Vimizim US approval

February 18, 2014

The US Food and Drug Administration has approved Vimizim (elosulfase alfa) which is the first FDA-approved treatment for Morquio A syndrome - also known as Mucopolysaccharidosis Type IVA. Morquio A syndrome is described by the FDA as “a rare, autosomal recessive lysosomal storage disease caused by a deficiency in N-acetylgalactosamine-6-sulfatase (GALNS)”. There are about 800 patients with Morquio A syndrome in the USA, with the condition leading to problems with bone development, growth and mobility. Vimizim was granted priority review, with the move underscoring the FDA’s “commitment to making treatments available to patients with rare diseases,” according to a spokesperson for the agency.

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Pharmacy Daily | Vimizim US approval

Vimizim US approval

Other recent headlines

PD breaking news – Top Western Australian talent honoured

Legal action for Montu

Call to unblock wait times

SEARCH PHARMACY DAILY

Featured video

Recent issues

INDUSTRY JOB VACANCIES