FDA Approves Enzyvant’s Rethymic for Pediatric Congenital Athymia

  • RETHYMIC by Enzyvant Sciences becomes the first and only therapy that is approved by the US Food and Drug Administration for Congenital Athymia.
  • Enzyvant’s RETHYMIC is indicated for use in patients with an Ultra Rare Pediatric Disease for immune reconstitution.

On October 8, 2021, Enzyvant Sciences received the approval of RETHYMIC for immune reconstitution in pediatric patients diagnosed with Congenital Athymia, a type of rare immune disorder. With this, Rethymic becomes the first and only treatment option for such an ultra-rare pediatric disease.

Children diagnosed with Congenital Athymia typically die by age two or three with only supportive care. This is because such patients are born with no thymus. The thymus is an essential component of the body’s defense system. It is the primary specialized organ of the lymphatic system that is located in the upper part of the chest. This organ plays a vital role in protecting the body from infections. Thus, individuals born without the thymus experience immune dysregulation and severe immunodeficiency.

RETHYMIC is an allogeneic processed thymus tissue-agdc. It is a one-time regenerative tissue-based therapy for patients with Congenital Athymia. Ten prospective open-label clinical (prospective single-arm) studies were used to determine the efficacy of Rethymic. 105 patients were enrolled from the year 1993 to 2020 and surgically implanted with this tissue-agdc under the IRB-approved (Institutional Review Board) protocol. The EAS (Efficacy Analysis Set) included around 95 patients whereas the Safety Analysis Set included 105 patients. Significantly, the longest follow-up period was 25.5 years. In the EAS, the estimated survival rates were 77% and 76% at one year and two years respectively. The median follow-up period was 10.7 years in patients who lived more than a year after the implantation with an estimated long-term survival rate of 94%. Moreover, during the first two years after transplantation, there was a notable reduction in the number of infections (p<0.001).

View full prescribing information for RETHYMIC.