The U.S. Food and Drug Administration authorized Ryoncil (remestemcel-L-rknd), an allogeneic (donor) bone marrow-derived mesenchymal stromal cell (MSC) therapy in order to steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients 2 months of age and older. The application received Orphan Drug, Fast Track and Priority Review designations by the FDA.
Ryoncil is the first MSC treatment to receive FDA approval. It contains MSCs, a type of cell that can differentiate into a variety of other cell types and play a variety of roles in the body. The bone marrow of healthy adult human donors is where these MSCs are extracted.
Steroid-refractory acute graft-versus-host disease is a serious and life-threatening condition that can occur as a complication of allogeneic hematopoietic (blood) stem cell transplantation (allo-HSCT). In allo-HSCT, a patient receives hematopoietic stem cells from a healthy donor to replace their own stem cells and form new blood cells, a procedure often done as part of treatment for certain types of blood cancers, blood disorders or immune system disorders.
The safety and effectiveness of Ryoncil were evaluated in a multicenter, single-arm study in 54 pediatric study participants with SR-aGVHD after undergoing allo-HSCT. Ryoncil’s effectiveness was based primarily on the rate and duration of response to treatment 28 days after initiating Ryoncil. Sixteen study participants (30%) had a complete response to treatment 28 days after receiving Ryoncil, while 22 study participants (41%) had a partial response.
Infusion of Ryoncil should be monitored by the treating physician, and the infusion should be discontinued if there is any evidence of a reaction which may include dyspnea (shortness of breath), hypotension (low blood pressure), fever, tachypnea (rapid breathing), cyanosis (blue discoloration of skin, lips or nails) and hypoxia (low oxygen in the blood). The most common adverse reactions in study participants who received Ryoncil were infections, fever, hemorrhage, edema, abdominal pain and hypertension. Complications such as hypersensitivity and acute infusion reactions, transmission of infectious disease or agents and ectopic tissue formation may occur following treatment with Ryoncil. Ryoncil is contraindicated in patients with known hypersensitivity to dimethyl sulfoxide or porcine and bovine proteins. Patients should be premedicated with corticosteroids and antihistamines prior to infusion and monitored for hypersensitivity reactions during treatment with Ryoncil.
