European Medicines Agency (EMA) Waives Requirement to Test StemEx(R) in Paediatric Patients 17 Years of Age and Under
- The International Study of StemEx, an Experimental Treatment forLeukemia, Lymphoma and MDS, is now Enrolling at Clinical Sites in Italy,Hungary, Spain, Israel and the U.S.
The Gamida Cell-Teva Joint Venture (JV) announced today that thePaediatric Committee (PDCO) of the EMA has granted product specific waiversfor StemEx for all subsets of the paediatric population (birth to 17 years ofage) in all conditions. The PDCO emphasized that the granting of the waiversshould not prevent the JV from considering developing StemEx for childrenwith conditions where there is a need. StemEx is being developed by a jointventure equally owned and operated by Gamida Cell and Teva Pharmaceuticals(NASDAQ: TEVA).
In order to register a new medicinal product in the European Union, acompany generally needs to clinically prove efficacy and safety in paediatricpatients in addition to adults. Moreover, a company has to submit to the PDCOa Paediatric Investigational Plan (PIP) for the four existing paediatric agesubsets which cover from birth to 17 years of age. The JV submitted a PIP forthe age group 12 -17 together with product specific waiver requests for thethree age groups between birth to 11 years of age.
As the added value of StemEx lies primarily in the treatment of largerchildren and adult patients, the PDCO waiver is congruent with the JV'sstrategy to develop StemEx initially for adolescents and adults.
Ms. Michal Austin, director of regulatory affairs said, "It is well knownthat the limited number of stem cells in cord blood inhibits the therapeuticuse of cord blood transplantation in adolescents and adults. Gamida Cell'stechnology takes this small population of cord blood stem cells and culturesit with limited differentiation, to a size that has the potential to offer atherapeutic treatment to larger children and adults. Thus, the goal of the JVis for StemEx to significantly widen the scope of a base treatment, alreadycommon practice in children. As such, we believe, and the Agency has agreed,that our first priority in developing StemEx will be to offer the treatmentto those patients with the most unmet clinical need, meaning adolescents andadults. This means, we are still on track for a 2011 market launch."
Gamida Cell is currently enrolling for the study of StemEx as aninvestigational treatment for adolescents (larger children) and adults withleukemia 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.
About Gamida Cell
Gamida Cell Ltd. is a world leader in stem cell expansion technologiesand therapeutic products. The company is developing a pipeline of products instem cell transplantation and in tissue regeneration to effectively treatdebilitating and often fatal illnesses such as cancer, hematological,autoimmune and ischemic diseases. Gamida Cell's therapeutic candidatescontain populations of adult stem cells, selected from non-controversialsources such as umbilical cord blood and bone marrow, which are expanded inculture. Gamida Cell was successful in translating these proprietaryexpansion technologies into robust and validated manufacturing processesunder GMP. Gamida Cell's flagship product, StemEx, is now being studied as atherapy for patients with blood cancers in an international pivotal trial atleading transplant centers in the U.S., Europe and Israel. StemEx has orphandrug designation in the U.S. and in Europe. Gamida Cell's currentshareholders include: Elbit Imaging, Biomedical Investment, Israel HealthcareVenture, Teva Pharmaceutical Industries, Amgen, Denali Ventures and AurigaVentures. For more information, please visit: http://www.gamida-cell.com.Press contact: Marjie Hadad Media Liaison/IR Gamida Cell firstname.lastname@example.org +972-54-536-5220
SOURCE Gamida Cell Ltd.
You May Also Like