BioMarin to present data at Society for the Study of Inborn Errors of Metabolism

BioMarin Pharmaceutical Inc. on Monday announced they will present data in two oral and 19 poster presentations at the Society for the Study of Inborn Errors of Metabolism 2015 Annual Meeting being held today until Sept. 4 in Lyon, France.

Long-term efficacy and safety data from a Phase 2 extension study of pegvaliase in adults with phenylketonuria (PKU) will be presented in an oral platform session. PKU is a rare genetic condition where the body can’t metabolize the essential amino acid phenylalanine.

A separate oral platform presentation will provide an overview of the reveglucosidase alfa (BMN 701) clinical program in late onset Pompe disease.

Pompe disease is an inherited condition caused by the deficiency in the enzyme acid alpha-glucosidase, which leads to progressive weakening of muscles in the body, including diaphragm muscles essential for breathing.

"We are committed to patients with rare inherited metabolic disorders,” Hank Fuchs, executive vice president and chief medical officer at BioMarin, said. “We continue to evaluate multiple therapies across disease states over the long term to both understand the impact of our medicines on patients, as well as to better understand the biology of the disease. With promising results from our late stage and long-term clinical studies, we are one step closer to potentially bringing new treatments to patients in need. Our more than 20 presentations across multiple conditions underscores our growing research to advance the standard of care for children and adults with rare conditions."