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

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

Shiwani Sharma

Shiwani Sharma is a Senior Editor at PharmaShots. She has in-depth knowledge of lifesciences industry including pharma and biotech sectors. She also covers news in field of Digital Health, Medtech and regulatory approvals from the US FDA, EU, and other global regulatory bodies in the industry. Additionally, she also writes Whitepapers, analysis reports, blogs on pharma and biotech industry. She is graduate in Biotechnology. She can be contacted on shiwani@pharmashots.com

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