Maralixibat on path to new drug application for Alagille syndrome-pruritus
Mirum Pharmaceuticals completed a clinical pre-new drug application meeting with the FDA for maralixibat, the company’s therapeutic candidate for the treatment of Alagille syndrome-associated pruritus, according to a press release.
“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,” Chris Peetz, president and CEO of Mirum, said in the release. “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.”
The company is planning a rolling NDA submission, which will be initiated in the third quarter of 2020, and plans to pursue full approval of the pruritus indication based on FDA feedback and the results from the ICONIC study.
The study comprised 31 children with Alagille syndrome-associated pruritus who received either maralixibat or placebo for 18 weeks. After a randomized withdrawal period, all treated received treatment for the remainder of the study’s 48-week period.
Treatment led to clinically relevant reductions in serum bile acid levels at week 18 (P < .001) and week 48 (P < .01), and pruritus at week 18 and week 48 (P < .0001) compared with baseline. These effects continued through 191 weeks of treatment.
Additionally, clinician scratch scores improved (P < .0001) and xanthomas continued to diminish (P < .05) during long term follow-up.
“The potential that maralixibat shows is incredibly exciting,” investigators Emmanuel Gonzales MD, PhD, and Emmanuel Jacquemin, MD, PhD, from the Hôpital Bicêtre in Paris, France, jointly stated. “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.”