Cerepro was originally filed for marketing approval under exceptional circumstances in late 2005 and was reviewed by the European Medicines Regulatory Agency (EMEA) with reliance on Phase II data. The review established that the technical chemistry and manufacturing controls, preclinical and environmental sections appeared acceptable, but more clinical data were needed to confirm the Phase II findings and to demonstrate the reproducibility of the results in a larger multi-center Phase III trial.
The Phase III study of Cerepro, completed in July 2007, showed that treatment with Cerepro resulted in a significant therapeutic benefit, supporting the results of the Phase II clinical studies.
The marketing authorization application (MAA) was filed following a pre-submission meeting with the EMEA rapporteur in the third quarter of 2008 and is for a full marketing approval. In accordance with the regulations for gene therapy trials, patients in the Phase III study will be monitored until death and an update of the trial will be provided annually.
Cerepro has orphan drug status in Europe and the US and is manufactured by Ark at its GMP facility in Finland.
David Eckland, R&D director at Ark, said: The acceptance of the MAA filed with the latest Phase III data was the next logical step for us to achieve with the EMEA and confirms that all the necessary components in the submission are acceptable for review. We now look forward to working with the EMEA during the review.