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August 23, 2022

FDA grants Orphan Drug status to Ocelot’s hepatorenal syndrome therapy

A placebo-controlled, randomised, multicentre Phase II trial of OCE-205 is underway in patients with HRS-AKI.

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to Ocelot Bio’s OCE-205 to treat hepatorenal syndrome. 

A lead candidate of the company, OCE-205 is a peptide therapeutic.

Possessing a distinguished mechanism of action (MOA), it acts on serious hemodynamic complications caused by liver fibrosis and portal hypertension in end-stage liver disease (ESLD). 

It is currently being analysed for treating hepatorenal syndrome with acute kidney injury (HRS-AKI).

A placebo-controlled, randomised, multicentre, double-blind, adaptive dose-ranging Phase II clinical trial of OCE-205 in HRS-AKI is enrolling participants. 

It will analyse the safety and efficacy of OCE-205 in adult HRS-AKI patients having cirrhosis with ascites. 

Ocelot Bio president and CEO Katherine Vega Stultz said: “Receiving Orphan Drug Designation for OCE-205 underscores the urgent unmet medical need for patients with hepatorenal syndrome. 

“We are actively engaged with leading medical centres to advance our Phase II clinical study, and our team is working towards better understanding patient outcomes to continue to progress with urgency our development program for OCE-205.”

​According to data from a Phase I trial of OCE-205, healthy participants showed to have predicted vasoconstriction activity in a dose-response way with the expected variation in mean arterial pressure. 

It also showed to possess a promising safety profile. 

Orphan Drug status is granted by the regulatory agency to therapies and drugs intended to treat, diagnose or prevent rare diseases or ailments that impact less than 200,000 people in the country. 

A serious and quickly progressive consequence of ESLD, HRS-AKI causes acute renal failure and is linked to increased morbidity rates. 

Currently, there exist no therapies approved by the Food and Drug Administration for treating HRS-AKI.

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