Research & Development
Mirum Pharmaceuticals Submits European Marketing Authorization Application for Maralixibat in Alagille Syndrome Supported by New Positive Results from Natural History Study Comparison
13 September 2021 - - US-based biopharmaceutical company Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) has submitted a Marketing Authorization Application for the treatment of cholestatic liver disease in patients with Alagille syndrome to the European Medicines Agency, the company said.

In conjunction with the ALGS submission, Mirum's MAA for progressive familial intrahepatic cholestasis type 2 (PFIC2) was withdrawn with plans to re-submit after availability of results from the ongoing MARCH-PFIC Phase 3 study in a broader set of PFIC sub-types and with higher doses of maralixibat.

The submission of the ALGS MAA presents an opportunity to deliver the first ever pharmacologic treatment option for this devastating disease for which there is significant and urgent unmet need.

This submission follows discussion with the EMA on the appropriate strategy for seeking marketing approval for both ALGS and PFIC broadly as soon as possible.

The MAA is comprised of the long-term ICONIC study in patients with ALGS, which showed a significant improvement on pruritus (p
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