FDA approves Rethymic for pediatric congenital athymia.- Enzyvant

Ten prospective single-arm, open-label studies with patient enrollment from 1993 to 2020 form the basis of the Rethymic data set. One hundred and five patients were surgically implanted with Rethymic under one of 10 Institutional Review Board (IRB)-approved protocols. Ninety-five patients were included in the Efficacy Analysis Set (EAS) and 105 patients were included in the Safety Analysis Set.

Survival rates were analyzed with the longest follow-up period of 25.5 years. In the EAS, Kaplan-Meier estimated survival rates (95% CI) were 77% (0.670–0.841) at one year and 76% (0.658–0.832) at two years. For patients who were alive at one year post implantation, the Kaplan-Meier estimated long-term survival rate was 94% at a median follow-up time of 10.7 years. For the patients in the clinical trials, naïve T-cell levels were measured using flow cytometry at six, 12, and 24 months after implantation with Rethymic. Patients in the clinical trials started out with very few naïve T cells but naïve CD4+ and CD8+ T cells began to reconstitute over the first year, with a durable increase through year two. Reductions in the number of infections over time during the first two years after treatment were statistically significant (p<0.001).></0.001).>