Ark still on track to register gene therapy despite EMEA reservations

06 Jan 2010 | News
Ark Therapeutics has filed a formal request for a re-examination of its application to market Cerepro, its gene-based product for brain cancer.

Ark Therapeutics Group plc has filed a formal request for a re-examination of its application to market Cerepro, its gene-based product for brain cancer and believes it is still on track to be the first company to get approval for a gene therapy product in a regulated market.

The re-filing took place earlier this week after the European Medicines Agency (EMEA) said in mid-December that it would not grant approval because it believed investigators in the trial may have been biased in favour of Cerepro.

But critically for Ark, there were no objections to any of the underlying gene delivery technology, or to the manufacturing of the product. For the company that is a leader of Europe’s charge into standardised, off-the-shelf gene therapy that was significant hurdle to have cleared.

Nigel Parker CEO pointed out the file was deemed approvable in all aspects of chemistry, manufacturing and environmental control. “We are pleased that Ark’s underlying adenovirus platform has cleared all other barriers to a full approval. This is a major milestone for [us] and the industry,” he said. Ark has other products in development that are based on the adenovirus platform.

But the evidence for clinical efficacy of Cerepro was questioned by EMEA. The endpoint in the trial was the time from receiving standard treatment or standard treatment plus Cerepro, to the first re-intervention. The trial was not blinded and EMEA said clinical investigators may have shown bias in waiting longer to re-intervene in those patients they knew to have been treated with Cerepro.

However, Ark says that scans show the volume of recurrent tumours at the point of first re-intervention were similar in active and control patients. This would indicate there was no bias since both groups were treated again at the same stage of disease progression.

Parker said, “In the last two weeks we have assessed the full range of evidence from the Phase III study. We are confident that we have the necessary data to address the main underlying objection concerning the re-intervention decision in the Cerepro trial.  The body of evidence is substantial and robust and we are thus comfortable to file for re-examination.”

“Ark has successfully pioneered the adenovirus platform to an approvable standard and we are now dealing with a final clinical issue.”

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