Australian stem cell and regenerative medicine company, Cynata Therapeutics (ASX:CYP), says it has received written advice from the FDA regarding the regulatory approval path for its Cymerus mesenchymal stem cell (MSC) products.
The company said the advice follows a pre-Investigational New Drug meeting it recently held with the FDA’s Office of Cellular, Tissue and Gene Therapies.
"The FDA confirmed that the scope and substance of Cynata’s 'Chemistry, Manufacturing and Controls' (CMC) dossier is commensurate with its expectations, which indicates that Cymerus MSC products are expected to be of suitable quality for clinical trial use in the US," said the company.
Cynata said it received clarification on the design of preclinical studies required to support a US IND. It said it anticipates conducting those studies in parallel with the ongoing clinical trial of CYP-001 for the treatment of graft-versus-host disease (GvHD) being conducted in the UK and Australia. The FDA also provided advice regarding the protocol for a planned GvHD clinical trial in the US.
"Additionally, the FDA clarified that Cynata may submit a request for 'Regenerative Medicine Advanced Therapy' (RMAT) designation for its CYP-001 product to treat GvHD once preliminary results of the world first clinical trial are available, assuming those results support such a request. RMAT designation is an initiative that arose from the 21st Century Cures Act, which recently came into law in the US. The initiative allows companies with RMAT designated products to avail of additional and earlier interactions with the FDA and to seek priority review and accelerated approval," it said.
According to Dr Kilian Kelly, Cynata’s vice president of product development, “The pre-IND meeting was an enormously valuable exercise for Cynata. The outcome of the meeting was very positive, and we are optimistic that we will be able to open an IND and include clinical centres in the US in future trials.
“This will be an important step in the commercial development of CYP-001 in the world’s largest market for pharmaceutical products."