Ark’s groundbreaking gene therapy filed in Europe
pharmafile | December 9, 2008 | News story | Sales and Marketing |Â Â Ark, CereproÂ
Cerepro, Ark's gene-based therapy for the brain cancer malignant glioma, has been filed with the European medicines agency.
The drug uses groundbreaking gene technology to help healthy cells fight the cancer cells, and could significantly extend the lives of patients.
Cerepro was rejected by the EMEA in 2005 because of a lack of data but, after extensive preliminary discussions with the regulator, Ark is now confident the drug could be approved in 2009.
Malignant glioma is a fatal brain tumour for which there are currently few treatment options. The current standard therapy for patients with operable tumours involves surgically removing the solid tumour mass and then using radiotherapy and/or chemotherapy to extend life.
Most patients still die within one year of diagnosis, with average survival being about eight months, and Ark's gene-based treatment offers new hope.
Dr David Eckland, research and development director at Ark, said: "We are working in a breakthrough area of medicine, and in a terrible disease where new treatments are very much needed. This is an exciting time and we look forward to working with the EMEA during the review."
How it works
Cerepro works by harnessing healthy cells to produce the substances needed to target and destroy newly growing cancer cells.
A small phase III trial carried out in 2000 showed Cerepro extended average survival times by seven months compared to standard care.
The treatment is comprised of a gene encased in a virus 'shell' or 'vector'. Vectors transfer their gene 'payload' into target cells, which use this new genetic material to produce a protein called thymidine kinase (TK).
Another drug, ganciclovir, is then given intravenously. The healthy brain cells surrounding the tumour site express TK, which converts ganciclovir to a substance which specifically kills dividing cells.
Nearby healthy brain neurones are non-dividing and are therefore not affected by the treatment.
"We are very pleased to report this filing in accordance with our plans," said Dr Nigel Parker, chief executive of Ark. "Increasingly we see that gene based medicine has the potential to deliver solutions for many diseases that are untreatable today, and Ark has become recognised as a world leader in this area of medicine."
Ark has its origins in ventures launched in the mid-1990s by Professor John Martin and Stephen Barker of UCL in London and Professor Seppo Yla-Herttuala of the AI Virtanen Institute at the University of Kuopio, Finland. The company's operations are split between the UK and Finland and all of the founders play leading roles in its R&D programmes.
There are approximately 16,000 cases of malignant glioma in the EU that are operable and Cerepro has Orphan Drug Status in Europe and the US.
The product is manufactured by Ark in Finland, where it was a major undertaking for the company to gain Good Manufacturing Practice (GMP) approval for manufacturing the pioneering technology.
Finland's National Agency for Medicines (NAM) recently renewed Ark's licence to produce gene-based medicine for commercial supply in Europe, and allowed Ark to double production capacity and greatly increase its manufacturing flexibility.
Ark's first Finnish facility was approved in 2005 as the first ever site allowed to manufacture adenoviral gene-based medicines for commercial supply in Europe.
