Vimizim elosulfase alfa regulatory update

FDA's Endocrinologic and Metabolic Drugs Advisory Committee recommended approval of Vimizim elosulfase alfa from BioMarin to treat mucopolysaccharidosis IVA (MPS-IVA, Morquio's syndrome). The panel voted 18-1 in favor of approving Vimizim for all patients, with 2 votes for a third option of limiting the approval to a subgroup of MPS-IVA patients with worse baseline walking abilities. One of the members who voted for approval in the subgroup subsequently changed her vote in favor of full approval.

The panel also voted 16-5 in favor of Vimizim's safety profile for all patients. On efficacy, the committee voted 12-1 in favor of Vimizim being effective for all patients, while 8 members voted in favor of it being effective only for the subgroup with worse baseline walking abilities. One of the members who voted for efficacy in the subgroup subsequently changed their vote in favor of efficacy for all patients. ...