Geographic atrophy, an eye disorder that starts as a loss of central vision and progressively worsens to total blindness, now has its first treatment. The FDA on Friday approved an Apellis Pharmaceuticals drug that slows the progression of this disease, which affects more than 1 million people in the U.S.
Approval of the Apellis drug, pegcetacoplan, covers all patients with geographic atrophy, a broad label that reflects the representative patient population tested in pivotal studies, Chief Medical Officer Caroline Baumal said during a Friday evening conference call. The Waltham, Massachusetts-based company plans to launch the drug in March, marketing the product under the name “Syfovre.”
“As a retinal physician, having to tell your patient that they will ultimately go blind, and nothing could be done about it, was one of the hardest conversations to have,” Baumal said. “Thankfully, with this approval, this will no longer be the conversation.”
Geographic atrophy, or GA, is an advanced form of a degenerative retinal disorder called age-related macular degeneration (AMD). People who have GA develop lesion growths that destroy retinal cells. The progression of this disease is associated with excessive activity of the complement system, a part of the immune system. The Apellis drug is comprised of two peptides that together block a complement system protein called C3. This approach is used in Apellis’s first FDA-approved product, Empaveli. That infused therapy treats the rare blood disorder paroxysmal nocturnal hemogloblinuria. Syfovre was designed for injection into the eye, similar to other retinal drugs.
FDA approval of Syfovre is based on the results of two sham-controlled Phase 3 studies, each with cohorts for monthly dosing as well as every-other-month dosing. Treatment with the Apellis drug led to reductions in the rate of lesion growth measured at 24 months that was comparable across both pivotal studies. Patients from both studies are also participating in an extension study that will follow them for another three years to see if the effects observed at 24 months continue. The greatest reduction in lesion growth was 36% measured in months 18 to 24 in a cohort that received monthly dosing of the Apellis drug.
Baumal said that the ability to offer monthly dosing or every-other-month dosing gives physicians flexibility. She thinks many patients will prefer every-other-month dosing, but she added that the extension study will provide more information about how patients respond to each injection regimen. In the two pivotal studies, the most common adverse reactions reported were eye discomfort, neovascular age-related macular degeneration (the “wet” form of AMD), vitreous floaters, and conjunctival hemorrhage. The label cautions that eye injections may lead to a type of inflammation called endophthalmitis as well as retinal detachment. Baumal noted that those problems were reported in less than 1% of patients in the clinical trials.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Apellis will charge $2,190 per vial of Syfovre, a price that Chief Commercial Officer Adam Townsend said reflects the drug’s status as the first GA treatment while also staying in line with the prices of currently available AMD drugs. The number of vials needed will depend on whether a patient receives monthly or every other month dosing, and whether the patient has GA in one or both eyes. But Baumal estimates that patients will need six to eight vials of Syfovre per year.
Syfovre may be the first GA treatment but competition looms. On Thursday, Iveric Bio announced that the FDA accepted its application for avacincaptad pegol, a drug designed to block a complement system protein called C5. A regulatory decision for Iveric’s drug is expected in August. Ionis Pharmaceuticals is developing an antisense oligonucleotide designed to block production of a protein called complement factor B. The Ionis research is partnered with Roche. But some have dropped out of the GA chase. Merck-partnered NGM Biopharmaceuticals exited after its drug candidate failed a Phase 2 test last year.
Geographic atrophy affects an estimated 5 million people worldwide, according to Apellis. Syfovre is under review in Europe, where a regulatory decision is expected next year. A marketing application has been submitted in Canada. Apellis expects to file additional applications in Switzerland, the U.K., and Australia later in the current quarter.
Photo by Apellis Pharmaceuticals
