Given that BioMarin Pharmaceuticals’s Kuvan is potentially the only treatment for the rare genetic metabolic disorder phenylketonuria, Datamonitor believes that Wall Street may be underestimating Kuvan’s market potential. Taking into account the high levels of unmet need in this disease, combined with promising late-stage data, Datamonitor predicts US sales of $403 million in 2011 for Kuvan.
Phenylketonuria (PKU) is a rare genetic metabolic disorder that affects approximately one in 15,000 births in the US. PKU is characterized by the body’s inability to metabolize phenylalanine (Phe) to tyrosine. Presently, the only treatment for elevated Phe levels is life-long diet restriction and/or low-Phe medical formula. Adherence to a restricted diet after infancy is particularly challenging, creating the demand for a therapeutic drug that would grant patients dietary freedom.
In the pivotal Phase III trial, Kuvan showed that it provided a clinically significant decrease in blood Phe levels compared to placebo, while the extension trial produced positive data regarding dose responsiveness and tolerability. Additionally, the diet study confirmed that Kuvan could be used in combination with diet to increase Phe intake. Kuvan is expected to receive a timely regulatory approval without any setbacks in Q4 2007(PDUFA date set for November 25th 2007), and Datamonitor believes that Kuvan will be indicated for patients aged four and up, which would exclude newborns.
As Kuvan will be the only medical treatment for PKU, health plans have indicated that they will have to bear the cost of Kuvan therapy regardless of how high that cost is. Given that a yearly therapy for Schircks Laboratories’s tetrahydrobiopterin (BH4), if commercially available, would be approximately $33,000, and assuming a 35% price premium for Kuvan compared to tetrahydrobiopterin, the price point for Kuvan could be $20,000 higher than the Wall Street consensus. Indeed, the price could still be higher than this estimate.
Based on interviewed physicians’ excitement surrounding Kuvan, Datamonitor expects the drug to achieve rapid adoption upon FDA approval. Following an anticipated late Q4 2007 launch, US sales are forecast at $7 million in 2007, rising to $180 million in 2008 and $403 million in 2011. This forecast does not include patients aged 0-3 years, but note that this patient group could account for an additional $13 million to $24 million of sales at peak, depending on the final price point.
For more information on this and similar research, please contact Datamonitor at +1 212 686 7400 or visit www.datamonitor.com/healthcare.
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