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BioMarin to buy Repligen's pre-clinical compounds to treat Friedreich's Ataxia

PBR Staff Writer Published 22 January 2014

BioMarin Pharmaceutical has entered into an agreement to acquire Repligen's histone deacetylase inhibitor (HDACi) library and associated intellectual property.

According to the company, potential applications of the HDACi portfolio include Friedreich's ataxia and other neurological disorders.

As part of the deal, , BioMarin will pay an upfront payment of $2m to Repligen as well as potential future milestone and royalty payments for the development, regulatory approval and commercial sale of compounds.

BioMarin chief executive officer Jean-Jacques Bienaime said the deal represents a natural extension of the company's business strategy of acquiring promising pre-clinical programs that it can develop and commercialize for people with serious, unmet medical needs.

"Fredreich's ataxia is a neurological disorder that affects tens of thousands of young people in the developed world and we look forward to the opportunity to move a compound into the clinic in this indication," Bienaime said.

About 20,000 people in the US and Europe are affected by Friedreich's ataxia (FA), a progressive, neurological disorder, which results in wheelchair dependence in young adulthood and early death due to cardiac failure.

The disorder is caused by mutations in the FXN gene, and is inherited in an autosomal recessive manner.