BioWorld International Correspondent

BRUSSELS, Belgium - The European medicines agency gave clearance June 1 to the first medicine derived from transgenic biotechnology - antithrombin alfa, extracted from the milk of goats that have the human antithrombin gene inserted.

It recommended that ATryn, from Genzyme Europe, should be authorized for use in patients with congenital antithrombin deficiency undergoing surgery, to prevent deep-vein thrombosis and thromboembolism.

The application had been turned down by the agency in February, because the data were at the time judged insufficient. It was prepared to take into consideration only the data on five surgical cases, and it dismissed data on nine cases in childbirth and five cases of compassionate use in surgery. However, Genzyme sought a re-examination, with input of additional expert advice, and the agency now has reversed its earlier view and concluded an authorization was merited.

What changed the agency's mind was a meeting with European specialists in hematology and hemostasis, who suggested that data on effectiveness could be based on all patients studied, pregnant and non-pregnant, as they presented the same high risk of deep-vein thrombosis and thromboembolism. The experts also advised that the application should not be discounted because of a slight difference in the manufacture between the product used in the studies and the product to be marketed.

The recommendation for authorization - which still has to be completed by formal endorsement from the European Commission - has been made under the European Union's new regulation allowing authorization under exceptional circumstances for products in which the applicant can demonstrate that comprehensive data cannot be provided, for example because of the rarity of the condition. It is necessary, however, to demonstrate that the benefits outweigh the risks. And the agency asked the company to look in greater detail into the use of the medicine in pregnant women, so that a suitable dosing schedule can be developed.

ThromboGenics Plans Belgian Listing

ThromboGenics NV, based in Leuven, Belgium, and focused on vascular disease, announced plans June 2 for an initial public offering in Brussels on Eurolist by Euronext. It aims to use the proceeds principally to boost clinical and preclinical studies. The company claims a significant pipeline of drug candidates for visual disorders, cardiovascular diseases and cancer. Clinical development programs include microplasmin, a proteolytic enzyme under evaluation for macular edema and diabetic retinopathy, and as a thrombolytic for acute stroke and peripheral arterial occlusive disease. Professor Désiré Collen and KULeuven, one of the leading Belgian universities, jointly founded the company in 1991, initially to make use of their expertise with tissue plasminogen activator. Collen's laboratory was the first to produce tPA for clinical use.

Industry Wants EU To Push New Medicines

Doubtless inspired by the confidence of attaining its 10th anniversary, the European biotechnology association EuropaBio on May 30 called for the European Union to force its member states to make new medicines available to patients.

"The decision of access to medicines should not be left in the hands of the member states," Andrea Rappagliosi, Chairman of EuropaBio's health care council and a senior executive at Serono, told an audience of EU officials and politicians.

His approach foresees a framework that would oblige member states to adequately fund medicines that treat life-threatening and severely debilitating diseases - a radical departure from the 50-year-old EU principle that each member state remains sovereign in health care matters.

Rappagliosi accused the EU of "a lack of political leadership" in finding responses to the new therapies and technologies that the industry now is capable of providing. "Access to medicines by patients is at best haphazard because all 25 health care budgets are controlled by individual member states," he complained. "Some member states are simply inefficient at managing their systems," and the industry is a victim of discrimination, he alleged. There is no point in imposing expensive and time-consuming clinical and regulatory requirements for medicines authorization if patients do not get the lifesaving drugs they need and research is not adequately rewarded, he said.

EuropaBio has elected Philippe Archinard, CEO of Transgene, as chair of its Emerging Enterprise Council. It also has signed up 29 new member companies and associate members, including companies as diverse as Total, BP and Novo Nordisk.