Mirum Pharmaceuticals has secured the breakthrough therapy designation for maralixibat from the US Food and Drug Administration (FDA) for the treatment of pruritus associated with Alagille syndrome (ALGS) in patients, aged 1 year and older.

The California-based biopharma company is engaged in the development and commercialization of new therapies for debilitating liver diseases.

Alagille syndrome is considered to be a rare genetic disorder in which bile ducts are unusually narrow, malformed and lowered in number, which results in bile accumulation in the liver and eventually progressive liver disease.  One in every 30,000 to 50,000 births in the US and Europe is estimated to be affected by the Alagille syndrome.

Maralixibat is a minimally-absorbed, orally administered investigational drug, which is being studied in various rare cholestatic liver diseases for pediatric populations. The drug blocks the apical sodium dependent bile acid transporter, which leads to more bile acids being excreted in the feces, thereby resulting in lower levels of bile acids systemically, and possibly lowering bile acid mediated liver damage and associated effects and complications.

Maralixibat’s breakthrough therapy designation was given based on data from the phase 2b ICONIC clinical trial in children with Alagille syndrome. The findings from the clinical trial were presented recently at the International Liver Congress (EASL).

The breakthrough therapy designation is given by the FDA to investigational drugs intended for the treatment of a serious or life-threatening condition for which preliminary clinical evidence may show significant improvement on at least one clinically significant endpoint over existing therapy.

The drug designation will help in expediting development and review, while conveying all of the fast track designation program features, such as more intensive FDA interaction and guidance.

In November 2018, Mirum Pharmaceuticals, which is a clinical stage pharma company in San Diego, raised $120 million through a Series A funding round to support the development of maralixibat for the treatment of rare cholestatic liver diseases.

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  California, Cholestatic liver diseases, liver diseases, Maralixibat, Mirum Pharmaceuticals, US Food and Drug Administration, USA