FDA releases pediatric action plan, but voucher guidance still to come

FDA released a BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) became the first company to receive a voucher under the program after FDA approved its Vimizim elosulfase alfa to treat mucopolysaccharidosis IVA (MPS-IVA, Morquio's syndrome). Nancy Goodman, founder and executive director of the not-for-profit Kids v Cancer, said she expects interest in the incentive to accelerate after FDA releases a draft guidance on the program (see BioCentury Extra, Feb. 14). ...