Mapreg compound gains orphan status

A lead compound used to develop treatments for central nervous system diseases has gained European Commission (EC) approval.

MAP4343 from biotech company Mapreg can be used to treat spinal cord injury and has been entered into the EC register of orphan drugs.

This decision follows a favourable opinion from the Orphan Drugs Committee of the European Medicines Evaluation Agency (EMEA). MAP4343 is patent-protected and has an identified and validated molecular target, namely Microtubule Associated Protein type 2 (MAP2). The product's main advantage is in its efficiency when taken by peripheral injection up to 24 hours after the trauma. The results of studies on animal models yielded so far are unmatched elsewhere. Regulatory preclinical studies will be completed shortly (having shown no significant signs of toxicity so far) and a Phase I clinical trial could be initiated towards the end of 2008.

"The granting of orphan drug status in the European Union is an important step for Mapreg and for the future benefit of patients having suffered a spinal cord injury," said Etienne-Emile Baulieu, president of Mapreg. "I am particularly pleased about this recognition at the European level, which will enable us to exploit neurosteroid derivatives discovered in my laboratory. The results on animal models are without parallel and we want to move forward to initiate the first clinical trials."

The orphan drug status granted by the EMEA facilitates the development of products designed to treat rare diseases, which would not otherwise be profitable enough because of the small number of patients involved. The official definition of an orphan drug in the European Union is a product designed to treat a disease that affects less than five people in 10,000. Orphan status gives a product 10 years' exclusivity in a particular therapeutic indication in the European market, including France, Germany, Italy, Spain and the UK. Orphan drug status also enables a company to obtain scientific assistance and advice from the EMEA for demonstrating the product's quality, safety and efficacy during the entire development cycle. It also ensures the company easy access to the EU's Mutual Recognition Procedure, grants it total or partial exemption from fees associated with the regulatory approval of the product, and gives it the possibility of obtaining funding under the European Union's program for supporting research into rare diseases.

There are more than 20,000 new cases of spinal cord injury every year in the western world, and no drug exists at present to treat them. The only therapeutic option is surgery, involving one or more operations. The consequences of such traumas are paraplegia or tetraplegia, and the total cost of caring for a patient is estimated at between Euro 1m and €2m.