Merck KGaA-BioMarin's Kuvan may Undergo Label Update - Analyst Blog
Updates on the regulatory front are eagerly awaited by investors in the pharma/biotech sector as they impact the share price of the concerned company. Regulatory updates include events like filing of marketing applications for a candidate or applications for updating product information, acceptance of these applications for review by the regulatory agencies, reviews by advisory panels and finally, a response from the regulatory agency regarding the approval status.
Late last week, Merck KGaA’s MKGAF biopharmaceutical business, Merck Serono, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency granted a positive opinion on an update to the product information for Kuvan.
Kuvan is already approved in Europe for the treatment of hyperphenylalaninemia occurring either due to phenylketonuria (PKU) in patients aged 4 years and above (who are responsive to Kuvan) or tetrahydrobiopterin (BH4) deficiency in patients of all ages.
Based on the results of the phase IIIb SPARK study on Kuvan, the CHMP recommended that Kuvan can be used for the treatment of PKU patients below 4 years of age, who are responsive to such a therapy.
We note that the European Commission (EC) generally takes the CHMP’s opinion into account while making a decision regarding the approval of a candidate. A positive opinion from the CHMP increases the odds of receiving a nod from the EC for updating Kuvan’s product information. Upon approval, Kuvan’s Summary of Product Characteristics (SmPC) will be updated to include details on extended use of the drug.
According to Merck KGaA, PKU affects approximately 1 out of 10,000 newborns in Europe. Given that no therapy is yet approved in Europe for the treatment of this disease in children less than 4 years of age, a potential label update will increase Kuvan’s eligible patient population.
We note that in 2014, Kuvan generated net revenue of $203 million, up 21% year over year. It is marketed by Merck KGaA outside the U.S., Canada and Japan, while BioMarin Pharmaceutical Inc. BMRN commercializes the drug in the U.S. and Canada (under the trade name Biopten). Kuvan is sold by Asubio Pharma in Japan. The drug enjoys orphan drug status in the U.S. and Europe.
Investors looking for well-ranked stocks in the health care sector may consider OncoGenex Pharmaceuticals, Inc. OGXI and Valeant Pharmaceuticals International, Inc. VRX. Both carry a Zacks Rank #1 (Strong Buy).
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