Picture this: You've spent more than a decade researching the immune system signaling pathway known as the complement system and how it contributes to blindness in the elderly. Despite the lack of success in clinical trials, you don't get discouraged because it's your passion. And then, on February 17, 2023, there is this breakthrough. Apellis Pharmaceuticals' drug Syfovre is approved by the FDA to treat dry age-related macular degeneration (AMD) by preventing activation of the complement pathway and slowing, at least in part, the progression of cell death in the retina.
Since 1996, when the first peptide that inhibits the function of complement protein C3 was isolated using PhageDisplay technology, it has been continuously developed and approved in various formats for the treatment of different diseases (Compstatin, APL-2, Pegcetacoplan, Empaveli). And now, after more than 25 years, ophthalmologists worldwide can finally offer Syfovre treatment to millions of patients with dry AMD/geographic atrophy who previously had limited options, such as "live healthy," "don't smoke" or "protect eyes from sunlight" (all important tips!!!!!!!).
Syfovre is administered at a concentration of 150 mg/ml monthly to bi-monthly as a 0.1-ml injection into the eye, a routine procedure for ophthalmologists but one that carries a cost of $2190 per dose.
However, our research is even more relevant and significant than before. These complement inhibitors used to treat AMD do not stop the disease, but they do reduce its progression. Treated patients have 20% fewer dead areas in the retina after 2 years than untreated patients. We need to continue research to understand the underlying mechanisms and to be able to help the five million people affected by this disease worldwide. (The approval decision in Europe is planned for early 2024.)
Despite the challenges, we can finally celebrate because there is now a light at the end of the tunnel for patients with dry AMD. And we will continue to work hard to ensure that this hope becomes a even a better reality for all who need it.
Nice Review - describing the history (develeopment of the drug)