An FDA rejection leaves families reeling
Katie Luckesen thought her life was going to change Wednesday. A treatment for the rare and deadly pediatric athymia was expected to win FDA approval, news that would mean her 2-year-old son, Charlie, could receive a potentially life-saving therapy.
As STAT’s Eric Boodman reports, that’s not what happened. Enzyvant, the company developing the treatment, said yesterday that the FDA had rejected its application over concerns about manufacturing. The future of the treatment is now uncertain.
Enzyvant’s would-be product is a tissue-based therapy for pediatric athymia, a condition in which children are born without a thymus and thus unable to produce infection-fighting T cells. The process involves removing a segment of thymus from a baby, washing out the immune cells, and then implanting the tissue into a kid with congenital athymia.
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As STAT’s Eric Boodman reports, that’s not what happened. Enzyvant, the company developing the treatment, said yesterday that the FDA had rejected its application over concerns about manufacturing. The future of the treatment is now uncertain.
Enzyvant’s would-be product is a tissue-based therapy for pediatric athymia, a condition in which children are born without a thymus and thus unable to produce infection-fighting T cells. The process involves removing a segment of thymus from a baby, washing out the immune cells, and then implanting the tissue into a kid with congenital athymia.
Read more.
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