BRUSSELS, Belgium - The new European Union advisory committee for orphan medicinal products has recommended orphan status for three products under development: two for Fabry disease and one for AIDS wasting. These are the first such recommendations since the launch this summer of the EU's new system to boost research into rare diseases. Orphan status is granted based on epidemiological or economic criteria for life-threatening or debilitating conditions for which there is no satisfactory method of treatment authorized in the EU, or in cases where the manufacturer can establish that the product will be of significant benefit to patients affected by the condition.
The recommendations have been forwarded to the European Commission, which will now make the formal designations, so that the products can qualify for the benefits the new scheme provides for - including reduced fees for EU marketing authorization and scientific advice from the EU regulatory authorities, and, most importantly, a period of marketing exclusivity after authorization. However, the committee stresses that designation as an orphan is not an unqualified endorsement of a product, which still will have to satisfy criteria on quality, safety and efficacy in the proposed indication to obtain a marketing authorization.
So far, 24 applications have been made for orphan status, and are now undergoing the assessment procedure; coordinators and experts have also been appointed for two further applications expected to be formalized shortly. The committee's next meeting is Sept. 12-13.
Austria Amends Gene Law
First Austria defied the European Union by banning the use and sale of Novartis' Bt-Maize 176, and the planting of Monsanto's Bt-Maize MON 810 and of Aventis' herbicide-tolerant Maize T 25, despite the fact that they have won EU approval. Now the government has introduced other changes to its national biotechnology regulation.
The most recent amendments to the 1995 "gene law" (Gentechnikgesetz), which covers contained use, deliberate release, genome analysis and human gene therapy, have also introduced provisions covering, among other things, civil liability, increased public participation, the creation of a GMO register, and penalties for non-compliance, according to an OECD update on Austria's regulation.
The OECD also points out that the Federal Ministry of Social Security and Generations is now responsible both for contained-use and deliberate-release applications from industry and research institutions, with the exception of universities, which come under the Federal Ministry of Education, Science and Culture. The Federal Ministry of Agriculture, Forestry, Environment and Water Management and the Federal Environment Agency give comments in cases of deliberate release and of placing products on the market. An advisory Commission for Gene Technology, with representatives from science, industry, government, non-governmental organizations and trade unions, comments on regulations and establishes technical guidelines; its scientific subcommittees give advice on actual applications of contained use and deliberate release.
In other European news:
¿The European Plant Biotechnology Network reports that investment and innovation are being discouraged by the current European Union moratorium on new approvals of GMO crops, by the complexity of EU marketing authorization rules, and by the lack of EU research funding. The EU-funded body of industry representatives and scientists is organizing a major science-industry contact meeting in Wageningen, the Netherlands, on Oct. 26-28. In addition, European plant science research is facing additional problems because "its public relations is in bad shape." Opposition to the new technology "has grown into a media war, where scientific facts have been taken out of context and emotive arguments have gained weight," says EPBN.
¿The European Medicines Evaluation Agency, of London, said Executive Director Fernand Sauer is leaving the agency to take over as director in the European Commission's restructured health department in Brussels. His successor at EMEA has yet to be named, but as part of an EMEA restructuring, Nokl Wathion of Belgium has been named as head of the unit responsible for post-marketing issues for human medicines; he is currently head of pharmacovigilance and regulatory affairs at the EMEA.
¿The European Medicines Evaluation Agency, of London, and the Biotechnology Speciality Section of the British Toxicology Society are organizing an Oct. 9 seminar in London on new vaccine development. With a focus on preclinical safety evaluation of vaccines in the light of new adjuvants and other future challenges, the seminar will assess the need for updating EU guidelines on the evaluation of vaccines. The industry point of view will be provided by speakers from Celltech Chiroscience, Glaxo Wellcome, Aventis, SmithKline Beecham and Merck & Co.
¿The government of Estonia is introducing a law on the study of human genes. The law is a step toward a seven-year, US$200 million project to create a nationwide genetic data bank that will house data on 1 million people within five years. The law is geared toward ensuring that participation in genetic studies is strictly voluntary. It will also prevent discrimination on the basis of genetic information. The government hopes the project will drive the establishment of new genetic and information technology firms in Estonia. Genetic studies have already been legally regulated in Iceland, where a similar genetic reserve project has been launched, and to a lesser extent in Great Britain and Canada.
¿A new scientific association called ADEBIR has been set up in the Spanish wine-producing region of La Rioja, to help harness biotechnology to its wine production. It aims to promote agricultural and ecological biotechnology research projects, to preserve the region's biodiversity, and to link local industry and academic research in genomics and new technologies into European research programs.
¿The Biotechnology and Finance Forum, an organization set up by the European Union to promote industrial biotechnology in Europe, will hold its third conference Nov. 30-Dec. 1 in Cambridge, UK, with a focus on mobilizing stakeholders in research, industry and finance toward maintaining sustained growth and facilitating consolidation in the sector.
¿Greenpeace says it will sue the seed company Golden Harvest, which was responsible for marketing GMO-contaminated maize seed in France. More than 4,000 hectares of farmland was sown with "conventional" maize found to be contaminated with three genetically modified varieties, one of which is not approved for cultivation in the European Union, says Greenpeace. It has accused Golden Harvest of "deliberately commercializing and spreading in the environment unauthorized GMOs."
¿Roche and DeCode Genetics, of Iceland, reported the successful mapping of a novel gene that contributes to the occurrence of late-onset Alzheimer's disease. The finding underscores the role of genetics in the pathogenesis of the most common form of Alzheimer's, says Roche, which plans to start development programs for new diagnostics and therapeutics building on this genetic information.
¿Genetics Company, of Zurich, has concluded its first financing round, involving CHF6 million (US$3.5 million). This spin-off of the University of Zurich and the Swiss Institute for Experimental Cancer Research is working mainly in cancer and Type II diabetes. Listing on the stock exchange is planned "in the medium term," the company said. According to chief operating office Mario Jenni, "We are present and available in those areas where the pharmaceutical industry has indicated its lack of innovation": functional identification of new sites of drug action and drug precursors, and development of procedures for discovery of tissue and organ development modulators for reprogramming human tumors.