BioMarin Pharmaceutical, Inc. (BMRN)

The following is the abstract from an article titled, "Is sapropterin treatment suitable for all subjects with phenylketonuria," published recently in the journal Expert Opinion in Pharmacotherapy.

In a Phase I study, 20% of subjects with phenylketonuria (96/485) responded with a > or = 30% reduction in blood phenylalanine at day 8 of treatment with sapropterin. Subsequently, some of the responders from this study were enrolled in a Phase III study. The subjects with phenylketonuria were randomised to sapropterin 10 mg/kg/day (n = 41) or placebo (n = 47) for 6 weeks and continued their usual diet. The primary end point was the mean change from baseline in phenylalanine level after 6 weeks and there was a reduction of 235 micromol/l in the sapropterin group compared with 2.9 micromol/l in the placebo group. Thus, in the 20% of subjects with phenylketonuria that responded to a trial treatment with sapropterin, there were reduced mean blood phenylalanine levels over 6 weeks of treatment. In summary, sapropterin effectively reduces blood phenylalanine levels in a small proportion of subjects with phenylketonuria.

This is NOT good news for BioMarin. (Kuvan is the brand name for sapropterin). Although investors seem to believe that Kuvan can generate significant revenue, the medical community disagrees. In order for the drug to be paid for by insurance, documentation of response will be necessary. With only 20% of subjects being able to show objective evidence of beneficial response, it will be very difficult to get authorization for insurance reimbursement for this drug. This must be very disappointing for BioMarin.

It is interesting to see that insiders continue to sell shares at an alarming rate. I would guess that for those at the company it is fairly obvious that sales of Kuvan will not even come close to the analysts' predictions.