The procedure, which has been implemented only 25 times worldwide, consists on transfering cells from another liver, without the necessity of transplanting the whole organ
Press release -18/12/2008
- It is the first drug approved in Europe for the treatment of hyperphenylalaninemia in patients with phenylketonuria or deficit of BH4
- PKU is a rare disease which can cause serious brain damage in newborns and children, and neurocognitive disability in teenager and adult patients
Merck Serono, the biotech division of the German chemical pharmaceutical company Merck, has announced that the European Commission has authorized the marketing of Kuvan ® (sapropterine octenidine) as a hyperphenylalaninemia (HPA) oral treatment for patients with phenylketonuria (PKU) or deficit of tetrahydrobiopterin (BH4). Kuvan ® had previously received the designation of Orphan Drug by the EMEA as the first medication indicated for the treatment of the hyperphenylalaninemia.
“Up to today, there were no drugs approved in Europe for the treatment of PKU, a disease that can cause serious brain damage in newborns and children, and neurocognitive disability in teenager and adult patients if they do not follow a strict diet over a lifetime,” said José Cabrera, Medical Director of Merck in Spain. “From Merck, and specifically from our biotechnology division Merck Serono, we are very pleased with the approval of the European Commission.” The availability of Kuvan ® across Europe will allow patients that have this chronic disease to improve the control of their phenylalanine blood levels, while improving their quality of life and, ultimately, reducing the risk of mental damage “, Cabrera added.”
The marketing authorisation is based on the data provided by two international clinical trials in phase III, double-blind, randomized and controlled with placebo in patients with HPA/PKU. The data shows that treatment with Kuvan ® reduces phenylalanine blood levels and increases phenylalanine tolerance in the diet, which reduces the need to limit your intake
The European Commission has granted the authorization of sale for the 27 countries of the European Union, as well as Iceland, Liechtenstein and Norway. Having received the designation of orphan drug, Kuvan ® will receive 10 year marketing exclusivity in the European Union for this therapeutic indication. Kuvan ® in Europe launch is scheduled for the first half of 2009.
Incidence of the disease
Currently there are approximately 35,000 patients in the EU diagnosed with phenylketonuria or hyperphenylalaninemia by BH4 deficit.
PKU and BH4 deficit are known as orphan diseases due to their low frequency in the population. They are diseases caused by genetic defects in the metabolism of the phenylalanine amino acid, which result as a hyperphenylalaninemia, i.e. abnormally high levels of phenylalanine in blood.
In order to avoid the consequences of PKU, It is essential an early diagnosis, reason why there are ongoing state metabolic screening programmes. In Spain this screening is done between the first 48 hours and 10 days of life, through a small blood sample in the heel of the infant, enabling to detect inborn metabolic disorders, among them, hypothyroidism and phenylketonuria.
Up until today, no drugs were approved for the treatment of this disease in Europe. The only alternative treatment for patients with PKU in order to control the disease is a diet restricting phenylalanine (foods containing high levels of this essential amino acid are meat, fish, nuts, dairy products and some vegetables and fruits), associated with a daily supplement of amino acids. Not following this diet can lead to a progressive mental and behavioral deterioration.
About Merck and the Merck Serono Division
Merck, the German multinational with the most pharmaceutical and chemical tradition of the world, is today a company with sales of 7.1 billion Euros in 2007, whose history began in the year 1668 and a with future forging about 32,500 employees in 59 countries. Its success is based on innovations created by contributors with an entrepreneurial spirit. Its activity focuses on three business divisions: Merck Serono (specialized in the biotechnology sector), Merck Consumer Health Care (medicines for health self-care) and Merck Chemicals (leader in liquid crystals and in pigments and other reagents and solutions for life science)
Merck brings together its activities under Merck KGaA, whose shares belong to the Merck family, 70% approximately. The remaining 30% is publicly traded
Merck Serono is the Division of Merck, created on January 5, 2007 after the purchase of the Serono biotechnology company. This new Division employs about 14,500 people worldwide and is present in the areas of Oncology, Neurology, fertility, Cardiometabolism and treatment of other diseases, including psoriasis and lack of growth hormone.
This piece of information is exclusively addressed to journalists specialized in health. The content is of a scientific nature and its purpose is not disclosing it to the general public. It may contain some early information about the business of the company concerning the future and whose results are subject to possible variations.