Rare Daily Staff

Mirum Pharmaceuticals said it has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher for $110 million to an undisclosed company.

The PRV was granted by the U.S. Food and Drug Administration in September 2021 with the approval of Livmarli (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.

Alagille syndrome (ALGS) is a rare genetic disorder in which bile ducts are abnormally narrow, malformed and reduced in number, which leads to bile accumulation in the liver and ultimately progressive liver disease. In patients with ALGS, multiple organ systems may be affected by the mutation, including the liver, heart, kidneys and central nervous system. The accumulation of bile acids prevents the liver from working properly to eliminate waste from the bloodstream and almost three quarters of patients with ALGS have a liver transplant before reaching adulthood. The pruritus experienced by patients with ALGS is among the most severe in any chronic liver disease and is present in most affected children by the third year of life.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, which includes access to the FDA’s expedited review and approval process and makes the developing company eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA. The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are also potentially lucrative because they are transferable. Most recently, Albireo sold a priority review voucher for $105 million.

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