Mirum Pharmaceuticals Inc said on Wednesday the US Food and Drug Administration has approved its drug to treat itching in patients with Alagille syndrome, making it the first approved therapy for treating symptoms of the genetic disorder.
Mirum said the oral drug maralixibat, the exclusive rights to which it acquired from Shire in 2018, would be available for distribution immediately under brand name Livmarli.
“We expect it to be shipped out to first patients in a matter of weeks (from approval),” Chief Operating Officer Peter Radovich told Reuters in an interview prior to approval.
Alagille syndrome (ALGS) is a rare genetic disorder in which bile ducts are abnormally narrow, malformed and fewer in number, which lead to bile accumulation in the liver. This has a number of effects, the most prevalent is cholestatic pruritus or itching.
“(Itching) is really devastating for children and families dealing with this disease…and ultimately that itching is a driving factor for liver transplant decisions,” CEO Chris Peetz told Reuters.
Mirum executives said there are currently 2,000 to 2,500 children suffering from ALGS in the United States and they estimate the total market opportunity in the indication to be about $500 million.
A mid-stage trial of 31 pediatric patients showed the use of maralixibat drove a statistically significant reduction in severity of itching and small skin blemishes, as well bile acids that are the root cause of these symptoms.
Mirum has also applied for approval of the drug in the European Union for the same indication. (Reporting by Mrinalika Roy and Bhanvi Satija in Bengaluru; Editing by Shinjini Ganguli)