Asphalion participated in the first Scientific Advice procedure with the European Medicines Agency (EMA) for the MYOCURE project. The meeting was arranged in order to ask the EMA some guidance to further design and elaborate two prototype adeno-associated vectors (AAV) for the treatment of two severe orphan diseases that affect children, the glycogen storage disease II (GSDII, or Pompe disease) and the myotubular myopathy (MTM). Ultimately, the MYOCURE goal is to obtain the orphan status for both products and diseases.
The meeting went through a friendly atmosphere and the result had been very positive and in agreement with what was proposed with enriching proposals given by the EMA. The attendees were: Marinee Chuah, as coordinator of the MYOCURE project, Kirsty Harper, as toxicology expert representing Envigo, and Marta Rayo Lunar and Joel Montane Mogas from Asphalion as regulatory affairs experts and organizers of the meeting.
Asphalion participates in the MYOCURE project as a regulatory partner addressing the regulatory issues related to the clinical development of the MYOCURE innovative gene therapy platform to ultimately treat patients suffering from GSDII or MTM. In particular, Asphalion develops and conducts the following activities:
–Early Scientific Advice given by EMA for the treatment of MTM and GSDII
–Orphan Drug Designation (ODD) for prototype AAV vector for the treatment of MTM
–Orphan Drug Designation (ODD) for prototype AAV vector for the treatment of GSD II
–Late Scientific Advice given by EMA including the design of clinical trial based on the prototype AAV vectors for the treatment of MTM and GSDII
The MYOCURE project brings together the expertise of a number of European groups (Spain, Belgium, France, UK, and Germany) in a number of fields required for an adequate development of the gene therapy project.
MYOCURE: This project has received funding from the European Union?s Horizon 2020 research and innovation programme under grant agreement No 667751.