EMEA gives go-ahead to first advanced therapy
This article was originally published in RAJ Devices
A cell-based medicine for repairing knee damage has become the first product to be recommended for approval by the European Medicines Agency under the European Union’s new advanced therapy regulatory framework1.
ChondroCelect, which is produced by the Belgian biomedical company TiGenix, is used to repair defects in the cartilage of the femoral condyle (the end of the thighbone) in the knee. It consists of chondrocytes taken from a healthy region of the patient’s cartilage that are grown outside the body and then reinserted during surgery.
Under the regulatory framework for advanced therapy medicinal products (Regulation (EC) No 1394/2007), products based on gene and somatic cell therapy and tissue engineering can be evaluated by the EMEA’s new Committee on Advanced Therapies. The CAT’s opinion is then confirmed by the agency’s main scientific committee, the CHMP.
ChondroCelect is the first product to be given a positive opinion under this new system. The opinion now has to be turned into a centralised EU marketing authorisation by the European Commission, a process that normally takes two to three months.
According to the EMEA, ChondroCelect allows single symptomatic cartilage defects to be repaired and functional cartilage restored, with the aim of reducing the risk of osteoarthritis of the knee over the longer term. The therapy has some possible side effects, including arthralgia, cartilage hypertrophy, and joint crepitation and swelling. The product was originally submitted for approval in mid-2007, based on a 118-patient study comparing it with the current standard treatment, microfracture, although the EMEA evaluation process was held up when the company had to answer some questions raised by the agency towards the end of 2008.
Risk management plan required
As part of the approval process, TiGenix must submit a risk management plan for ChondroCelect with a series of measures including further studies to ensure that safety and efficacy are “robustly” followed up once the product is launched.
Once EU approval has been granted, the product will be launched gradually in Europe, starting probably with Germany, the UK and France, said TiGenix spokesman Kris Motman2. The EU data will also be used to seek approval of the product in the US, he added.
Wilfried Dalemans, head of regulatory affairs at TiGenix, said obtaining regulatory approval of the first ATMP product in Europe “shows that advanced cell therapy products can be developed according to the medicinal product regulatory requirements”3.
References
1. EMEA press release, 26 June 2009, www.emea.europa.eu/humandocs/PDFs/EPAR/chondrocelect/39474109en.pdf
2. Personal communication, TiGenix, 26 June 2009
3. TiGenix press release, 26 June 2009, www.tigenix.com/download/090626_TiGenix_Press_Release_Pos_CHMP_opinion_EN.pdf