BRIEF—EMA grants approval for Mepsevii

Ultragenyx, a US biopharmaceutical company focused on developing novel therapies for ultra-rare diseases, announced on Monday that the European Commission had granted marketing authorization for Mepsevii (vestronidase alfa), a therapy for non-neurological manifestations of mucopolysaccharidosis VII (Sly syndrome).

"William Sly’s science for this treatment has been around for over 25 years and we are honored to be able to develop and finally make this medicine available to Sly syndrome patients and families in Europe, people who did not know whether a therapy would ever be available to them," said Emil Kakkis, chief executive of Ultragenyx. "This would not have been possible without the commitment and dedication of the patients, their families, and the physicians who participated in our clinical program, and I would like to thank them."

The approval follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in June, and orphan drug designation as far back as 2012. In the USA, Mepsevii has been available to both pediatric and adult patients since November 2017.

Ultragenyx shares have risen 0.6% since the announcement on Monday.