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BioMarin’s Orphan Drug Clinical Strategy Is Rooted In Natural History

 
• By Bridget Silverman

BioMarin has emerged at the forefront of orphan drug development by grounding its clinical trials in natural history studies. The specialty firm’s recent investor day highlighted how clinical trials in ultra-orphan disorders are informed by efforts to quantify the clinical course of often poorly-understood conditions to determine appropriate endpoints and dosing strategies.

The lack of therapeutic options in many orphan diseases makes them an appealing prospect for specialty drug developers looking for the regulatory flexibility and market benefits that accrue to the first therapies for serious unmet medical needs, but BioMarin Pharmaceutical Inc.’s recent investor day highlighted the extra effort that orphan drug developers must put in to elucidate the natural history of often poorly-understood rare diseases in order to design clinical trials and justify clinical endpoints.

Orphan drug development is a hot field right now. Novel orphan drug approvals reached their highest count ever in 2014...

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