Enzyvant has just announced that they have resubmitted their application of RVT-802 to the FDA as a therapy for pediatric congenital athymia. Their first application was submitted in 2019. The FDA replied with many requests related to chemistry, controls, and manufacturing. Manufacturing control issues were specifically noted in the FDA’s letter.
Enzyvant replied saying that they were aware of all of the notes made by the FDA, and that they didn’t believe they were required for the approval. Nonetheless, the company got to work addressing each and every request from the FDA.
Now that the application has been submitted again, a decision is expected by October 8th of 2021.
This therapy had already received Regenerative Medicine Advanced Therapy Designation as well as Breakthrough Therapy Designation in 2017. Then in 2018, they began their rolling submission of the application for the therapy.
This therapy is a one-time treatment. It is a cultured human tissue-based regenerative therapy. RVT-802 was investigated by researchers at Duke University. A total of 10 clinical trials have brought this therapy where it is today.
Pediatric congenital athymia is considered an ultra-rare condition. At birth, there is an absent thymus. It is suspected that this therapy is due to a genetic mutation but the exact cause is still unknown.
Congenital Athymia causes
- Infection susceptibility
- Immune dysregulation
Unfortunately, there are currently no approved treatments for the condition. Supportive care is the only option for patients. The majority of patients pass from autoimmune issues or infection by 2 to 3 years of age.
This resubmission is a signal of optimism for patient’s families. The researchers emphasize their thanks to the patients and families throughout the trials, as they have truly made this resubmission possible.
You can read more about this therapy and resubmission process here. It could become the first approved therapy for pediatric congenital athymia.