BMS grabs key approval for Reblozyl in MDS

Bristol-Myers Squibb's blockbuster aspirations for Reblozyl have been given a boost by the FDA after it approved the drug as a first-line treatment for anaemia in patients with myelodysplastic syndrome (MDS), a rare group of bone marrow cancers.

This is the third indication for Reblozyl (luspatercept), which is already used to treat anaemia in patients with rare blood disorder beta thalassaemia and as a second-line therapy for MDS patients who don't respond to conventional treatment with erythropoiesis-stimulating agents (ESA) like epoetin.

Approval in previously untreated, lower-risk MDS patients who may need red blood cell transfusions represents a big step up in the eligible patient population for the drug and has been viewed as central to BMS' objective of making $4 billion or more in peak sales.

Approximately 30,000 patients are diagnosed with MDS each year in the US, with 70% or more classified as having lower-risk disease. Among these, around two-thirds are thought to be at risk of needing blood transfusions.

The green light has been given largely on the strength of the phase 3 COMMANDS study, reported at ASCO earlier this year, which showed that the drug was able to achieve clinically meaningful improvements in haemoglobin levels and reduce the reliance on transfusions when used first-line.

Reblozyl was found to be superior to standard ESA therapy with Amgen and Johnson & Johnson's Epogen/Procrit (epoetin alfa) at achieving those objectives, with almost 59% of patients on BMS' drug transfusion-free at 24 weeks, compared to 31% of the control group.

It also offers less frequent dosing, given as a subcutaneous injection once every three weeks, versus a once-weekly injection with epoetin.

Moreover, while Reblozyl was only cleared for use as second-line therapy in MDS patients with ring sideroblasts – blood cells with a characteristic circle of iron deposits around their nucleus – the new first-line approval allows the drug to be used regardless of ring sideroblast status.

While most of the benefit of the drug was seen in patients with ring sideroblasts, it performed as well as the ESA in those without the distinctive cells, according to results published earlier this year in medical journal The Lancet.

"For patients with lower-risk MDS, current standard therapies, including ESAs, have provided limited benefit in controlling anaemia, with only one in three patients responding for a duration of six-18 months," said Guillermo Garcia-Manero of MD Anderson Cancer Centre, the lead investigator in COMMANDS.

The results "showed nearly twice as many patients treated with Reblozyl achieved transfusion independence of at least 12 weeks and concurrent haemoglobin increase compared to epoetin alfa," he added.

BMS is also testing Reblozyl in the phase 3 INDEPENDENCE trial as a treatment for anaemia in patients with myelofibrosis already being treated with JAK inhibitor drugs.

Patients with lower-risk MDS could see another new therapy approved by the FDA in the coming months if the regulator approves Geron's telomerase inhibitor imetelstat. A verdict on that is due by next June, after an advisory committee meeting.