Biomarin’s drug wins Europe’s first approval for type of dwarfism


(Reuters) – U.S.-based Biomarin Pharmaceutical Inc’s (BMRN.O) treatment for one of the most common forms of dwarfism received clearance from the European Commission, becoming the first therapy to get an approval in the region for achondroplasia.

The approval comes months after the European Medicines Agency endorsed Voxzogo, also known as vosoritide. There are no approved drugs to specifically treat achondroplasia in most parts of the world. Growth hormones have been approved to treat the condition in Japan.

Biomarin’s Voxzogo is now approved in Europe for use in children aged two and above with growth plates still open, typically those under 18 years of age.

“This regulatory approval is based on improved height gain, one important determinant of day-to-day function for people with achondroplasia,” said Klaus Mohnike, professor of Paediatrics at Magdeburg University Hospital who conducted trials for the drug.

Biomarin said on Friday France had granted Voxzogo a temporary authorization for use allowing immediate access to the drug in the country.

The drug would cost roughly $300,000 per patient annually in France, excluding discounts, and Biomarin said it expects the list price in Germany to be consistent with that in France.

Analysts expect the drug to bring in sales of more than $750 million by 2027, according to Refinitiv data.

Achondroplasia, a form of disproportionate short stature caused by a mutation in a gene that regulates bone growth, occurs in about one in 25,000 newborns. Complications from the condition can even result in a spinal decompression surgery.


Reuters source: