Ultragenyx Announces Approval of Mepsevii in Brazil for the Treatment of Mucopolysaccharidosis VII

Ultragenyx Pharmaceutical (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Brazil’s National Health Surveillance Agency (ANVISA) has approved Mepsevii (vestronidase alfa) for the treatment of Mucopolysaccharidosis VII (MPS VII; Sly syndrome) for patients of all ages.

As quoted in the press release:

“The approval of Mepsevii in Brazil is an important milestone, particularly for children with this progressive and debilitating disorder, and also for Ultragenyx because it marks the first regulatory clearance for this important medicine outside of the U.S. and Europe,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “Occurring less than one year after Mepsevii was approved in the U.S., this approval validates our strategic plan to rapidly expand into other regions of the world, including Latin America.”

Mepsevii was approved by the U.S. Food and Drug Administration (FDA) in November 2017 for the treatment of pediatric and adult patients with MPS VII. In August 2018, the European Commission (EC) approved the Marketing Authorization Application (MAA) for Mepsevii under exceptional circumstances for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII. With this decision, Mepsevii is approved in all 28 EU countries, Iceland, Liechtenstein and Norway.

Click here to read the full press release.