Edition of 15 March 2006

The European Medicines Agency has published its Work Programme for 2006. For orphan medicinal products, the report states that the EMEA will propose "fee reductions which will provide maximum possible incentives during the development and marketing-authorisation phases."

Amongst the trends cited, the EMEA expects activities to stabilise at the 2005 level, and cites the outcome of the EC report on orphan medicinal products as being decisive for long-term trends and the possible evolution of orphan policy at the Community level, which could impact the EMEA and its practices.

A new chairperson and vice chairperson are due to be elected to the Committee on Orphan Medicinal Products at the new term beginning in April 2006.

The bone dysplasias are a diverse and complex group of rare genetic disorders affecting the development of the skeleton. Due to the rarity of these conditions, which also exhibit extensive clinical variability and genetic heterogeneity, determining an accurate diagnosis can be an extremely difficult, time-consuming and expensive undertaking for the non-expert.

In order to address these diagnostic problems within the wider European Community, the European Skeletal Dysplasia Network (ESDN) was established as a referral and communication network to provide help and diagnostic expertise to the medical community. In this context ESDN employs state-of-the-art DNA diagnostic and research technologies to provide diagnostic services and research collaborations respectively.

ESDN is composed of eight partners from six European countries and is coordinated by Dr Mike Briggs, a bone biologist at the University of Manchester. The project was initially established in January 2002 with funding from the European Commission’s Fifth Framework Programme (contract number QLG1-CT-2001-02188) and financial support from the Swiss Government (contract number OFES 01.0258). The primary goal of the project was to provide an integrated research and diagnostic network for skeletal dysplasias, which will ultimately provide a better understanding of the cellular, molecular and genetic factors that cause bone dysplasias, and to develop effective approaches for their diagnosis.

Using a unique web-based electronic referral system, ESDN partners in the United Kingdom, Belgium, Germany, Finland, Switzerland and France provide medical practitioners from anywhere in the world with specialist clinical advice and expert molecular diagnosis services for bone dysplasias.

Read more about ESDN

At its meeting on 7-8 March, 2006, the EMEA's Committee for Orphan Medicinal Products (COMP) adopted 9 positive opinions on orphan designation for medicinal products for the following indications:

The Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion on initial marketing authorisation application for Evoltra (clofarabine) manufactured by Bioenvision Ltd for the treatment of acute lymphoblastic leukaemia in paediatric patients. Evoltra is the twenty-fifth orphan-designated medicinal product to receive a positive CHMP opinion.

The CHMP has adopted a positive opinion for a similar biological medicinal product. Valtropin (somatropin), produced by BioPartners, is a growth hormone produced by recombinant DNA technology similar to Humatrope, already authorised in the EU. Somatropin is indicated for growth disturbance and growth hormone deficiency.

In January, the CHMP adopted its first positive opinion for a similar medicinal product, recommending to grant marketing authorisation for Omnitrope (somatropin) intended for growth disturbance and growth hormone deficiency, including Turner syndrome, chronic renal sufficiency and Prader-Willi syndrome in children. Omnitrope, produced by Sandoz GmbH, is similar to Genotropin, already authorised in the EU.

Also in January, the CHMP adopted a positive opinion recommending to grant marketing authorisation for Myozyme (recombinant human acid alpha-glucosidase) for the treatment of infantile-onset Pompe disease. Efficacy of Myozyme (produced by Genzyme Corp.) in late-onset Pompe disease has not been established to date.

Details of all orphan designations and authorisations granted to date by the European Commission are entered in the Community Register of Orphan Medicinal Products

Authors: Jesse L. Goodman, David T. Dennis and Daniel E. Sonenshine
Publisher: American Society for Microbiology Press, Washington, DC, May 2005