BioMarin Pharmaceutical Inc.'s (BMRN) Phenylketonuria Candidate Drug Impresses in Phase 3 Trials

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the best beginner stocks to buy, according to analysts. On September 6, the company delivered positive pivotal Phase 3 data for PALYNZIQ (pegvaliase-pqpz) in Adolescents with Phenylketonuria.

Clinical data showed that the candidate drug successfully triggered a 49.7% decrease in mean blood phenylalanine (Phe) levels in adolescents aged 12 to 17 with phenylketonuria (PKU) treated with PALYNZIQ. The study evaluating the efficacy and safety demonstrated statistically significant blood lowering compared to diet alone. PALYNZIQ is the first and only enzyme substitution therapy approved to treat adults with PKU

According to Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin, the study confirms that PALYNZIQ can help adolescents experience relief from the burden of their conditions.

“BioMarin has been deeply committed to advancing scientific progress for people living with PKU over the past two decades, and we look forward to sharing these results with global regulators with the aim of bringing the unequaled efficacy observed with PALYNZIQ to an even younger group of people,” said Freiberg.

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is a global biotechnology company that develops and commercializes transformative medicines for people with severe and life-threatening genetic diseases, often rare conditions that have been largely ignored.

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Disclosure: None. This article is originally published at Insider Monkey.