Positive Kuvan Pivotal Phase III Trial Results Published

BioMarin Pharmaceutical announced that final results from the Kuvan Phase III trial were published in the Aug. 11 issue of The Lancet.

The study suggests that treatment with Kuvan (sapropterin dihydrochloride) results in reductions in blood phenylalanine (Phe) levels in some phenylketonuria (PKU) patients, BioMarin said.

PKU is a genetic disorder that can result in brain damage, mental illness, seizures, tremors and limited cognitive ability.

Kuvan, an investigational oral small molecule for the treatment of PKU, is being developed in partnership with Merck Serono, a division of Merck KGaA, the company added.

According to the company, the Phase III clinical study enrolled 89 patients, 8 years and older with elevated blood Phe levels at 29 sites in the U.S., Europe and Canada. Patients were evaluated every two weeks for changes in blood Phe levels and adverse events.

After six weeks of treatment, 54 percent of patients treated with Kuvan and 23 percent of patients in the placebo group had a blood Phe concentration below the recommended level, the company said.