The Pharmaceutical Journal
Vol 268 No 7193 p487-492
13 April 2002

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European Agency for the Evaluation of Medicinal Products (EMEA) (www.emea.eu.int)

EMEA caught on the hop by increased number of orphan drug applications

Applications for designating medicines as orphan drugs have exceeded the expectations of the European Agency for the Evaluation of Medicinal Products (EMEA).

A spokesman told The Journal that it had expected fewer than two dozen applications a year. But 165 applications had been received since orphan status was introduced in 2000. As a result, the agency has had to ask the European Commission to double the budget allowance for the waiving of application fees for marketing authorisations for orphan medicines. Orphan medicines are those intended to treat serious and life-threatening diseases which affect fewer than 5 in 10,000 people across the community and which are unlikely to be developed under normal market conditions.

Successful applications for orphan status result in the waiving of fees for marketing authorisations and 10-year marketing exclusivity for the licensed indication.

The EMEA has started publishing summaries of opinions on designated orphan medicines on its website (www.emea.eu.int). To date, there have been 101 opinions from the Committee on Orphan Medicinal Products, 98 of which have been formalised by the EC. Six have been made public.

For example, fumagillin will be used to treat microsporidia-induced diarrhoea, which affects from 50,000 to 90,000 people in the European Union. Azacitidine is indicated to treat myelodysplastic syndromes (bone marrow failure and ineffective blood formation), which affect 41,000 to 113,000 Europeans. Surfaxin is to be used to treat acute lung injury resulting from a variety of causes, including toxic gas inhalation and chest trauma, which affects about 70,000 people a year in the EU.

Human engineered monoclonal antibody specific for transforming growth factor beta 1 (TGF-beta 1) will be used to treat systemic sclerosis (scleroderma), which affects from 11,000 to 49,000 EU residents annually. Adenovirus-mediated Herpes simplex virus thymidine kinase gene will be used to treat high grade glioma with subsequent use of ganciclovir sodium. High grade glioma is estimated to affect about 26,000 people in the EU. The sixth published opinion is for clofarabine (2-chloro-9-[2-fluoro-beta-D-arabino-fur-anosyl]adenine) which will be used to treat the 15,000 people in the EU who have acute lymphoblastic leukaemia.

Five orphan drugs have had positive opinions for marketing authorisation from the Committee for Proprietary Medicinal Products, four of which have been formalised by the European Commission decision. The five are:

1. Fabrazyme (agalsidase beta) for Fabry disease
2. Replagal (agalsidase alfa) for Fabry disease
3. Glivec (imatinib mesilate) for chronic myeloid leukaemia
4. Tracleer (bosentan) for pulmonary arterial hypertension
5. Trisenox (arsenic trioxide) for acute promyelocytic leukaemia

Two products have been exclusively authorised in parallel for Fabry disease because both applications were received by the EMEA on the same day and both progressed to final authorisation independently at the same time.