FDA Grant Rare Pediatric Disease Designation to Omeros’s Antibody for C3 Glomerulopathy
Biopharma company Omeros have announced that their monoclonal antibody complement inhibitor zaltenibart has received rare pediatric disease designation from the FDA. The agency has given the designation to the antibody for the treatment of complement 3 glomerulopathy (C3G), an extremely rare and progressive renal disorder affecting children and young adults.
There’s currently no approved treatment for C3G, which is caused by dysregulation of the alternative pathway of complement. As a result, most patients develop end-stage renal disease within 10 years after diagnosis.
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Zaltenibart, Omeros’s solution to this clinical need, targets and blocks mannan-binding lectin-associated serine protease-3 (MASP-3), the key activator of the alternative pathway of complement. This stops the conversion of pro-complement factor D (pro-CFD) to mature CFD.
Omeros has scheduled Phase III clinical trials for zaltenibart in C3G to begin next year.
“C3G is devastating for children as well as for adults, and our receipt of FDA’s rare pediatric disease designation is a welcome acknowledgment of zaltenibart as a potential therapeutic for this disease that has no approved treatment,” said Gregory A Demopulos, Chairman and CEO of Omeros.
“With zaltenibart clinical studies ongoing in both PNH and C3G and preparations underway to begin Phase III trials, we look forward to bringing zaltenibart to market, expanding its list of targeted indications and demonstrating its advantages over other alternative pathway inhibitors.”
The FDA awards rare pediatric disease designation to encourage the development of drugs for rare diseases primarily affecting children. With this designation, Omeros receives a rare pediatric disease priority review voucher.
The voucher allows the user to obtain priority review of either a New Drug Application (NDA) or Biologics License Application (BLA), which can cut down the review time by at least four months. Omeros can choose to use the voucher themselves or sell it to another company for their use.
Zaltenibart is also being investigated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), another ultra-rare and life-threatening blood disease. The FDA gave the company orphan drug status for this indication.
Omeros says that they have held productive meetings with regulatory authorities including an end-of-phase 2 meeting with the FDA and a scientific advice meeting in Europe.
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