The Committee for Orphan Medicinal Products (COMP) has released positive opinions for orphan drug designation of 15 medicines in Europe.
IHS Life Sciences perspective | |
Significance | The COMP has issued positive recommendations for the approval of orphan drug designation for 15 active ingredients in the European Union. |
Implications | Following the orphan drug designations and their approval for marketing authorisation, the molecules obtain 10 years' market exclusivity in the EU as well as incentives for drug development. |
Outlook | The European Commission will make a final decision within 30 days from the receipt of the COMP's recommendations. |
The Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has issued 15 positive opinions for the inclusion of 15 medicines in the EU register of orphan medicinal products for human use. The full COMP document is available here.
COMP recommendations | ||
Drug | Company/organisation | Indication |
Endorsements adopted at second session | ||
1-(4-(N-glycylamido)phenyl)-3-trifluoromethyl-5-(phenanthren-2-yl)-pyrazole-hydrochloride | Arno Therapeutics (US) | Treatment of cryptococcosis |
1-(4-(N-glycylamido)phenyl)-3-trifluoromethyl-5-(phenanthren-2-yl)-pyrazole-hydrochloride | Arno Therapeutics | Treatment of tularaemia |
Ecothiophate iodide | JJGConsultancy (United Kingdom) | Treatment of Stargardt's disease |
Fluciclovine (18F) | Blue Earth Diagnostics (United Kingdom) | Treatment of glioma |
Human reovirus type 3 Dearing strain | Oncolytics Biotech (Canada) | Treatment of pancreatic cancer |
Lenalidomide | Celgene (US) | Treatment of marginal zone lymphoma |
Recombinant monoclonal IgG1 antibody against T-cell immune response cDNA 7 | Nekonal S.a.r.l. (Luxemburg) | Prevention of graft rejection following solid organ transplantation |
Rimeporide | EUDRAC (Germany) | Treatment of Duchenne muscular dystrophy |
Rintatolimod | NV Hemipsherx BioPharma (US) | Treatment of Ebola virus disease |
Endorsements adopted at first session | ||
Adeno-associated viral vector serotype 5 containing the human CHM gene | HORAMA (France) | Treatment of choroideremia |
Nitric oxide | Biological Consulting (US) | Treatment of cystic fibrosis |
Phenol, 4-[2-(aminomethyl)-4-thiazolyl]-2,6-bis(1,1-dimethylethyl) monohydrochloride | Ipsen (France) | Treatment of Huntington's disease |
Recombinant human mesencephalic astrocyte-derived neurotrophic factor | Clinipace (US) | Treatment of retinitis pigmentosa |
Sodium 2-hydroxylinoleate | Ability Pharmaceuticals (Spain) | Treatment of neuroblastoma |
Xenon | Neuroprotexeon (UK) | Treatment of perinatal asphyxia |
Source: COMP | ||
Outlook and implications
Among the positive recommendations issued by the COMP, NV Hemipsherx BioPharma (US)'s investigational treatment rintatolimod has received an endorsement for orphan drug designation in the treatment of Ebola. Meanwhile, the company and the United States Army Medical Research Institute of Infectious Diseases (USAMRIID) have reported evidence from studies on the medicine in vitro and in an animal model. The producer has planned further studies to establish the treatment's efficacy in humans.
Furthermore, Celgene's lenalidomide has been recommended for orphan drug designation in the EU for the treatment of marginal zone lymphoma. The medicine secured orphan drug designation in the European Union for its use in myelodysplastic syndromes and multiple myeloma in 2005 (see United States: 10 April 2006: E.U. Marketing Authority Accepts Celgene's Application for Revlimid).
The endorsements given by the COMP are encouraging news for the applicants as they pave the way for the manufacturers to gain orphan drug designation in the EU. In fact, following the publication of the COMP recommendations, the European Commission will make a final decision within 30 days from the receipt of the endorsements.
Orphan drug status is granted to compounds developed for the treatment of rare diseases that occur in a maximum of five in 10,000 people in the EU. Upon a final decision from the European Commission, the drug manufacturers gain a 10-year period of patent protection in the EU. Furthermore, market exclusivity can be extended by two years for paediatric approval. In addition, a number of incentives, including reduction of fees, are provided with the designation.