BioCentury
ARTICLE | Regulation

Wanted: More Orphan innovation

Ultragenyx's Kakkis lists innovations that could spur Orphan drug development

February 24, 2014 8:00 AM UTC

New economic incentives and regulatory policies could lead to a dramatic acceleration of drug development for rare conditions, according to Emil Kakkis, president and CEO of Ultragenyx Pharmaceutical Inc.

Kakkis, who led the development of three Orphan drugs as CMO at BioMarin Pharmaceutical Inc., is calling for Congress to consider granting additional market exclusivity to drug sponsors for developing approved drugs for rare conditions. He argues this not only will create new treatments, but provide a foundation for lower-cost rare disease treatments...