FDA Requested Change from Type-C Meeting to pre-NDA Meeting
Company Plans to Initiate Rolling NDA Submission in Third Quarter 2020
Rare Pediatric Disease Designation Granted for Maralixibat for Alagille Syndrome
Conference Call and Webcast today at 8:30 a.m. ET / 5:30 a.m. PT
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced the successful completion of its clinical pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of pruritus associated with Alagille syndrome. In addition, the company had a chemistry, manufacturing and controls (CMC) meeting with the FDA. The purpose of the meetings was to discuss and confirm the clinical, non-clinical and CMC requirements for the company’s proposed NDA submission. The company also today announced that Rare Pediatric Disease Designation has been granted for maralixibat for Alagille syndrome, and thus may qualify for the receipt of a priority review voucher if the NDA is approved by the FDA. Maralixibat was recently granted Breakthrough Therapy Designation by the FDA for Alagille syndrome.
The company plans to pursue full approval for the indication of pruritus associated with Alagille syndrome based on feedback from the FDA and the results from the ICONIC study of maralixibat in children with Alagille syndrome. As a result of the discussions with the FDA, the company is planning a rolling NDA submission, which will be initiated in the third quarter of 2020, with full CMC sections submitted in the first quarter of 2021.
“We are pleased that the FDA recommended that we change the classification of our requested Type C meeting to a pre-NDA meeting and that we have a path to a rolling NDA submission for maralixibat beginning in the third quarter of 2020,” said Chris Peetz, president and CEO of Mirum. “We look forward to working with the FDA to bring this much-needed treatment option to children living with Alagille syndrome as rapidly as possible. As part of our commitment to patients and their families we also plan to initiate an expanded access program in the second half of 2020.”
Commenting on the recent progress, Emmanuel Gonzales M.D., Ph.D., Professor of Pediatrics, and Emmanuel Jacquemin M.D., Ph.D., Professor of Pediatrics and the study’s principal investigator from the Hôpital Bicêtre, Paris, France jointly stated, “The potential that maralixibat shows is incredibly exciting. The data generated both from the ICONIC study and the long-term data presented at the Liver Meeting in November 2019 demonstrated encouraging results on cholestasis and quality of life of patients with this severe disease. New therapeutic options are desperately needed to manage cholestasis and the associated manifestations in children with Alagille syndrome.”
About the ICONIC Study in Alagille Syndrome
The ICONIC study is a Phase 2b placebo-controlled randomized drug withdrawal study. Analyses include pruritus measures using the ItchRO (Observer) score, Clinician Xanthoma Severity Scale, Pediatric Quality of Life Inventory (PedsQL) and safety parameters.
A total of 31 children (mean age 5.4 years) were enrolled in the study. All patients in the study were treated for 18 weeks with maralixibat (up to 400 mg/kg/day) before they were randomized to receive either placebo (n=16) or continued maralixibat (n=13) for 4 weeks. After the randomized withdrawal period, all patients were treated with maralixibat for the remainder of the 48 weeks. 29 of the 31 patients enrolled in the study were included in the Week 48 analysis.
Overall, treatment with maralixibat led to clinically relevant and statistically significant reductions of serum bile acid (sBA) levels and pruritus compared to baseline at Weeks 18 and 48 (sBA: week18/48: p