Two years ago, Megan Selser was folding her 7-week-old infant’s clothes, clutching his fuzzy red head to her chest, when her phone rang. She answered and heard on the other end the pained voice she had used herself many times as an ICU nurse delivering bad news.
Her son Ben, she learned, had mucopolysaccharidosis (MPS) type 2, or Hunter Syndrome, one of a group of rare diseases that is invisible at birth but can, by the time a child is in kindergarten, slowly take his physical and cognitive abilities. Some children die as young as 10.
There was reason for hope, though. Ben was caught early, thanks to a newborn screening pilot project in North Carolina. Doctors explained there were new treatments in clinical trials, including two gene therapies and a protein-based drug. They might alter the disease’s trajectory, if given early enough.
This month, those hopes took a substantial blow. The Food and Drug Administration rejected a gene therapy from Regenxbio, with regulators telling the company it needed to collect more data that could delay an approval by years.
Regenxbio’s MPS drug is one of at least five cell or gene therapies for deadly rare diseases the agency has rejected or seemingly reversed course on over the last year, since the Trump administration installed new leaders. Many advocates and executives are worried the agency may be retreating from the flexible approach regulators have promised in recent years.
