A war of words has broken out between Swiss-based drug company Serono, manufacturers of the human growth hormone Serostim, and the Committee for Proprietary Medicinal Products (CPMP) [part of the European Agency for the Evaluation of Medicinal Products (EMEA)] which recommended not granting approval for Serostim to be marketed for the treatment of AIDS wasting in the European Union on April 25th.
“The CPMP negative opinion marks a sad day for the European patient community suffering from rare diseases, and for all other interested parties,” claimed an angry Serono press release dated April 30th.
In an April 25th statement that accompanied a summary of its findings, the CPMP announced that “there is an unfavourable benefit to risk balance for Serostim and therefore [the CPMP] cannot recommend the granting of the marketing authorisation.”
Serostim was granted US FDA approval in 1996 for the treatment of HIV-associated wasting. However, in the age of HAART, AIDS wasting became designated a rare condition by the US Centers for Disease Control and Prevention (CDC) and consequently the EC’s Committee for Orphan Medicinal Products (COMP) made Serostim an orphan drug in August 2000. According to Serono, COMP confirmed that the indication of AIDS wasting in the EU should also be in agreement with the CDC disease definition.
Yesterday, Serono responded strongly to the CPMP statement, arguing that the EMEA committee failed to recognise HIV-related wasting as previously agreed. “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. 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.”
But the CPMP made it clear in their press release there was a difference between orphan drug designation and EU marketing approval: “The criteria for orphan designation mainly focus on the very low prevalence of a disease and are not the same as the criteria (quality, safety, efficacy) used in deciding whether a product should be granted a marketing authorisation,” they said.
This is not the first controversy to plague Serostim, which is also being investigated here and the US as a treatment for lipodystrophy (see Lipodystrophy treatment under investigation by US Attorney). Although the cost of Serostim was not mentioned by the CPMP as a factor in non-approval, it remains the biggest stumbling block in clinical practice. There is also the major issue of physician unwillingness to prescribe anabolic agents to patients with HIV-related wasting. Although much cheaper alternatives exist for the treatment of HIV-related wasting - including steroids like nandrolone - here in the UK they are rarely available to patients who would benefit.
Serono have 15 days from April 25th to appeal the decision, and are currently “considering the appropriate next steps.” In the meantime, Serono reassures those European patients currently receiving Serostim in clinical trials and through compassionate use schemes, that they will do all they can to make sure access to the drug continues.
Further information on this website
Moyle G et al. Recombinant Human Growth Hormone is effective to treat HIV/AIDS associated wasting in the era of highly active antiretroviral therapy. Fourteenth International Conference on AIDS, Barcelona, abstract LbPeB9012, 2002.