Posted on: 8 January 2009, source: Biospace
Ark Therapeutics announced that the Marketing Authorisation Application (MAA) for Cerepro®, Ark’s novel gene-based therapy for operable malignant glioma (brain cancer) which was recently filed with the 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 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.”

Malignant glioma is a devastating and fatal form of brain tumour that is usually confined to the brain. The current standard therapy involves surgically removing the solid tumour mass (when possible) and initiating radiotherapy and/or chemotherapy. Even with the latest approved treatments, most patients die within one year of diagnosis, with average survival being about eight months. Little therapeutic progress has been made in recent years and the prognosis for malignant glioma patients is poor. A high unmet clinical need exists for new treatments that prolong life in this devastating disease. There are approximately 16,000 cases of malignant glioma in the EU which are operable.

Cerepro® is an adenoviral mediated gene-based medicine (ad.HSV tk) given by multiple injections into the healthy brain tissue of patients following surgical removal of the solid tumour mass. In the following days, ganciclovir, is given intravenously. Once treated, healthy brain cells surrounding the site where the tumour was removed express the enzyme thymidine kinase. This converts the ganciclovir to a substance which specifically kills dividing cells. The healthy neurones surrounding the tumour in the brain are non-dividing and are therefore not susceptible to this substance. In this way Cerepro® harnesses healthy brain cells to help prevent a new tumour from growing.

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