Rocket Pharma has been cleared by U.S. regulators to begin selling a gene therapy for a rare and deadly immune disorder in a needed lift for a company that’s lost most of its market value over the last five years.
The Food and Drug Administration granted an accelerated approval to Rocket’s therapy, Kresladi, for use in treating severe leukocyte adhesion deficiency Type 1, a disease that weakens the body’s ability to fight off pathogens. Infants born with it are often struck with bacterial or fungal infections and hospitalized. They typically need a bone marrow transplant to survive. Kresladi is indicated for children whose disease is caused by specific genetic mutations and cannot get a transplant.
In testing, Kresladi showed the potential to keep patients alive, out of the hospital, and dramatically reduce the rate of infections they struggle to keep at bay. Anemia, low platelet and white blood cell counts and mouth sores were among the most common side effects observed. The therapy’s benefits must be confirmed through longer-term follow-up in an ongoing study, according to Rocket.
In a statement from the FDA, Vinay Prasad, the outgoing director of the agency office regulating gene therapies, called Kresladi a “breakthrough treatment” for LAD-I. He also noted how the agency, which has been closely scrutinized of late for controversial decisions involving rare disease therapies, “continues to exercise significant regulatory flexibilities as applicable.”
“For rare diseases, the FDA considers small patient populations in clinical trials and all available sources of evidence to advance life-changing treatments while still meeting its rigorous scientific standards,” Prasad said.
The clearance marks the transition to a commercial-stage company for Rocket, which was once one of the industry’s most valuable gene therapy developers before losing most of its value amid a series of setbacks.
Rocket sported a market capitalization exceeding $3 billion in 2021 and was advancing a group of “ex vivo” treatments built from patient stem cells as well as “in vivo” therapies administered via infusions.
Rocket has since had a rough run, though. Manufacturing issues led the FDA to reject Kresladi in 2024. Safety concerns then slowed a closely watched program for a rare heart condition, leading to layoffs, a pipeline restructuring, and a decision to pull a planned application for a blood disease treatment. Those setbacks have sent shares once worth over $60 free falling to around $4 apiece. They’ve also “undermined confidence in execution,” wrote Leerink Partners analyst Mani Foroohar, last November.
Kresladi isn’t seen as a big seller. LAD-1 is estimated to affect about 1 in every 100,000 to 200,000 live births in the U.S., and roughly two-thirds of those affected are diagnosed with the disease’s “severe” form. Analysts at William Blair have estimated that, at peak, the therapy might generate $294 million in yearly sales. Rocket has also recently prioritized its “in vivo” gene therapies over more complex treatments like Kresladi.
Still, with the approval, Rocket has won a “priority review” voucher that could be sold for nine figures, extending its ability to operate beyond the second quarter of 2027. That prospective funding has been seen as critical by Wall Street analysts, as it’ll enable Rocket to support the rest of its pipeline — particularly the cardiovascular program that’s now in pivotal testing and is considered a potentially lucrative product.
Rocket is evaluating its options to “monetize” the voucher in a way that can “enhance financial flexibility” as well as shareholder value, the company said in a statement.
