The FDA has just approved Syfovre (pegcetacoplan), developed by Apellis Pharmaceuticals, as the first ever treatment for geographic atrophy (GA). This rare form of blindness affects millions of people worldwide, but now there is finally hope for those affected. Syfovre has been tested and proven safe and effective, offering a glimmer of hope to those who have been struggling with this vision-robbing condition.
Age-related macular degeneration affects more than five million people worldwide and can cause the retinal cells to waste away and die, resulting in a decline in vision. Until now, there have been no treatments to slow the progression of this condition. However, recent developments have made it possible to take steps to protect and preserve eyesight.
On average, the effects of this condition start to become visible within 2.5 years, eventually impairing your central vision.
Eleonora Lad, the lead investigator of the Phase III OAKS and DERBY trials that was the key to the treatment’s approval, described the FDA decision as a groundbreaking moment in retinal ophthalmology, proclaiming it to be “the most important event in more than a decade.”
The DERBY clinical study revealed the remarkable potential of Syfovre as an ongoing treatment for GA, showing up to a 36% reduction in lesion growth over a period of 6 months. With its flexible dosing, from every 25 to 60 days, Syfovre’s efficacy increases as treatment goes on.
The long-awaited injection is finally set to hit the market in the beginning of March – but it doesn’t come cheap. According to the company, each vial of the medication will cost a staggering $2,190 before any discounts are applied.
The FDA has given a second approval to pegcetacoplan, a drug used to treat paroxysmal nocturnal hemoglobinuria – a rare, life-threatening blood disorder where the immune system attacks red blood cells and platelets. Marketed as Empaveli, pegcetacoplan has the potential to bring relief to those suffering from the debilitating condition.
Pegcetacoplan is a revolutionary approach to controlling inflammation and motor neuron damage: by blocking C3 in the complement system, its effects on the downstream immune response can be regulated, providing vital protection to the body.
Developing treatments for some of the most debilitating and difficult-to-treat conditions, this drug is being studied for the treatment of Amyotrophic Lateral Sclerosis (ALS), rare kidney diseases, coronary artery disease, Wet Age-Related Macular Degeneration (AMD) and a common complication of stem cell transplantation. Researchers hope that these treatments will provide a ray of hope to those suffering from these conditions.
Competition in the GA market has been intensifying recently, with the FDA’s recent decision adding to the fire. With more players entering the arena, the race to provide the best quality of care is well and truly on. It’s a thrilling time to be a part of the GA industry, and the future looks bright.
Last week, Iveric Bio made a breakthrough in the medical world with the news that the FDA had accepted its New Drug Application (NDA) and granted it priority review for a C5 inhibitor, a potential treatment for the debilitating eye disease, Geographic Atrophy (GA). In two Phase III trials, the candidate drug showed unprecedented efficacy rates of up to 35% in 12 months – a significant milestone in the fight against this life-altering condition.
In a major move to acquire cutting-edge gene therapy technology, Novartis shelled out $1.5 billion to acquire UK-based Gyroscope Therapeutics in late 2021. The drug, GA Gene Therapy, is still in its early stages, with first planned submissions for the drug slated for 2026 or later. It will be an exciting journey for the company and its stakeholders as they look to bring this innovative therapy to the market.