ProQR’s Sepofarsen Receives FDA’s Rare Pediatric Disease Designation to Treat Leber’s Congenital Amaurosis 10

ProQR’s Sepofarsen Receives FDA’s Rare Pediatric Disease Designation to Treat Leber’s Congenital Amaurosis 10

Shots:

  • The US FDA has granted RPD designation to Sepofarsen for the treatment of Leber’s congenital amaurosis 10 (LCA10) which is the most common cause of blindness due to genetic disease in pediatric patients
  • The RPD program provides PRV to the sponsor which can be redeemed to receive a priority review by the FDA of a subsequent marketing application for a different product
  • Sepofarsen (intravitreal injection) is an investigational RNA-based oligonucleotide, addressing the cause of LCA10 due to the p.Cys998X mutation in the CEP290 gene and has received FDA’s ODD & FT designation with EMA’s ODD, FT and PRIME designation

Click here to­ read full press release/ article | Ref: ProQR| Image: Troutaccess