. Damage to the macula  — the part of the eye responsible for central vision — causes progressive deterioration in sight and retinal cell death.

As a result, patchy lesions form that, under a microscope, look like minute geographic maps. Those lesions get larger with time, creating blind spots.

Their growth can’t be halted. While people with “wet” AMD, an aggressive form of the disease, have several drugs available that can restore vision, people with geographic atrophy don’t.

“It’s like a forest fire you cannot stop,” Francois said. “You start losing retina in a relentless, irreversible process, and there's nothing for these patients.”

Apellis isn't alone in trying to change that. Its medicine and another from a rival drugmaker, Iveric Bio, block activation of the complement system, a part of the body’s innate immune response. Study results from Iveric also showed its drug could slow lesion growth, and the FDA is set to decide on approval by Aug. 19.

In one of the key studies supporting Apellis’ approval, a monthly dose led to a 12% relative reduction in lesion growth compared to sham injections after a year, which wasn’t statistically significant. After two years, that relative reduction increased to 19%. An every-other-month injection seemingly worked better over time, too, rising from a 11% relative difference in lesion growth after one year to 16% after two in that same trial.

Apellis hasn’t yet proven slowing lesion growth corresponds to improved vision. In November, it reported that patients in its late-stage trials didn’t experience any visual acuity benefit after two years of treatment. There also weren’t meaningful differences on secondary study goals that evaluate vision, such as a person’s reading speed.

Lad claims those benefits could occur in the future, as the effects on lesion growth become more pronounced. “You get more and more prevention of loss over time,” said Lad. “To me it's pretty clear that this is working.”

It’s unclear how long patients would need to be treated before that point, though, and skeptics argue the risks of treatment could offset any positives.

“As the potential benefit goes up, the risk goes up,” said Vavvas, of Mass Eye and Ear. “And the benefit is potential. The risk is real.”

For example, the abnormal blood vessel growth observed in Apellis’ studies can make vision loss worse. A pooled safety analysis of the two Phase 3 trials showed about 6% of the patients who received monthly injections developed this condition after one year, and 12% after two years.

The risk was less with an every-other-month regimen, but still grew with time and was higher than those who’d received sham injections in Apellis’ trial. Rates of eye inflammation or infection were smaller, but climbed as well.

Vavvas is concerned a similar trend will play out in the real world on a larger scale. After an initial surge in use, he said, “side effects will come about, and people will stay away from it.”

A recent survey of 25 ophthalmologists, conducted by analysts with investment bank Jefferies, indicated Vavvas isn’t the only skeptic. Jefferies analysts found the group would likely only recommend drugs like Syfovre for about half of their geographic atrophy patients, and expected about a third of their patients to refuse treatment.

Francois, for his part, argues the risk of infection or inflammation is comparable to that of other injectable eye drugs. The formation of new blood vessels can be controlled with existing medicines, he added. “We do not expect the safety to be something that prevents the widespread use of this drug,” he said.

Lad, too, noted that AMD treatment has “improved substantially” and doesn’t expect issues. “It’s my belief that this is a safe regimen,” she said.

She is anticipating a learning curve, however. There’ll be logistical complexities integrating it into practice, for instance. Specialists will need to figure out who should get treated first, who might benefit the most and which regimen they should receive.

“There’s a lot of coming together and deciding things as a community that needs to happen with this,” she said. “It's brand new.”

Editor’s note: This story has been updated to include Syfovre’s list price.