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Europe’s May 2015 Products Recommended For Orphan Drug Designation

May 20, 2015

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting May 12 – 13, 2015.

At this meeting, there are 11 positive opinions recommending the following medicines for designation as orphan medicinal products. COMP’s opinions are forwarded to the European Commission (EC). The EC will then decide whether to grant an orphan designation for the medicines in question. Public summaries of the opinions will be available on the EMA website following adoption of the respective decisions on orphan designation by the EC.


Recommended ODDs for:

•   2 Lymphoma indications for Roche’s Gazyva (Obinutuzumab)

•   Ultragenyx’s Triheptanoin for very long-chain acyl-CoA dehydrogenase deficiency

•   Mitsubishi Tanabe Pharma’s Edaravone for ALS

•   2 Gene Therapies (adeno-associated viral vectors):

  1. Hemophilia B (Baxter)
  2. Spinal Muscular Atrophy (AveXis).


EMA COMP May 2015 ODDs Recommended

Product Name Sponsor Company Indication
3-{[2,3,5,6-tetrafluoro-3′-(trifluoromethoxy)biphenyl-4-yl]carbamoyl}thiophene-2-carboxylic acid Panoptes Pharma Ges.m.b.H. Non-infectious uveitis
Allogeneic ex-vivo-expanded human umbilical cord blood-derived mesenchymal stem cells PSR Group B.V. Prevention of bronchopulmonary dysplasia
Antisense oligonucleotide directed against TGF-β2 mRNA Isarna Therapeutics GmbH Prevention of scarring post glaucoma filtration surgery
Obinutuzumab Roche Marginal zone lymphoma
Obinutuzumab Roche Follicular lymphoma
Synthetic 47-amino-acid N-myristoylated lipopeptide, derived from preS region of Hepatitis B virus MYR GmbH Hepatitis delta virus infection
Adeno-associated viral vector containing human factor IX gene Baxter Hemophilia B
Adeno-associated viral vector serotype 9 containing human SMN gene AveXis EU, Ltd Spinal Muscular Atrophy
Edaravone Mitsubishi Tanabe Pharma Europe Ltd ALS
Trehalose Dr. Ulrich Granzer Spinocerebellar ataxia
Triheptanoin Ultragenyx UK Limited Very Long-chain acyl-CoA dehydrogenase deficiency


Please Note: “Erlenmeyer Flasks” From Argonne US National Lab  [Public domain in the US] | Wikimedia Commons.

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