Gamida Cell has scored an expansion for Omisirge, with the FDA endorsing the stem-cell-based therapy to treat severe aplastic anemia (SAA).
The approval, which comes more than two years after Omisirge’s original FDA nod as the first treatment to reduce the risk of infection in blood cancer patients during hematopoietic stem cell transplant, finds Gamida in a very different position. The 27-year-old Israeli company, once public, is now a subsidiary of London-based private firm Ayrmid.
Gamida was cash-strapped when it got Omisirge across the regulatory finish line in April 2023. Eleven months later, its principal lender, Highbridge Capital Management, took Gamida Cell private in a restructuring deal that allowed it to stay afloat. In June 2024, Highbridge reintroduced Gamida as a wholly owned subsidiary of the U.K.'s Ayrmid.
The new Omisirge approval, meanwhile, is based on a single-center study at the National Institutes of Health (NIH), which showed Gamida's cell therapy provided sustained hematopoietic recovery and transfusion independence in 86% of patients.
The treatment also helped patients achieve rapid neutrophil recovery post-transplant at a median of 11 days. There were no cases of severe acute or chronic graft-versus-host disease (GVHD) in the study, Gamida's parent Ayrmid said in an early December press release. Gamida's drug further helped patients log a 92% disease-free and overall survival rate in the trial, Ayrmid added.
“Patients in the study with aplastic anemia were high risk but had significantly better than expected outcomes and demonstrated remarkably fast and high rates of neutrophil engraftment,” Richard Childs, M.D., of the NIH’s National Heart, Lung, and Blood Institute, said in a release, adding that patients “achieved a rapid return to a normal life.”
SAA is a rare, life-threatening hematologic disorder in which the bone marrow fails to produce sufficient blood cells. While stem cell transplant offers a potential cure, many patients lack a matched sibling donor, Ayrmid said.
“This approval is potentially transformative for patients living with SAA, given the rapid and sustained recovery of blood counts observed,” Ayrmid CEO Joe Wiley said in the release.
The company added that in anticipation of the increased demand for Omisirge, it has partnered with third-party manufacturer RoslinCT. Commercial production will begin in 2027 at RoslinCT’s cell therapy manufacturing facility in Hopkinton, Massachusetts, in the U.S.
Gamida's Omisirge isn’t the only product in Ayrmid’s portfolio. In November of last year, the company struck a $97 million licensing deal with BioLineRx to commercialize its cell therapy Aphexda. The treatment, which was endorsed by the FDA in 2023, is used in tandem with colony-stimulating factor (CSF) filgrastim to mobilize hematopoietic stem cells to peripheral blood for collection and autologous stem cell transplant in patients with multiple myeloma.
Earlier this year, Ayrmid made an unsuccessful attempt to buy out bluebird bio for $4.50 per share. The struggling gene therapy specialist opted for Carlyle and SK Capital's competing offer at $3 per share after Ayrmid failed to obtain financing and did not deliver a binding offer after a three-week diligence period.