In a strategic meeting that was held in London early this month, the Committee for Orphan Medicinal Products (COMP), a body set up by the European Medicines Agency (EMA) to review applications from people or companies seeking 'orphan medicinal product designation', adopted a total of nine recommendations for orphan designation, including four designation applications for very rare forms of lipodystrophy.
During the meeting, COMP adopted nine positive opinions recommending the following medicines for designation as orphan medicinal products to the European Commission (EC). Orphan designation can be granted early in the development process of 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.
COMP adopted the use of 1-[(2-Chloro-4-methoxyphenoxy)methyl]-4-[(2,6-dichlorophenoxy)methyl]benzene for prevention of poliomyelitis in patients with immunodeficiencies deemed at risk. This product is being developed by ProPhase Development Ltd. It also acknowledged the use of metreleptin developed by Aptiv Solutions (UK) Limited for the treatment of Barraquer-Simons syndrome, Berardinelli-Seip syndrome,familial partial lipodystrophy and Lawrence syndrome.
Other .approvals .include the use of Albireo AB's ((2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro-1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid for the treatment of progressive familial intrahepatic cholestasis. Use of hexasodium phytate for treatment of calciphylaxis developed by Sanifit Laboratoris, S L and human apotransferrin for treatment of congenital hypotransferrinaemia, developed by Sanquin Blood Supply Foundation.
COMP also adopted the use of recombinant human pentraxin-2 developed by Appletree Europe S.à.r.l for the treatment of idiopathic pulmonary fibrosis. Public summaries of opinions will be available on the EMA website following adoption of the respective decisions on orphan designation by the EC. Since its meeting held in May this year, the EC granted 14 final designations as orphan medicinal product.
During the two day meeting held on June 12 and 13, the COMP also confirmed the orphan designation for Kalydeco. This follows the recommendation made by the European Medicines Agency's (EMA's) Committee for Medicinal Products for Human Use (CHMP) in May 2012 to grant a marketing authorisation for this novel treatment for cystic fibrosis. The COMP confirmed that Kalydeco continues to fulfil the orphan designation criteria.
This means that once the EC 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.
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 next COMP meeting will be held on July 10 and 11 in Uppsala, Sweden.