Brussels, 29/01/2004 (Agence Europe) - Four years after it came into force, a positive assessment has been made of the orphan medicines regulation.
Under this regulation, designed to encourage research into rare diseases affecting fewer than 5 in every 10000 people in the EU, laboratories can receive aid and a reduction in fees for the centralised Authorisation to Market in the EU procedure (AM). Orphan medicines are given 10 years exclusive rights from the date of award of the AM. Over the period April 2000 to December 2003, the Orphan Medicines Committee at the European Medicines Agency received 322 applications and issued 187 positive opinions and 5 negative. 174 products have been given the status of orphan medicines by the European Commission. Of fourteen orphan medicines authorised to be marketed in Europe since April 2000, twelve are for treating metabolic diseases, the latest three being cancer treatments. The first two (AM granted in August 2001) are treatments for Fabry's disease, a form of breakdown of the neurons due to an abnormal concentration of lipids in the cells of the nervous system. Another neurolipidosis, hereditary and congenital, is Gaucher's malady, for which AM was granted for a new treatment in November 2002. The same medicine is indicated in the treatment of two diseases, namely chronic myeloid leukaemia and digestive tumour. The other ten orphan medicines (with year of AM in brackets) are. (2002) acute promyelocytic leukaemia; (2002) pulmonary arterial hypertension; (2002) non-congenital hypertrophy of the extremities and the head); (2003) deficiency of N-acetylglutamate synthethase (enzyme protecting the liver from ammonia); (2003) Hurler's disease (congenital disease causing malformation of the skeleton and delayed mental development, leading to death by the age of 15); haematopoiethical progenitor transplants (2003); primary and secondary pulmonary hypertension (2003); a type of polyposis (2003); - non-Hodgkins lymphoma; high level dysplasia connected with Barett's oesophagus.