The first therapy was approved for MASH by the FDA earlier this month. Image credit: Shutterstock/Cinemanikor.

The European Medicine’s Agency (EMA) has granted priority medicines (PRIME) status to 89bio’s pegozafermin, a drug being evaluated to treat patients with non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with advanced forms of fibrosis.

The EMA’s PRIME scheme is designed to expedite the development of medicines that meet an unmet need, such as rare diseases. The agency upgraded the scheme last year to enhance scientific advice and regulatory package preparation for sponsors of the scheme.

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89bio’s pegozafermin was granted PRIME status based on positive data from the company’s Phase IIb ELIVEN trial (NCT04929483), according to a 27 March press release. The randomised trial saw three doses of the drug administered either once weekly or biweekly in 192 patients with non-cirrhotic MASH with fibrosis (F2-F3) and MASH with compensated cirrhosis (F4).

Patients in the biweekly 44mg and once-weekly 30mg treatment groups saw significant one-stage fibrosis improvement without MASH worsening. MASH resolution without worsening of fibrosis and liver parameter improvements were also reported.

Pegozafermin is currently being evaluated in two Phase III trials from the 89bio’s ENLIGHTEN programme. The ENLIGHTEN-Fibrosis trial, initiated earlier this month, is slated to enrol around 1,000 patients. The second trial, ENLIGHTEN-Cirrhosis, is planned to begin in Q2 2024.

89bio said results from the ENLIGHTEN programme 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.

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Pegozafermin, an engineered glycoPEGylated analogue of fibroblast growth factor 21 (FGF21) works by exerting anti-fibrotic and anti-inflammatory effects on the liver. It is also being trialled in treating severe hypertriglyceridemia by 89bio in the Phase III ENTRUST trial.

Formerly known as non-alcoholic steatohepatitis (NASH), MASH can develop in patients with metabolic dysfunction-associated steatotic liver disease (MASLD). The accumulation of fat in the liver leads to inflammation that can cause liver damage over time.

While the MASH treatment landscape had become a graveyard of failed candidates, with key pharma players such as AstraZeneca and Gilead falling short of the regulatory mark, the race for the first FDA-approved therapy for the disease finally came to an end earlier this year, when Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) secured the agency’s approval.

Rezdiffra is forecast to generate $3.8bn in global sales by 2029, according to GlobalData’s Pharma Intelligence Centre.

GlobalData is the parent company of Clinical Trials Arena.

89bio’s CEO Rohan Palekar said: “We look forward to working closely with regulatory agencies as we continue to advance our Phase III clinical development programme, ENLIGHTEN, aimed at potentially benefiting advanced MASH patients and MASH patients with compensated cirrhosis.”