With the green light from the FDA over the weekend, Rocket Pharmaceuticals is officially on the commercial track.
Still, the company is taking a more cautious approach to launching its new gene therapy drug, Cressradi, to save fuel for future commercial forays into cardiovascular space.
On Friday morning, Roquette announced that the FDA has granted accelerated approval for its hematopoietic stem cell-based gene therapy drug Cressradi (also known as marnetegragen autotemsel) to treat certain children with severe leukocyte adhesion deficiency-1 (LAD-1).
Given the accelerated nature of the approval, the clinical benefits of Cresradi will need to be confirmed through evaluation of long-term follow-up data for treated patients both in ongoing trials and in post-market registries, Lockett said in a March 27 press release.
According to the company, the FDA’s current assessment is based on increased neutrophil CD18 and CD11a surface expression in Rocket’s Kresladi data.
Meanwhile, along with approval, Rocket has also secured priority review vouchers for rare pediatric diseases from regulators, which the company plans to sell to support its research and development ambitions.
The FDA approval marks the first gene therapy for patients with severe LAD-1 due to biallelic mutations in the ITGB2 gene and marks Rocket’s first commercialization. But Rocket isn’t counting on Mr. Kresradi to be a big money maker, and CEO Gaurav Shah, M.D., said on an investor call Friday that the company would carry out a “minimum viable ramp-up” of the therapy.
This meant that Rocket could make Kresladi available to patients and physicians who wanted it, while avoiding more intensive marketing efforts and significant investment in commercialization, and instead saving its commercial capital for the gene therapy developer’s emerging cardiovascular focus.
LAD-1 is an ultra-rare genetic disease caused by mutations in the ITGB2 gene, which encodes the protein CD18, Lockett explained in a statement. The company says critically ill patients often have “very reduced” expression of CD11a, and infants with the disease suffer from recurrent, life-threatening bacterial and fungal infections.
Until now, the standard treatment for severe LAD-1 has relied on allogeneic hematopoietic stem cell transplantation, which comes with its own significant risks, including graft-versus-host disease, Shah said.
As for the target market for gene therapy, it is estimated that about 25 children are born with LAD-1 in the United States each year, and about two-thirds of that group typically have severe symptoms, Salvani Chaudhry, Rocket’s chief commercial and medical officer, added by phone.
Nevertheless, given the complexities inherent in delivering a gene therapy like Cressradi, Roquette expects the number of patients treated annually with the drug to “remain in the single-digit range, including in the years after launch,” Chaudhry said.
In terms of launch timing, Roquette expects patients to be able to enroll in Cressradi treatment in the fourth quarter of this year, giving the company several months to ramp up “product supply readiness” with external manufacturers, coordinate commercial treatment processes, and bring in “a limited number of experienced and qualified treatment centers,” Chaudhuri explained.
“Given the complexity of the treatment and the vulnerability of the pediatric patient population, Cressradi will initially be available in a limited number of specialty centers to support operational excellence and patient safety during early commercialization,” she added.
Chaudhuri noted that the company is not speculating on pricing or access for now and plans to reveal those details closer to the gene therapy’s planned fourth-quarter rollout.
Lockett added that he expects the first patients to be injected with Cressradi by 2027, and that “thus early product revenue” will begin to flow in.
As of the end of 2025, Rocket had approximately $188.9 million worth of cash, cash equivalents and investments on hand, which Mr. Shah said he expects will help accelerate the company’s operations through the second quarter of 2027 and, if the sale of priority examination notes obtained is successful, into 2028.
While Roquette may not have made Cresradii a commercial priority, other players in the field appear keen to greenlight the gene therapy, especially given recent regulatory uncertainty at the FDA.
“This is welcome news for the rare disease patient community and for the cell and gene therapy field in general,” Tim Hunt, CEO of the Alliance for Regenerative Medicine, said in a statement Friday.
“I hope that this approval, coupled with the search for a new director at CBER, begins a new patient-centered chapter at the FDA,” he continued, referring to the recent controversy surrounding the FDA’s soon-to-be-departed head of biologics and gene therapy, Dr. Vinay Prasad.
He added, “For the currently stalled CGT program for rare diseases, FDA should honor its existing collaboration with companies, take advantage of the regulatory flexibility granted by Congress, and grant advisory committee meetings to companies that request them.”
Roquette initially sought FDA review of Cressradi’s LAD-1 indication in late 2023, but the regulator declined, citing the need for additional information regarding the manufacturing of gene therapy drugs.
Meanwhile, Rocket made a major change in trajectory last summer after a patient died in one of its gene therapy trials in early 2025.
In late July, the company announced it would cut its workforce by 30%, narrow its development focus to cardiovascular disease and set aside much of its hematology focus. The move was part of a broader prioritization of the adeno-associated virus platform.
Regarding Cresradi, Jefferies analysts said in a note issued Friday morning that while the launch itself represents only a “small” commercial opportunity for Rocket, the approval de-risks the company’s gene therapy platform more broadly and positions it well for future product approvals.
