FDA grants orphan drug designation to Ocelot Bio’s therapeutic peptide for ascites
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The FDA has granted orphan drug designation to Ocelot Bio’s OCE-205, a mixed vasopression 1a receptor agonist-antagonist peptide for the treatment of ascites resulting from all causes except cancer, according to a company release.
This is the second orphan drug designation for OCE-205, the first being for hepatorenal syndrome in 2022, the company noted. Ocelot Bio’s sole drug candidate is now positioned to address multiple complications of end-stage liver disease with an additional indication for ascites, or excess fluid in the peritoneal cavity, a known complication of advanced decompensated cirrhosis.
“The FDA granting orphan drug designation for OCE-205 in ascites is validation of the tremendous need for improved therapies offering novel approaches for patients and supports our clinical focus on this important indication,” Lise Kjems, MD, PhD, chief medical officer at Ocelot Bio, said in the release. “This milestone serves as a catalyst as we work with patients, health care professionals and health authorities to pave a brighter path forward for these patients who have limited treatment options. We are well positioned to further advance our clinical development program for OCE-205 and look forward to progressing this program in ascites.”
In previous preclinical trials, OCE-205 demonstrated it can reduce portal hypertension and improve ascites volume, suggesting its potential use to treat decompensated cirrhosis complications such as ascites, the release noted.
The company plans to begin clinical studies of OCE-205 in refractory ascites in 2024.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
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