Biomarin Pharmaceutical Inc. (Nasdaq: BMRN)’s preclinical studies of Phenylase™ (phenylalalanine ammonia lyase) suggests a successful accomplishment in addressing key technical challenges associated with the company’s research development for the treatment of phenylketonuria (PKU).
Affecting about 50,000 diagnosed patients in the developed world, PKU is a genetic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH) that is required for the metabolism of phenylalanine (Phe), an essential amino acid found in most protein-containing foods.
Insufficiency of the active enzyme leads to Phe accumulation to abnormally high levels in the blood and brain, thereby resulting to a variety of complications including severe mental retardation and brain damage, mental illness, seizures and tremors, and cognitive problems.
Biomarin’s Phenylase™, an investigational enzyme substitution therapy, contains PEGylated phenylalanine ammonia lyase as an active ingredient designed to catabolize the Phe that builds-up in the body due to lack of activity of the enzyme phenylalanine hydroxylase.
Phenylase™ is being developed as a once-weekly, self-administered subcutaneous injection.
BioMarin is planning to conduct additional preclinical studies of Phenylase, with the intention of filing an Investigational New Drug application (
Together with Phenoptin™ (sapropterin dihydrochloride), a small-molecule oral therapeutic currently in Phase 3 clinical development, Phenylase™ is being evaluated by BioMarin for the treatment of PKU in partnership with Serono S.A. (NYSE:SRA, VTX:SEO) as part of a strategic alliance established in May 2005.
Find more details from the Biomarin Pharmaceutical Inc. press release.
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