Merck KGaA says that Kuvan, a treatment for a rare brain disorder licensed from BioMarin Pharmaceutical has been recommended for approval in Europe.

The German firm’s Merck Serono unit has received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP), for Kuvan (sapropterin) as an oral treatment for hyperphenylalaninemia (HPA) caused by phenylketonuria (PKU) or tetrahydrobiopterin deficiency.

Merck Serono and BioMarin estimate that Kuvan could be a potential treatment option for 30%-50% of the estimated 50,000 identified PKU patients in the developed world. HPA is characterised by increased amounts of the amino acid phenylalanine in the bloodstream which is toxic to nerve cells and, if left untreated, can result in brain damage.

Kuvan has orphan drug designation in the USA, where it was approved in December last year and is being sold by BioMarin. It has similar status in Europe, and if Merck Serono gets the thumbs-up, the drug will enjoy 10 years of market exclusivity for this indication.

The European Commission will deliver its final decision on the granting of marketing authorisation within 67 days and approval will trigger a $30 million milestone payment to BioMarin.

Historic thumbs-up for Cancidas
The CHMP has also recommended Merck & Co's Cancidas (caspofungin) for use in children with severe fungal infections, It represents the first recommendation in Europe for the use of a centrally-authorised medicine in children on the basis of clinical trial data generated in accordance with a paediatric investigation plan (PIP).