Ark Therapeutics Group plc (Ark) announced that the Marketing Authorisation Application (MAA) for Cerepro, its novel gene-based therapy for operable malignant glioma (brain cancer), which was recently filed with the European Medicines Regulatory Agency (EMEA), has cleared the validation stage. The Cerepro MAA application now commences formal review via the centralised procedure which is the standard route for all advanced therapies.
Cerepro was originally filed for marketing approval under exceptional circumstances in late 2005 and was reviewed by the EMEA with reliance on phase-II data. The review established that the technical chemistry and manufacturing controls (CMC), 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-centre 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 MAA was filed following a pre-submission meeting with the EMEA rapporteur in late Q3 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. As has already been announced, the first such update will be in Q1 2009.
Cerepro has Orphan Drug Status in Europe and the USA and is manufactured by Ark at its GMP facility in Finland.
Dr David Eckland, Research and Development 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."
Dr Nigel Parker, CEO of Ark, added, "The acceptance of the Cerepro submission is a further significant milestone for Ark. Gene-based medicine has made enormous progress both clinically and with the regulatory authorities in the last couple of years and it is the most advanced of the new biological therapeutics. The interest we are beginning to see in Cerepro as we get nearer to market is indicative of the potential that this whole area offers in finding new therapies that can make a real clinical difference for patients with hitherto untreatable diseases."