Mirum Pharmaceuticals Inc (NASDAQ: MIRM) was one of the top gainers in Monday’s trading session, as the stock closed the day with gains of 110.99%. This came after the company announced that, it had a successfully pre-NDA meeting with the FDA for Maralixibat, a treatment for pruritus, which is associated with Alagille Syndrome.
The company also had a meeting with the FDA with regards to chemistry, manufacturing, and controls (CMC). It further announced that, the primary role of this meeting was to discuss the clinical and non-clinical requirements for the company’s proposed NDA submission.
On top of that, the company announced that, rare pediatric disease designation had been given for Maralixibat Alagille Syndrome. It added that, this designation qualifies it to receive a priority review if it is approved by the NDA. The company has also stated that, it plans to seek full approval for pruritus association with Alagille syndrome that is based on feedback from the NDA. It is planning to roll NDA submission, and will be started in Q3 of 2020, and full CMC submitted in Q1 of 2021.
Speaking on this development, the company CEO, Chris Peetz stated that, they were happy that the FDA had recommended that they change the classification of their requested Type C meeting, to a pre-NDA meeting. He added that, they had plans of rolling NDA submission starting in Q3 of 2020. The CEO further added that, they were looking forward to working closely with the FDA to give children suffering from Alagille Syndrome as quickly as possible.
Medical experts have also given a favorable opinion on this development. Emmanuel Gonzales, the professor of Pediatrics at Hopital Bicetre in Paris stated that, Maralixibat had shown incredible potential and that the data generated from the ICONIC study were encouraging for people suffering from severe diseases.
In yesterday’s session, the stock was trading between a low of $11.22 and a high of $22.43 before closing the day at $17.28. Volumes in the day stood at 8.45 million.
About Mirum Pharmaceuticals Inc
Mirum Pharmaceuticals Inc is a therapeutics company that develops therapies for rare pediatric conditions. It is headquartered in Foster City, California.