Thursday, June 21, 2012

Latest orphan designations and/or marketing authorisations

Medicinal products intended for rare diseases can receive an “orphan drug” label based on a defined number of criteria as summarised below:
  • The product is intended for an indication with a prevalence not exceeding 5 in 10 000 persons in the EU.
  • The disease is life-threatening, seriously debilitating or a serious and chronic condition.
  • No satisfactory method of diagnosis, prevention or treatment of the condition is already authorised in the EU. If any method exists, then the medicinal product has to demonstrate that it provides a significant benefit compared to the product for which orphan designation is being requested.

Committee for Orphan Medicinal Products

A positive opinion on orphan designation is given by the Committee for Orphan Medicinal Products (COMP) at the European Medicines Agency (EMA) based in London with the final decision made by the European Commission.
The COMP was created as part of the EU Regulation on Orphan Medicinal Products to review EU applications for the label "orphan drug”.

Orphan designation at any stage of development

Orphan designation is possible at any stage of drug development as long as proper scientific justification of the medical plausibility of the product in the indication requested is demonstrated.
The research may be pre-clinical (not yet tested on human subjects) or may have reached the human clinical trial phase.
Orphan designation does not indicate an endorsement for the use of the drug for the designated condition. Efficacy, safety and quality criteria first need to be satisfied for the granting of a marketing authorisation.
In addition to the position of Vice-Chair, EURORDIS has held two of the three seats assigned to patient organisation representatives on the COMP since its creation in 2000.
Detailed information on European orphan drug designation applications is available on theEMA website
A full list of designated and authorised orphan drugs in Europe available at:

6 June 2012

Treatment of meningioma
N-hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide

Treatment of schwannoma
N-hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide

Treatment of Wiskott-Aldrich syndrome
Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene

Treatment of cytomegalovirus disease in patients with impaired cell mediated immunity

Treatment of pancreatic cancer
Polyinosine-polycytidylic acid coupled with the polycationic polyethyleneimine

Treatment of ovarian cancer
1-(4-{4-amino-7-[1-(2-hydroxyethyl)-1H-pyrazol-4-yl] thieno [3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl) urea

Treatment of glioma
Adenovirus-associated vector containing human Fas-c gene

Treatment of adrenoleukodystrophy
Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA

25 May 2012

Treatment of soft tissue sarcoma
Yttrium (90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10