CHMP Backs Elosulfase Alfa (Vimizim) for Rare Morquio A Syndrome

Megan Brooks


February 21, 2014

The European Medicine Agency's (EMA's) Committee for Medicinal Products for Human Use (CHMP) has recommended approval of elosulfase alfa (Vimizim, BioMarin Europe Ltd) for treatment of mucopolysaccharidosis type IVA (Morquio A syndrome) in patients of all ages.

Morquio A syndrome is a rare, autosomal recessive lysosomal storage disease caused by a deficiency in N-acetylgalactosamine-6-sulfate sulfatase.

Elosulfase alfa (1 mg/mL concentrate for intravenous infusion) replaces the missing N-acetylgalactosamine-6-sulfate sulfatase enzyme involved in metabolism. Absence of this enzyme leads to problems with bone development, growth, and mobility. The drug received orphan status in Europe in July 2009.

The US Food and Drug Administration (FDA) approved elosulfase alfa for Morquio A syndrome on February 14, as reported by Medscape Medical News.

Based on the quality, safety, and efficacy data reviewed, the drug has a favorable benefit-to-risk balance, the CHMP said in a statement.

The benefits with elosulfase alfa are its "ability to improve patient's 6 minute walking test, and to improve other symptoms of Morquio disease, such as respiratory or anthropometric functions," the committee said.

At this time, there are no "satisfactory" means to treat mucopolysaccharidosis type IVA in Europe, the CHMP noted.

The most common adverse effects of the drug are infusion reactions, including anaphylaxis, hypersensitivity, and vomiting. In the United States, elosulfase alfa carries a boxed warning on the risk of anaphylaxis.

The CHMP said use of the drug should be supervised by a physician experienced in the management of patients with mucopolysaccharidosis type IVA or other inherited metabolic diseases. "Administration of Vimizim should be carried out by an appropriately trained healthcare professional with the ability to manage medical emergencies," the committee advised.

A pharmacovigilance plan for the drug will be implemented as part of the marketing authorization.

The European public assessment report will be revised to include the updated summary of product characteristics if the European Commission grants marketing authorization for this indication.

The European Commission generally follows the recommendations of the CHMP and usually delivers its final decision within 3 months of the CHMP recommendation.


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