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London, United Kingdom (December 13, 2011) – During its December 2011 meeting, the Committee for Orphan Medicinal Products (COMP) recommended granting orphan designation for nine substances.
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 or that would not be developed without incentives.
One of the applications recommended for designation in December is for ‘autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene’ for the treatment of X-linked chronic granulomatous disease (CGD). Patients with this rare genetic disorder suffer from long-term and recurrent infections because certain immune system cells cannot fight infections as they should. It is estimated that CGD affects less than two in one million people in the European Union (less than 1.000 people in total).
Gene therapy has the promise to directly target the cause of this rare disease, while the currently authorised treatments for this condition only treat patients’ complications. This was recognised as a benefit by the COMP when adopting its recommendation.
Another substance recommended for designation in December is ‘human monoclonal antibody against Fas ligand’ for the treatment of pemphigus, a group of rare auto-immune diseases that cause blistering of the skin and mucous membranes (mouth, nose, throat, eyes, and genitals). Some forms of the disease, including the most common form, may be fatal if left untreated. Pemphigus is thought to affect less than 2.4 in 10 000 Europeans (less than 122.000 people in total).
If developed for marketing authorisation, this monoclonal antibody treatment could offer a significant public health benefit as it offers an alternative to the standard approach of treating patients with immunosuppressive therapies.
The COMP recommendations for these substances are now being sent to the European Commission for the adoption of a decision. Once an orphan designation has been granted, sponsors have access to certain incentives 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 this website.
European Medicines Agency (EMA), 13.12.2011 (tB).