SAN RAFAEL, Calif. (AP) — BioMarin Pharmaceutical Inc. said Tuesday said it will proceed with further studies of an experimental treatment for the genetic disorder Pompe disease.
The company said patients treated with its drug, which is called BMN-701, were able to walk greater distances in a timed trial, and were able to inhale and exhale with greater force. BioMarin said it will advance the drug into a later stage of testing.
Patients with Pompe disease lack an enzyme in their blood that breaks down glycogen, a stored form of sugar used for energy. The glycogen collects in the muscles, heart, liver, and nervous system. That can cause muscle weakness and deadly respiratory problems. When patients have almost none of the enzyme, symptoms can appear in the first few months of life. If they have a partial deficiency, symptoms can appear in childhood or as late as middle age.
By the end of the year, BioMarin wants to begin testing the drug in patients with late-onset Pompe disease who have previously been treated with alglucosidase alfa, which is sold by French drugmaker Sanofi under the name Myozyme and Lumizyme. BioMarin said the main focus of the study will be patients' ability to inhale. The trial will also evaluate their ability to walk and exhale.
Shares of BioMarin lost 72 cents to $60.76 Tuesday. They were unchanged in aftermarket trading.