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Committee for Orphan Medicinal Products (COMP) January 2012 highlights

London, England (January 16, 2012) – During its first meeting in 2012, the Committee for Orphan Medicinal Products (COMP) adopted a total of 16 recommendations for orphan designation, including for a number of substances intended for the treatment of diseases for which no orphan designation has ever been granted, and a substance intended for the treatment of a neglected tropical disease.

Orphan designation can be granted early in the development process to substances that are intended for diagnosis, prevention or treatment of life-threatening or very serious conditions that affect not more than 5 in 10,000 people in the European Union (EU) or that would not be developed without incentives.

The COMP recommended the granting of an orphan designation for carbetocin for the treatment of Prader-Willi syndrome. This is the first orphan designation opinion for the treatment of this rare genetic condition. Prader-Willi syndrome causes a wide range of symptoms, such as a constant desire to eat food, which seems to be driven by permanent hunger and often leads to dangerous obesity, impaired function of the gonads, learning difficulties and behavioural problems. This is a seriously debilitating disease and patients often have a significantly reduced life span and require lifelong care.

At present there is no treatment approved for this rare condition, which is estimated to affect less than 2 in 10,000 people in the EU. Orphan designation gives the sponsor of carbetocin access to incentives that encourage and support the development of this medicine for the treatment of Prader-Willi syndrome.

The assessment of the designation application for carbetocin was supported by the expert advice from patient representatives from the European Organisation for Rare Diseases ( http://www.eurordis.org/) (Eurordis), who confirmed the high unmet medical need in the management of patients with Prader-Willi syndrome.

Another first-time orphan designation recommended by the COMP at its January 2012 meeting is for sialic acid in the treatment of hereditary inclusion body myopathy (HIBM). This is a very rare, debilitating neuromuscular disease with devastating consequences for patients, leading to many years of severe disability and near quadriplegia in muscle function. It is estimated that 4 in 10,000,000 people in Europe are affected by the condition.

The COMP adopted a recommendation for orphan designation for oleylphosphocholine for the treatment of leishmaniasis. The disease is caused by parasites that are transmitted through the bite of the sandfly.

The World Health Organization describes leishmaniasis as a poverty-related disease, which is endemic in many developing countries. The visceral form of leishmaniasis, the most serious form of the disease, is estimated to cause as many as 50,000 deaths per year. Nevertheless, in the EU the medicine is recommended for orphan designation because of the small number of patients affected in this region (less than 5 in 10,000). The framework for orphan medicines can also be used to support the fight against neglected diseases by providing incentives to encourage the development of medicines for these diseases that would not be developed under normal market conditions.

For the full list of opinions adopted by the COMP during its January 2012 meeting please see the COMP monthly report (http://www.ema.europa.eu/ema/pages/includes/document/open_document.jsp?webContentId=WC500120411).

Next steps

The COMP recommendations for these substances are sent to the European Commission for the adoption of a decision. Once an orphan designation has been granted, sponsors have access to certain incentives (http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000393.jsp&mid=WC0b01ac0580024c5a) during the development of the medicine.

For each medicine that has been granted an orphan designation, the Agency publishes a summary of the opinion on its website.

European Medicines Agency (EMA), 16.01.2012 (tB).a