EMA Waiver Paves Way for StemEx Leukaemia Study

17 January 2010 (Last Updated January 17th, 2010 18:30)

An international study of StemEx, an experimental treatment for leukaemia, lymphoma and MDS, will now begin enrolling patients after the European Medicines Agency (EMA) waived requirements to test the treatment in paediatric patients. To register a new medicinal product in the EU, a com

An international study of StemEx, an experimental treatment for leukaemia, lymphoma and MDS, will now begin enrolling patients after the European Medicines Agency (EMA) waived requirements to test the treatment in paediatric patients.

To register a new medicinal product in the EU, a company generally needs to prove efficacy and safety in paediatric patients aged 17 and under.

However, the agency has agreed that because the unmet medical need is for adolescents and adults rather than for paediatric patients, the EMA will waive the normally required paediatric investigational plan, which covers paediatric groups from birth to 17 years of age.

The Gamida Cell-Teva joint venture (JV), which is developing the treatment, will now begin enrolling at clinical sites in Italy, Hungary, Spain, Israel and the US.

Director of regulatory affairs Michal Austin said that the goal of the JV is for StemEx to significantly widen the scope of a base treatment already common in children.

"As such, the agency has agreed to offer the treatment to those patients with the most unmet clinical need, meaning adolescents and adults," Austin said.

"This means we are still on track for a 2011 market launch."