EMA grants CVD therapy ATMP certificate

German biopharma company, t2cure, has had its stem cell-based therapy for cardiovascular disease certified by the European Medicines Agency (EMA). The company says its t2c001 lead product is the first to be quality certified by EMA under its regulations for Advanced Therapy Medicinal Products (ATMPs).

ATMPs are advanced biotechnology products based on cell therapy, gene therapy, or tissue engineering and t2c001 is a somatic cell therapy product derived from autologous bone marrow.

"We are excited that our lead product has been confirmed to meet the rigorous quality requirements set by the EMA, the regulatory body that will decide on a future European market approval for our product," commented Dr Petra Rueck, CEO of t2cure.

"Together with upcoming five-year efficacy and safety data from our phase II trial, the EMA certification shows that we are on the right track to bring this groundbreaking therapy closer to the market, and to patients."

Rueck continued: "This achievement also proves that the regulations set out by the EMA for advanced therapy products and the expertise brought together by the EMA's Committee for Advanced Therapies effectively assist small- and medium-sized companies in developing advanced therapy products. These products offer novel treatment opportunities for patients who suffer from diseases, including orphan diseases, for which there are limited or no treatment options." 

Because of the novelty and complexity of ATMPs, evaluating their quality, safety, and efficacy often requires approaches that go beyond those needed for conventional medicines.

As a result, European Regulation 1394/2007 provides a consolidated framework for this innovative class of products, including a procedure allowing small- and medium-sized companies to apply voluntarily for certification of the pharmaceutical quality and the pre-clinical data of an ATMP. 

While the certification procedure is independent from a Marketing Authorisation Application (MAA), it follows the scientific and technical requirements necessary to facilitate a future MAA.