- The Food and Drug Administration on Thursday approved AstraZeneca's Lumoxiti for patients with relapsed or refractory hairy cell leukemia who have received two or more previous systemic therapies.
- Lumoxiti is a CD-22-directed cytotoxin, and is the first of this type of drug to win approval for the blood cancer. The green light from the FDA was based on data from a Phase 3 study of 80 patients, which showed about a third of patients experienced a durable complete response.
- Lumoxiti, which received Fast Track, Priority Review and Orphan Drug designations from the FDA, will carry a boxed warning for capillary leak syndrome.
Hairy cell leukemia is a rare, chronic and slow-growing blood cancer that affects around a thousand new patients in the U.S. each year. Roughly a third of patients will relapse, and few treatments are available for relapsed or refractory disease. AstraZeneca's Lumoxiti (moxetumomab pasudotox-tdfk) is the first new treatment in more than 20 years, the drugmaker said.
"While many patients with hairy cell leukemia experience a remission with current treatments, 30% to 40% will relapse five to ten years after their first treatment," said Robert Kreitman, head of clinical immunotherapy section, Center for Cancer Research, National Cancer Institute, in a statement provided by AstraZeneca. "With subsequent treatments, durations of response diminish and toxicities accumulate."
While this is a small indication, Steve Scala of Cowen suggests potential sales could eventually total "a couple hundred million" in a note to investors.
Lumoxiti is part of AstraZeneca's small, but growing, hematology franchise. The pharma regards Calquence (acalabrutinib), which gained approval in the U.S. in November last year, as a "cornerstone" hematology drug.
Through the first six months of 2018, Calquence had reached $20 million in U.S. sales, and expansions are planned out into chronic lymphocytic leukemia.