The US Food and Drug Administration (FDA) has announced an extended review of the Supplemental New Drug Application (sNDA) for Mirum Pharmaceutical’s Livmarli (maralixibat) to treat patients with cholestatic pruritus in progressive familial intrahepatic cholestasis (PFIC).

The company announced that the FDA extended the prescription drug user fee act (PDUFA) date to allow time for a full review of a submission provided in response to an FDA information request, which is deemed to be a major amendment. The date has been pushed from 13 December to 13 March, 2024.   The news was announced after markets closed on 17 October. Earlier today, Mirum’s stock opened more than 4% lower than the previous day.

Mirum submitted the sNDA in February of this year based on the Phase III MARCH trial. The study was the largest randomised trial conducted for patients with PFIC. A statistically significant difference in efficacy was reported between the Livmarli and placebo arms. Following this, Mirum submitted a new drug application for Livmarli’s approval in cholestatic pruritus in PFIC patients ages three months and older.  

The FDA has not requested any additional clinical trials to be conducted as part of the extended review.

Livmarli is an ileal bile acid transporter inhibitor and is the only medication approved by the FDA for the treatment of cholestatic pruritus in patients with Alagille Syndrome ages three months and older.  

In April this year, Mirum submitted a marketing authorisation variation application to the European Medicines Agency (EMA) for expanding the use of Livmarli to treat PFIC patients ages two months and above. Livmarli was approved for the treatment of Alagille Syndrome patients ages two months and older in the EU in December 2022.  

Livmarli generated $40,228, in accordance with Mirum’s 2022 annual report. According to GlobalData, Livmarli is expected to generate $9.07bn in sales by 2029.

Source:

https://www.pharmaceutical-technology.com/news/mirums-plans-to-expand-livmarlis-market-get-hit-as-fda-pushes-approval-date/?cf-view