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Recently, the US Food and Drug Administration (FDA) approved Enzyvant Sciences' Rethymic (Allogeneic Treated Thymus Tissue-agdc) for the treatment of congenital athymic children
.
Rethymic therapy is the result of more than 25 years of research aimed at improving the survival rate of children who had little hope of treatment before
.
The approved Rethymic therapy is a one-time tissue-based regenerative therapy
Congenital athymia is an extremely rare disease.
Children of this type have no thymus at birth
.
The disease can lead to life-threatening immune disorders
Rethymic is composed of allogeneic thymus tissue obtained from a donor.
After processing and culture, it is transplanted into the patient's body to help rebuild immunity
.
The most common side effects of Rethymic therapy are hypertension, cytokine release syndrome, low magnesium levels, skin rash, low platelets, and graft-versus-host disease
In fact, Rethymic's regulatory application has not been smooth sailing
.
In 2019, the FDA issued a complete response letter (CRL) to the treatment regarding manufacturing issues
It is worth noting that this therapy is not a quick cure and effective
.
The FDA pointed out that it takes at least six months or more to rebuild the immune function of children with congenital athymus, and strict prevention of infection is required in the process
Currently, the market pricing and availability of the therapy have not yet been announced, but BioWorld reports that the listing price of Rethymic may be quite expensive.
On the one hand, the number of children with the disease is small, and on the other hand, it provides support for children who are born without thymus.
The cost of care is higher
.
In August, the company pointed out that each child with this disease spends an average of 150.
Reference source:
Reference source:FDA Greenlights Enzyvant Drug for Ultra-Rare Pediatric Disease
FDA Greenlights Enzyvant Drug for Ultra-Rare Pediatric Disease
