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COMP recommends eight drugs for orphan designation

Published: 1/18/2013
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The Committee for Orphan Medicinal Products of the European Medicines Agency has adopted eight positive opinions in January, recommending these drugs to the European Commission for orphan designation.

IHS Global Insight perspective



The European Medicines Agency's Committee for Orphan Medicinal Products (COMP) has granted positive opinions for eight drugs in January, recommending these drugs to be designated as orphan medicinal products by the European Commission.


The decisions include positive recommendations for French firm ERYtech Pharma's investigational treatment of acute myeloid leukaemia, and US company Pfizer's drug candidate for the treatment of Duchenne muscular dystrophy.


The decisions will be sent to the European Commission for consideration. If accepted, the drugs will be granted 10 years' marketing authorisation in the EU.

At its 141st plenary meeting held on 8–9 January, the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) had adopted eight positive opinions, recommending these drugs for designation as orphan medicinal products to the European Commission. The decisions included five decisions taken at the committee's first discussion, and a further five decisions taken at the second discussion.

The decisions taken at the second discussion were adopted following the sponsors' responses to the list of questions from the COMP.

Opinions adopted at second COMP discussion





Apeptico Forschung und Entwicklung (Austria)

Treatment of high altitude pulmonary oedema


BHR Pharma (Belgium)

Treatment of moderate and severe traumatic brain injury

Treprostinil sodium

SciPharm S.a.r.L (Luxemburg)

Treatment of chronic thromboembolic pulmonary hypertension

Opinions adopted at first COMP discussion

Humanised monoclonal antibody against myostatin

Pfizer (US)

Treatment of Duchenne muscular dystrophy

Humanised IgG1 kappa antibody against serum amyloid A and AL amyloid

Onclave Therapeutics (part of Elan, Ireland)

Treatment of amyloid light-chain amyloidosis

L-asparaginase encapsulated in erythrocytes

ERYtech Pharma (France)

Treatment of acute myeloid leukaemia

Recombinant adeno-associated viral vector containing the human CNGB3 gene

TMC Pharma (UK)

Treatment of achromatopsia caused by mutations in the CNGB3 gene


High Tech Participations (Germany)

Treatment of systemic sclerosis

Source: COMP

Apart from the above, there were four other applications for orphan medicinal designations that were withdrawn, the details of which were not disclosed.

Outlook and implications

The COMP issues recommendations on drugs for orphan designation that are indicated in the treatment of life-threatening or very serious conditions that affect no more than 5 in 10,000 people in the EU. It issued eight positive opinions for orphan designation in January.

These include a positive opinion for French biotech ERYtech Pharma's candidate treatment for acute myeloid leukaemia. The recommendation comes as further positive news for the French company, given that it had previously received a positive recommendation from the COMP for orphan medicinal product designation for its human erythrocytes encapsulating inositol hexaphosphate, a candidate treatment for sickle cell disease, in May 2012 (see Europe: 25 May 2012: EMA Committee Issues 16 Recommendations for Orphan Designation).

The recommendations this month further include a positive opinion for Pfizer's investigational treatment of Duchenne muscular dystrophy. Earlier in 2012, the COMP had issued a positive recommendation for another candidate treatment of Duchenne muscular dystrophy, Italian firm Italfarmaco's givinostat.

Overall, the positive recommendations will come as welcome news for the respective manufacturers, given that, subject to being accepted by the European Commission, the orphan status will enable these manufacturers receive certain incentives during the development process of the drug, as well as 10 years of marketing exclusivity upon authorisation.

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