Mirum Pharmaceuticals Submits European Marketing Authorization Application for Maralixibat in Alagille Syndrome Supported by New Positive Results from Natural History Study Comparison

- Marketing Authorization Application for treatment of cholestatic disease in Alagille syndrome submitted.

- Natural history analysis shows significant improvement in event-free survival and transplant-free survival.

- PFIC regulatory strategy in Europe to focus on results of MARCH-PFIC Phase 3 study, expected in second quarter of 2022.

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the submission of a Marketing Authorization Application (MAA) for the treatment of cholestatic liver disease in patients with Alagille syndrome (ALGS) to the European Medicines Agency (EMA). 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conjunction with the ALGS submission, Mirum’s MAA for progressive familial intrahepatic cholestasis type 2 (PFIC2) was withdrawn with plans to re-submit after availability of results from the ongoing MARCH-PFIC Phase 3 study in a broader set of PFIC sub-types and with higher doses of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