Merck’s Kuvan set for European approval
pharmafile | September 26, 2008 | News story | Sales and Marketing | EU, Kuvan
Merck Serono's Kuvan is on track to become the first medicine approved in Europe for two rare neurological disorders.
Regulatory advisers at the CHMP recommended Kuvan (sapropterin) as a treatment for two forms of hyperphenylalaninemia (HPA), which is characterised by abnormally high levels of phenylalanine in the blood.
The inherited condition can lead to brain damage if left untreated and Merck Serono is seeking a license to treat HPA in patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency.
Roberto Gradnik, executive VP, commercial Europe at Merck Serono, said there is currently an unmet medical need for an effective treatment for the conditions.
"The availability of Kuvan throughout Europe will allow the patients suffering from these serious, debilitating diseases to better control their phenylalanine levels and at the same time improve their quality of life," he added.
Currently the only way for PKU patients to manage their disease is to follow a strict phenylalanine-restricted diet, which means cutting out all meat, eggs and diary products because of their high protein levels.
In phase III trials of patients with HPA due to PKU, Kuvan reduced blood phenylalanine levels and increased dietary phenylalanine tolerance and Merck Serono said it could reduce the need to limit phenylalanine intake in patients' diet.
HPA due to BH4 deficiency is by far the rarer of the two diseases, accounting for 1-2% of HPA cases, and often does not require protein to be limited in patients' diets.
HPA, due to PKU or BH4 deficiency, affects around 35,000 people in the EU and can cause serious brain damage in infants and children, and neurocognitive impairment in teenagers and adult patients.
Kuvan is seeking indications to treat HPA in both adults and children aged 4 and over with PKU who respond to such treatment and in adults and children with BH4 deficiency who respond to such treatment.
Merck Serono is developing Kuvan in partnership with California-based biopharmaceutical company BioMarin Pharmaceutical.
The German pharma company has exclusive rights to market Kuvan outside Japan, where Daiichi Sankyo has marketing rights, and North America, where BioMarin will market it.
But although Kuvan will provide a treatment option for PKU and BH4-deficient patients, once it is approved, it does not work for everyone.
Merck Serono said that published literature showed just 20-50% of PKU patients are responsive to treatment with Kuvan.
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