BioMarin rated 'Outperform' on Kuvan phase III data

Robert W Baird analysts have reiterated their "Outperform" rating on US-based BioMarin Pharmaceutical and set the target price USD 27.

In a research note published on 14 August, the analysts mention that the full publication of Kuvan's (sapropterin) phase III study may result in the uptake and increased usage in older phenylketonuria (PKU) patients.

PKU is an inherited metabolic disease caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). Insufficient quantities or reduced activity of PAH results in elevated levels of phenylalanine (Phe) in the blood. Sustained elevated blood Phe levels can result in serious neurological damage.

The US Kuvan revenue estimate for 2008 is set at USD 67m. According to the published data, the drug could be used for lifelong treatment, which may lend upside for the use of Kuvan as patients age, added analysts.

The final results from the Kuvan pivotal phase III clinical trial were published in the 11 August issue of The Lancet. The study suggests that treatment with the drug results in significant reductions in blood Phe levels in some PKU patients. Kuvan is being developed in partnership with Merck Serono, a division of German pharmaceutical company, Merck KGaA.

Jean-Jacques Bienaime, CEO of BioMarin, said in a Q2 FY07 conference call: "Our Kuvan programme is progressing on track, with three important milestones achieved recently: submission of the NDA filing, initiation of the expanded access programme, and receipt of priority review status.

The expanded access programme is actively enrolling patients, and we are hopeful that Kuvan will receive FDA approval by the end of November 2007."