Mesoblast submits application for key drug approval

Cellular medicines developer Mesoblast has resubmitted its BLA for approval of Ryoncil (remestemcel-L) in the treatment of children with SR-aGVHD.

Remestemcel-L is being developed for inflammatory diseases in children and adults, including steroid-refractory acute graft versus host disease, and biologic-resistant inflammatory bowel disease.

It was only in March that the Food and Drug Administration decided that data from its Phase 3 study was sufficient to support a proposed Biologics License Application (BLA) for the drug.

The company’s filing addresses remaining CMC (Chemistry, Manufacturing, and Control) items.

Mesoblast CEO Dr. Silviu Itescu said: “We have worked closely with the agency and thank them for their ongoing guidance, facilitating the potential approval of RYONCIL and addressing the urgent need for a therapy that improves the dismal survival outcome in children with SR-aGVHD.”

The FDA had granted remestemcel-L Fast Track designation, a process to facilitate the development and expedited review of therapies for serious conditions that fill unmet medical needs.

It was also given Priority Review designation, which is given to drugs that treat a serious condition and provide a significant improvement in safety or effectiveness over existing treatments.

The BLA resubmission is expected to have a review period of between two and six months from receipt.

Further reading:
Mesoblast gets FDA go ahead for pediatric drug

Picture: Dr. Silviu Itescu