Reblozyl has stumbled on its quest to reach a wider pool of blood disorder patients.
Bristol Myers Squibb is pulling the plug on Reblozyl’s bid to treat anemia in adults with non-transfusion-dependent (NTD) beta thalassemia, the company said Friday. The application withdrawal comes after the company failed to answer the FDA’s questions about the med’s benefit-risk profile in the patient population outlined in BMS’ phase 2 Beyond trial.
BMS did not specify what the FDA’s concerns were.
“Chronic anemia puts patients at significant risk for long-term clinical complications, and we’re continuing to evaluate Reblozyl in a broad clinical development program to bring this important therapeutic option to additional patients,” a BMS spokesperson told Fierce Pharma via email.
She pointed to the med’s ongoing studies like the phase 3 Commands trial in ESA-naïve, lower-risk patients with myelodysplastic syndromes, as well as the late-stage Independence trial, which is looking at Reblozyl in patients with myelofibrosis and anemia, who are on JAK2 inhibitor therapy and require red blood cell transfusions.
The midstage Beyond study pitted Reblozyl against placebo in beta thalassemia patients who don’t require frequent blood transfusions, BMS said in a release. The trial randomized 145 subjects 2:1 to receive Reblozyl versus placebo. Alongside BMS’ drug or a dummy med, patients were eligible to receive best supportive care, including red blood cell transfusions, iron-chelating agents, antibiotics, antivirals and antifungals, plus nutritional support as needed, BMS said.
The study is assessing Rebloyzyl’s ability to boost patients’ mean hemoglobin values over a continuous 12-week stretch from week 12 to week 24 of treatment in the absence of transfusions. Secondary endpoints include patient-reported outcomes on metrics of tiredness and weakness, plus baseline hemoglobin.
While an NTD beta thalassemia indication is now off the table in the U.S., BMS will continue to study Reblozyl in a “broad clinical development program” to further support patients living with anemia, Noah Berkowitz, M.D., Ph.D., senior vice president, hematology development at BMS, said in a statement.
BMS picked up Reblozyl, also known as luspatercept-aamt, in its massive $74 billion Celgene buyout. The drug bagged its first FDA approval back in Nov. 2019 to treat anemia in patients with beta thalassemia who need regular red blood cell transfusions.
In August of the following year, Reblozyl cruised past a green light to treat anemia in myelodysplastic syndrome (MDS) patients with ring sideroblasts, a low-risk subtype of the blood cancer where red blood cells house a ring of excess iron.
Reblozyl is among a quartet of new launches BMS expects to reach peak sales of more than $4 billion alone or in combinations, the company said earlier this year at the 2022 JP Morgan Healthcare Conference. Those new products should help—in part—to offset the sting of Revlimid generics.
Editor's note: This story has been updated to include comments from BMS.