In December 2020, CombiGene held separate meetings with the Swedish Medicines Agency and its UK counterpart MHRA (Medicines & Healthcare products Regulatory Agency). The meetings took place after the two authorities reviewed documentation regarding the current status of CombiGene’s epilepsy project CG01 with regard to the preclinical program and establishment of the manufacturing platform, as well as the design of the planned studies in toxicology and biodistribution. In addition, CombiGene had also submitted a preliminary version of the design of the first clinical trial.
Both authorities have now confirmed CombiGene’s approach to the studies in toxicology and biodistribution, which means that the company can proceed as planned without changes in the design of the studies. The two studies are expected to start as soon as the produced CG01 material has undergone the normal quality review. The authorities also gave valuable advice regarding the future GMP production and the first clinical study. CombiGene will now continue discussions with clinicians interested in being involved in the first clinical study scheduled to begin in 2022. The authorities also highlighted the possibility of further meetings for more guidance when CombiGene has prepared a more definitive study protocol for the clinical study.
“I am very pleased with the discussions with the two pharmaceutical authorities”, says Karin Agerman, Chief Research and Development Officer at CombiGene. “I am also very pleased about the possibility of continued dialogue with the authorities regarding the clinical study.”
About CG01
CG01 is a gene therapy developed to treat drug-resistant focal epilepsy. Every year, approximately 47,000 drug-resistant patients with this type of epilepsy are estimated to be added in the US, EU4, UK, Japan and China. CombiGene believes that it is realistic that 10-20 percent of these patients could be treated with the company’s gene therapy. The global market for drug candidate CG01 is estimated at USD 750 –1 500 million annually.