The U.S. Food and Drug Administration announced that it has approved Rethymic (allogeneic processed thymus tissue-agdc) from Cambridge, Massachusetts-based Enzyvant Therapeutics, Inc., for the treatment of pediatric patients with congenital athymia, a rare immune disorder.
According to the FDA, Rethymic is the first thymus tissue product approved in the U.S.
“Today’s action marks the first FDA approval of a therapy to treat this very rare and devastating disease in children,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research in a prepared statement. “We remain committed to helping advance the development of safe and effective medical products for patients affected by rare diseases – an area of such critical need.”
Children with congenital athymia are born without a thymus, an organ that plays a crucial role in helping the body learn to fight infections.
Congenital athymia is associated with several genetic and syndromic disorders including heart defects such as DiGeorge Syndrome.
Children affected by this disease usually die within the first two years of life and may have repeated life-threatening infections because they lack adequate working T cells.
In a press release, the FDA said: “The safety and efficacy of Rethymic were established in clinical studies that included 105 patients, with ages from one month to 16 years, who each received a single administration of Rethymic, from 1993 to 2020. Rethymic improved survival of children with congenital athymia, and most children treated with this product survived at least two years. Children treated with Rethymic who survive past the first year generally survive long-term. Rethymic also reduced the frequency and severity of infections over time.”