EMA PDCO grants specific waivers for Gamida Cell-Teva JV's StemEx study in paediatric patients

Jan 18 2010

The Gamida Cell-Teva Joint Venture (JV) announced today that the Paediatric Committee (PDCO) of the EMA has granted product specific waivers for StemEx for all subsets of the paediatric population (birth to 17 years of age) in all conditions. The PDCO emphasized that the granting of the waivers should not prevent the JV from considering developing StemEx for children with conditions where there is a need. StemEx is being developed by a joint venture equally owned and operated by Gamida Cell and Teva Pharmaceuticals ( TEVA).

In order to register a new medicinal product in the European Union, a company generally needs to clinically prove efficacy and safety in paediatric patients in addition to adults. Moreover, a company has to submit to the PDCO a Paediatric Investigational Plan (PIP) for the four existing paediatric age subsets which cover from birth to 17 years of age. The JV submitted a PIP for the age group 12 -17 together with product specific waiver requests for the three age groups between birth to 11 years of age.

As the added value of StemEx lies primarily in the treatment of larger children and adult patients, the PDCO waiver is congruent with the JV's strategy to develop StemEx initially for adolescents and adults.

Ms. Michal Austin, director of regulatory affairs said, "It is well known that the limited number of stem cells in cord blood inhibits the therapeutic use of cord blood transplantation in adolescents and adults. Gamida Cell's technology takes this small population of cord blood stem cells and cultures it with limited differentiation, to a size that has the potential to offer a therapeutic treatment to larger children and adults. Thus, the goal of the JV is for StemEx to significantly widen the scope of a base treatment, already common practice in children. As such, we believe, and the Agency has agreed, that our first priority in developing StemEx will be to offer the treatment to those patients with the most unmet clinical need, meaning adolescents and adults. This means, we are still on track for a 2011 market launch."

Gamida Cell is currently enrolling for the study of StemEx as an investigational treatment for adolescents (larger children) and adults with leukemia at leading cord blood transplant clinical sites in Italy, Hungary, Spain, Israel and the U.S. For more information about the study: http://www.stemexstudy.com.