The Committee for Orphan Medicinal Products (COMP) has adopted a total of nine recommendations for orphan designation, including four designation applications for very rare forms of lipodystrophy. During its recently held meeting COMP also confirmed the orphan designation for Kalydeco. This follows the recommendation made by the Agency’s Committee for Medicinal Products for Human Use (CHMP) in May 2012 to grant a marketing authorisation for this novel treatment for cystic fibrosis.
One of the COMP’s responsibilities is to review whether a designated orphan medicine still fulfils the designation criteria by the time it receives a recommendation for marketing authorisation from the CHMP. The COMP looks at the seriousness and prevalence of the condition, the existence of other satisfactory methods of treatment or – if other treatments are authorised in the European Union (EU)— at the significant benefit of the product over existing treatments.
At its June 2012 meeting, the COMP confirmed that Kalydeco continues to fulfil the orphan designation criteria. This means that once the European Commission has adopted a marketing authorisation decision, Kalydeco will benefit from 10 years of market exclusivity in the European Union. During this time, no other similar medicines with a comparable therapeutic indication can be placed on the market, unless certain exceptions set out by the Orphan Regulation apply.
The COMP’s review of Kalydeco’s orphan designation will be published following the publication of the European public assessment report (EPAR) for the medicine.
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 five in 10,000 people in the EU or that would not be developed without incentives.
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 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.
EP News Bureau – Mumbai
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