Apellis Pharmaceuticals has just announced that they have received Orphan Drug Designation from the FDA for APL-2, a C3 complement inhibitor. The company is currently investigating the drug as a potential treatment for C3 glomerulopathy (C3G).
C3G causes an accumulation of C3 within the glomeruli. This is the part of the kidney which handles blood filtration and an excessive amount of C3 within it can ultimately lead to end-stage renal disease. Currently, there are no approved treatments for C3G and about half of patients advance to end-stage within 10 years of the time they receive their diagnosis. APL-2 works to inhibit C3, and Apellis believes it may be an effective treatment for this condition.
Apellis is currently working on a Phase 2 trial examining the effect of APL-2 as a treatment for four different types of glomerular conditions including C3G, lupus nephritis, membranous nephropathy, and igA nepropathy.
Orphan Drug Designation
Orphan Drug Designation for APL-2 provides Apellis with consultation by the FDA on their trial designs, fee exemptions/reductions, 7 years market exclusivity within the United States, and could potentially expedite the drugs development. This designation is provided to novel treatments which the FDA believes could aid those living with rare conditions.
Apellis is excited about their drugs new designation because it indicates that the FDA recognizes the potential it has to help patients. There is truly an unmet need for C3G and this drug could be life-changing for those affected by the condition. So far in studies APL-2 has been well tolerated and there have been no significant infections reported.
Fingers crossed that the current Phase 2 trial continues to show positive results for this new treatment and that the drug will receive FDA approval soon.
You can read more about this drug and its latest developments here.