BioMarin Pharmaceutical, Inc.

First patient enrolled into phase II trial of Phenoptin for PKU

Published in Medical Device Law Weekly, January 30th, 2005

BioMarin Pharmaceutical, Inc., (BMRN) announced that it has initiated its phase II clinical trial of Phenoptin (sapropterin hydrochloride), an investigational oral, small molecule therapeutic for the treatment of the genetic disease phenylketonuria (PKU).

Patients who respond to Phenoptin in the phase II clinical trial, as measured by a greater than 30% reduction in blood phenylalanine (Phe) levels following 8 days of treatment, will be eligible to enroll into the phase III trial expected to begin in the first quarter of 2005.

Margretta Seashore, MD, director of Genetic Consultation Service in the Department of Genetics at Yale University School of...

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Source: Medical Device Law Weekly (2005-01-30)

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