The positive results of BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) and Serono S.A. (NYSE: SRA, VTX: SEO))’s clinical studies of Phenoptin™ (sapropterin dihydrochloride), an investigational oral small-molecule therapeutic for the treatment of phenylketonuria (PKU), will be presented at the 56th Annual Meeting of the American Society of Human Genetics (ASHG) being held in New Orleans, Louisiana, October 9 to 13, 2006.
Results of the clinical trials are summarized below.
Phase 3 Clinical Study of Phenoptin™ for PKU:
Results from the Phase 3, double-blind, placebo-controlled clinical study of Phenoptin (commonly referred to as 6R-BH4, or BH4) in patients with elevated blood phenylalanine (Phe) levels demonstrated a statistically significant reduction at six weeks in blood Phe levels (p<0.0001) in patients receiving 10 mg/kg/day of Phenoptin, compared with those receiving placebo.Phase 2 Screening Study of Phenoptin™ for PKU:
Results from the Phase 2 screening study conducted to identify appropriate patients for inclusion in the Phase 3, double-blind, placebo-controlled study of PKU, demonstrate that Phenoptin was well tolerated and rapidly reduced blood Phe levels by varying degrees in PKU patients across the complete spectrum of PKU phenotypes.
In both trials, Phenoptin™ was well tolerated with mild adverse effects similar to experienced by patients in the placebo group.
Phenoptin™, an investigational oral small molecule therapeutic for the treatment of PKU, contains the active ingredient sapropterin dihydrochloride, the synthetic form of 6R-BH4 (tetrahydrobiopterin), a naturally occurring enzyme cofactor that works in conjunction with phenylalanine hydroxylase (PAH) to metabolize Phe.
Phenoptin™ has received orphan drug designation for the treatment of PKU from both the USFDA and EMEA. If and when Phenoptin™ becomes the first drug therapy approved for the treatment of PKU, it would receive seven years of market exclusivity in the United States and 10 years in the European Union for this indication.
Phenoptin™ has also been granted Fast Track Designation by the USFDA.
Read more details from the full press release.
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