Ocelot Bio, Inc., a clinical-stage biopharmaceutical company focused on the development of innovative therapeutics to treat complications of end-stage liver disease (ESLD), announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead candidate OCE-205 for the treatment of hepatorenal syndrome. Ocelot Bio is currently enrolling patients in a Phase 2 clinical trial of OCE-205 in hepatorenal syndrome with acute kidney injury (HRS-AKI), a serious and rapidly progressive consequence of ESLD that leads to acute renal failure and is associated with high rates of morbidity. There has been a lack of therapeutic drug innovation for HRS-AKI and, currently, there are no FDA-approved treatments for the disease.
“Receiving Orphan Drug Designation for OCE-205 underscores the urgent unmet medical need for patients with hepatorenal syndrome,” said Katherine Vega Stultz, president and chief executive officer at Ocelot Bio. “We are actively engaged with leading medical centers to advance our Phase 2 clinical study, and our team is working towards better understanding patient outcomes to continue to progress with urgency our development program for OCE-205.”
The FDA grants Orphan Drug Designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides Ocelot Bio certain benefits, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity for the drug for the designated orphan indication in the U.S. if the drug is ultimately approved for its designated indication.