BioWorld International Correspondent

PARIS - Serono SA issued a statement voicing its disagreement with the negative opinion issued by the Committee for Proprietary Medicinal Products, which said Serostim should not be granted initial marketing authorization for the treatment of AIDS wasting in the European Union.

The Geneva-based company said that, unlike the CPMP, it believes data confirm the safety and efficacy of Serostim and that they "justify the therapeutic benefits for patients who have no alternative treatment" for what it describes as a "chronic, debilitating and potentially life-threatening condition."

AIDS wasting was designated as a rare condition on the basis of the published criteria of the U.S. Centers for Disease Control and Prevention. In the EU, the European Commission granted Serostim orphan drug status in August 2000 based on the opinion of the Committee for Orphan Medicinal Products, which also recommended that the EU should apply the CDC disease definition to the indication of AIDS wasting.

Serostim is the only growth hormone approved by the FDA for the treatment of AIDS wasting. It was granted accelerated approval in the U.S. in 1996 and is now on the market in 13 countries.

Serono supported its application to the CPMP with the positive results of a confirmatory international trial (GF9037), which demonstrated a highly significant increase in both work output and lean body mass as well as an improvement in quality of life for patients treated with Serostim as compared to those treated with placebo. More than 700 patients in the U.S., the EU and elsewhere were involved in the trial.

"It is unfortunate that the current CPMP view of AIDS wasting is not aligned with the definition of the orphan drug designation granted in Europe," Serono said. "The CPMP opinion reflects the difficulties in the understanding of rare and continuously evolving diseases and in the assessment of appropriate treatments. The full implementation of the EU orphan regulation needs to be improved to ensure access to innovative treatments for rare diseases in Europe."