New Drug Approved for Children with Severe Immune Disease

Remestemcel-L, a new treatment for children ages 2 months and older with steroid-refractory acute graft-versus-host disease (SR-aGVHD), a major complication of donor bone marrow transplantation, has been approved by the U.S. Food and Drug Administration (FDA). The approval marks the first stem cell therapy for the condition and will be marketed under the brand name Ryoncil.

Remestemcel-L is derived from special stem cells taken from a donor’s bone marrow and is given to patients through a series of IV infusions. It helps control the immune system’s response in patients with SR-aGVHD by reducing harmful inflammation and boosting the body’s natural anti-inflammatory processes The drug is also the first-ever FDA-approved treatment for children ages 2 months and older, including adolescents and teenagers.

Approval is based on a clinical trial that tested the safety and effectiveness of Ryoncil in 54 children with SR-aGVHD after a donor stem cell transplant. The children received Ryoncil infusions twice a week for four weeks. After 28 days, 70 percent of them responded to the treatment, with 30 percent showing a complete response (improvement in all affected organs) and 41 percent showing a partial response (improvement in one organ, with either no change or worsening in another organ) to the treatment. Children showing partial improvement received infusions once a week for another four weeks. On average, improvements lasted for 54 days before the condition worsened, a new treatment was needed or the patient died. The most common side effects observed were infections, fever, bleeding, swelling, stomach pain and increased blood pressure. Other complications may include allergic or infusion-related reactions and unusual growth of tissues.