Bristol Myers Squibb's decade-old Vidaza goes oral with FDA nod in acute myeloid leukemia

Bristol-Myers Squibb
Bristol Myer Squibb has won FDA approval for Onureg, an oral drug that shares the same azacitidine ingredient as injectable Vidaza, in acute myeloid leukemia. (Bristol Myers Squibb)(Bristol-Myers Squibb)

Vidaza boasts a long-established place in conventional treatment for patients with acute myeloid leukemia (AML). Now, its active ingredient, azacitidine, has been spun into an oral option.

The FDA greenlighted Onureg, or azacitidine in its tablet form, to treat AML patients who are in remission after initial chemotherapy, but who aren't eligible for curative therapy, such as a stem cell transplant, BMS said Tuesday.

Although they are made with the same active ingredient, Onureg and Vidaza are processed very differently in the body. The new pill can't be directly substituted for intravenous or subcutaneous Vidaza because it could cause fatal adverse reactions.

Onureg’s approval was based on data from the Quazar AML-001 phase 3 trial. Results showed that continued treatment with Onureg in AML patients who showed no sign of cancer after induction chemo significantly cut the risk of death by 31% compared with placebo. Those on Onureg lived a median 24.7 months, while the placebo group showed a median overall survival of 14.8 months.

As a once-daily oral therapy, Onureg boasts a convenience advantage over injectables to control AML, Andrew Wei, the study’s lead investigator, said in a statement.

RELATED: Bristol Myers Squibb's targeted AML drug Idhifa fails to extend patients' lives

Like its injectable counterpart, Onureg patients suffered higher rates of adverse reactions such as neutropenia and thrombocytopenia in clinical trials. The drug is also linked to early death in patients with myelodysplastic syndromes, the only FDA-approved indication for Vidaza. The higher incidence of early death shut down Onureg’s AZA-MDS-003 study in myelodysplastic syndromes.

BMS got its hands on Onureg and Vidaza via its acquisition of Celgene, which itself obtained the drugs through a $2.9 billion takeover of Pharmion in 2008.

The Onureg nod comes on the heels of a clinical flop by Celgene’s targeted AML drug Idhifa. In patients with IDH2-mutated relapsed or refractory AML, adding Idhifa failed to improve survival on top of best supportive care, which may include Vidaza. In the first half of 2020, Vidaza brought in $284 million.