EMEA’s opinion to designate new orphan drugs


The COMP adopted six positive opinions recommending the following medicines for designation as orphan medicinal products to the European Commission:

For the following medicines the EMEA review began on 12 September 2008 with an active review time of 90 days. 

•  Recombinant human hepatocarcinoma-intestine-pancreas / pancreatic associated protein, from Alfact Innovation SAS, for treatment of acute liver failure.
•  Type I native bovine skin collagen, from arGentis Autoimmune Europe Limited, for treatment of systemic sclerosis.
For the following medicines the EMEA review began on 13 October 2008 with an active review time of 59 days.

•  Adeno-associated viral vector serotype 5 containing the human  ABCA4 gene, from Fondazione Telethon, for treatment of Stagardt’s disease.

•  Cyclopropane-1,1-dicarboxylic acid [4-(6,7-dimethoxy-quinolin-4-yloxy)-phenyl]-amide (4-fluoro-phenyl)-amide, (L)-malate salt, from PPD Global Ltd, for treatment of medullary thyroid carcinoma.

•  Recombinant human proinsulin, from ProRetina Therapeutics S.L., for treatment of retinitis pigmentosa.

•  Yttrium (90 Y)-DOTA-radiolabelled humanized monoclonal antibody against mucin 1, from Immunomedics GmbH, for treatment of pancreatic cancer.

Public summaries of opinion will be available on the EMEA website which the Agency updates following adoption of the respective decisions on orphan designation by the European Commission.

Source: EMEA website. Monthly report from the December meeting

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