89bio EMA Grants Priority Status for Pegozafermin MASH Candidate

The prioritization of medicines (PRIME) status has been granted to pegozafermin, a drug developed by 89bio for the non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with advanced forms of fibrosis, by the European Medicine's Agency (EMA). 

The PRIME initiative of the EMA is specifically intended to accelerate the development of medicines that can cater to an unmet need, such as rare diseases. Last year, the agency upgraded its scheme to improve scientific advice and regulatory package preparation for scheme sponsors.

89bio's pegozafermin drug was awarded PRIME status based on positive data from the company's Phase IIb ELIVEN trial, according to a press release on 27 March.

The randomized trial involved 192 patients with non-cirrhotic MASH with fibrosis (F2-F3) and MASH with compensated cirrhosis (F4), who were administered three doses of the drug either once weekly or biweekly.

Significant one-stage fibrosis improvement without MASH worsening was observed in patients who received the biweekly 44mg and once-weekly 30mg treatment. MASH resolution without worsening of fibrosis and improvements in liver parameters were also reported.

Pegozafermin is currently being evaluated in two Phase III trials from the 89bio's ENLIGHTEN program. The ENLIGHTEN-Fibrosis trial, initiated earlier this month, is expected to enroll approximately 1,000 patients. The second trial, ENLIGHTEN-Cirrhosis, is scheduled to begin in Q2 2024.

According to 89bio, the results obtained from the ENLIGHTEN program will be used to support regulatory filings in the US and Europe. The drug also received breakthrough therapy designation from the US Food and Drug Administration (FDA) in September 2023.

Pegozafermin, an engineered glycosylated analog of fibroblast growth factor 21 (FGF21), operates by exerting anti-fibrotic and anti-inflammatory effects on the liver. In the Phase III ENTRUST trial, 89bio is also testing its effectiveness in treating severe hypertriglyceridemia.