BioWorld International Correspondent

BRUSSELS, Belgium - The European Union's orphan drug system is successful and fully functional, according to comments from European biotechnology sources.

But its full development requires more incentives and more cooperation with member countries, too, according to a new evaluation of the five-year-old system.

The comments come from Amgen Inc., Genzyme Corp., and the two European biotechnology lobby groups, EuropaBio and EBE, as well as from other stakeholders who responded to an EU consultation on how the system was performing.

And according to the EU's just-published summary of the comments, incentives still need to be strengthened, particularly since the initial attractions of a 10-year period of market exclusivity have been diluted by the more recent adoption of a similar period of data protection for all innovative products.

One suggestion is to create a category of "super-orphan" drugs for extremely rare conditions, and to link the incentives inversely to disease prevalence, such as by making the duration of market exclusivity dependent on patient numbers.

Another suggested improvement is to widen the "significant benefit" criterion for designation as an orphan, to include improvement of the quality of life alongside improved clinical safety and efficacy.

However, some of the stakeholder views summarized by the EU call for reductions in incentives.

Those suggestions, understood to come from the international organization representing mutual health insurance groups, aim at removing the orphan status of medicines that become blockbusters, at focusing more on cost effectiveness and at limiting the future cost of reimbursing a growing arsenal of orphan drugs.

Meanwhile, at its April 4-5 meeting, the EU's Committee for Orphan Medicinal Products recommended designation of a further nine medicines as orphan products, including 1-deoxygalactonojirimycin hydrochloride, from Amicus Therapeutics UK Ltd, for treatment of Fabry disease; bi-layer engineered skin composed of autologous keratinocytes and allogeneic fibroblasts embedded in a plasma matrix, from Cellerix SL, for treatment of epidermolysis bullosa; and recombinant P-selectin glycoprotein immunoglobulin, from RJM Consultancy, for prevention of post-transplantation graft dysfunction.

Novo Withdraws Application To Extend NovoSeven

Novo Nordisk A/S has withdrawn its October 2005 application to extend the European marketing authorization for NovoSeven to cover acute intracerebral hemorrhage in adults for limiting hemorrhage growth and improving clinical outcomes.

The product - human recombinant coagulation Factor VIIa - currently is indicated in the EU for the treatment of bleeding episodes and prevention of bleeding during surgery or invasive procedures in specific patients with certain blood-clotting disorders.

When the European Medicines Agency requested additional safety and efficacy data in support of the application for an extension, the company decided to withdraw the current application and to resubmit an application when an ongoing clinical study is completed.

Vienna Meeting Heightens Coexistence Tensions

As expected, rival views on the merits of biotechnology-derived crops clashed repeatedly at the Vienna Conference on Coexistence of Genetically Modified, Conventional and Organic Crops on April 4-6. (See BioWorld International, April 5, 2006.)

Controversy was intensified by what many biotechnology industry executives saw as a negative stance by senior EU officials.

And industry anxieties were reinforced about the outcome of an extended internal debate on GMO safety that the European Commission was scheduled to hold today.

"The introduction of GM crops has provided a new challenge in finding the appropriate coexistence measures," Stavros Dimas, European commissioner for environmental affairs, and a former Greek agriculture minister, told the Vienna meeting.

"Applications for cultivation of GMO products raise a whole new series of possible risks to the environment, notably potential longer-term effects that could impact on biodiversity," he warned.

And he went on to talk of ensuring that "the environment is protected from potential risks arising from the cultivation of GMOs."

The commissioner gave strong emphasis to his perception of "concerns of member states and the public about the safety of GMOs," and underlined that in risk assessment of these products, "it is vital that not only short-term and direct effects, but also long-term and indirect effects should be assessed."

Industry executives felt that the commissioner's insistently negative tone - and frequent references to risk, to ensuring that scientific input into risk assessment "is of the highest possible quality," and to talking up the use of upgraded conventional varieties as an alternative to GM crops - presented an unbalanced review of what is a highly controversial topic in Europe.

"It must frustrate many in Europe that others, such as Commissioner Dimas, spoke about issues that are irrelevant to coexistence such as environmental risk assessments for approvals of new products; coexistence is about existing approved products," said Simon Barber of EuropaBio.

He claimed the commissioner was "confused" and misinformed the audience.

"It is not up to Commissioner Dimas to decide whether EU consumers do or don't want GMOs," Barber said.

Biotech industry organizations have been circulating strong rebuttals of many of the criticisms made of GM crops at the conference, initiating an Internet debate with the slogan: "Do you think the Vienna Conference could have been better orientated to the real questions around coexistence?"

It also has been widely remarked in industry circles that the governments in both Austria, which hosted the conference, and Greece, from where Commissioner Dimas comes, are fiercely opposed to GM crops.