The Committee for Orphan Medicinal Products (COMP) has released positive opinions for orphan drug designation of 11 medicines in Europe.
IHS Life Sciences perspective | |
Significance | The Committee for Orphan Medicinal Products (COMP) has issued positive recommendations for the approval of orphan drug designation for 11 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 and the company's receive 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 11 positive opinions for the inclusion of 11 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 | |||
3-{[2,3,5,6-tetrafluoro-3'-(trifluoromethoxy)biphenyl-4-yl]carbamoyl}thiophene-2-carboxylic acid | Panoptes Pharma (Austria) | Treatment of non-infectious uveitis | |
Allogeneic ex-vivo-expanded human umbilical cord blood-derived mesenchymal stem cells | PSR Group (Netherlands) | Treatment of bronchopulmonary dysplasia | |
Antisense oligonucleotide directed | Isarna Therapeutics (Germany) | Treatment of scarring post glaucoma | |
Obinutuzumab | Roche (Switzerland) | Treatmen of marginal zone lymphoma | |
Obinutuzumab | Roche (Switzerland) | Treatment of Follicular lymphoma | |
Synthetic 47-amino-acid N-myristoylated lipopeptide | MYR GmbH (Germany) | Treatment of hepatitis delta virus infection | |
Endorsements adopted at first session | |||
Adeno-associated viral vector containing the human factor IX gene | Baxter Innovations (Austria) | For treatment of haemophilia B | |
Adeno-associated viral vector serotype 9 containing the human SMN gene | AveXis (EU) | For treatment of spinal | |
Edaravone | Mitsubishi Tanabe Pharma Europe (UK) | For treatment of amyotrophic lateral sclerosis | |
Trehalose | Dr Ulrich Granzer (Germany) | For treatment of spinocerebellar ataxia | |
Triheptanoin | Ultragenyx (UK) | For treatment of very long-chain acyl-CoA dehydrogenase deficiency | |
Source: COMP | |||
Outlook and implications
Among the positive recommendations issued by the COMP, it is important to highlight that Roche's Gazyva (obinutuzumab) has received endorsement for double-orphan-drug designation for the treatment of two different types of lymphomas: follicular and the marginal zone lymphoma.
Furthermore, MYR GmbH's synthetic 47-amino-acid N-myristoylated lipopeptide has been recommended for orphan drug designation in the EU for the treatment of hepatitis delta virus infection, which is considered to be the most severe form of the virus. Incidence of hepatitis delta, which only occurs as a co-infection in patients with hepatitis B, varies from low in Europe, United States, and Japan, to relatively high (as a proportion of hepatitis B patients) in China, Russia, Africa, South America, Central Asia, and Turkey.
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 European Union. 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.