First-in-class Cell Therapy for Steroid-refractory Graft Versus Host Disease
The FDA approved the mesenchymal stromal cell therapy remestemcel-L-rknd for some pediatric patients as young as 2 months.
The U.S. Food and Drug Administration (FDA) has approved remestemcel-L-rknd (Ryoncil) for the treatment of pediatric patients 2 months of age and older who have steroid-refractory acute graft versus host disease (SR-aGVHD).
Immune-suppressing steroids are the standard first-line treatment for patients who develop GVHD, but they are ineffective for many patients. While another immune-suppressing drug, ruxolitinib (Jakafi), was greenlit in 2019 for patients 12 years of age and older with SR-GVHD, it is not approved for younger patients.
Remestemcel-L-rknd is a mesenchymal stromal cell (MSC) therapy. MSCs secrete anti-inflammatory cytokines that can lessen the inflammation caused by aGVHD. Remestemcel-L-rknd consists of MSCs harvested from the bone marrow of a healthy donor that are then infused into patients with aGVHD.
This is the first FDA approval for remestemcel-L-rknd, and remestemcel-L-rknd is the first FDA-approved MSC therapy.
The decision was based on results from the multicenter, prospective, single-arm, phase III MSB-GVHD001 clinical trial. Researchers enrolled and treated 54 pediatric patients who developed aGVHD following a hematopoietic stem cell transplant and whose aGVHD progressed within three days or did not improve within seven days of treatment with remestemcel-L-rknd. Patients with grade B, C, or D SR-aGVHD were eligible, except for patients with grade B SR-aGVHD only involving the skin.
Thirty percent of patients experienced a complete response and 41% of patients experienced a partial response by day 28 of treatment. Responses lasted a median of 54 days after they were evaluated on day 28.
The recommended dose of remestemcel-L-rknd is 2×106 MSCs/kg body weight, administered as an intravenous infusion twice a week for four consecutive weeks. Based on response after four weeks, some patients may continue treatment.
aGVHD is a condition in which donor immune cells from a stem cell transplant, including those used to treat some types of blood cancer, recognize the recipient’s body as foreign and attack healthy tissues. Unlike chronic GVHD, it is typically diagnosed within three months of the stem cell transplant. Estimates of aGVHD incidence vary based on many clinical and demographic factors with upper estimates of around 40% of patients who receive stem cell transplants.
The FDA rendered its decision on December 18, 2024. Check this resource for updated information on all therapeutics regulated by the FDA.
