BioMarin Pharmaceutical has entered into an agreement to purchase Repligen’s histone deacetylase inhibitor (HDACi) library and related intellectual property. Potential applications of the HDACi portfolio include Friedreich's ataxia (FA) and other neurological disorders. BioMarin will provide Repligen with an upfront payment of $2 million and potential future milestone and royalty payments.
"This agreement represents a natural extension of BioMarin's business strategy of acquiring promising preclinical programs that we can successfully develop and commercialize for people with serious, unmet medical needs," said Jean-Jacques Bienaime, CEO of BioMarin.
FA is a progressive neurological disorder that affects approximately 20,000 people in the U.S. and Europe, typically resulting in wheelchair dependence in young adulthood and early death due to cardiac failure. It is caused by mutations in the FXN gene and is inherited in an autosomal recessive manner. FXN mutations result in reduced expression of frataxin protein, manifesting in progressive neurological and cardiac damage. Major neurological symptoms include muscle weakness and ataxia, a loss of balance and coordination. These symptoms typically appear between 10 and 15 years of age, but FA has been diagnosed in people from ages 2 to 50, with earlier onset associated with a more severe course.