Bristol Myers dusts off an old Celgene chemotherapy for use in children with a rare blood cancer
Bristol Myers Squibb is bringing new life to a chemotherapy drug from the old Celgene pipeline as it touts another approval in a rare form of blood cancer affecting young children.
The FDA on Friday approved Vidaza (chemically known as azacitidine) for pediatric patients 1 month and older with newly diagnosed juvenile myelomonocytic leukemia (JMML). The new indication marks the first approval for Vidaza in more than a decade, though it’s commonly used to treat acute myeloid leukemia and myelodysplastic syndromes (MDS).
JMML occurs when white blood cells called monocytes and myelocytes don’t mature properly, causing bleeding and bruising, joint pain (often mistaken for “growing pains”), anemia and difficulty breathing, among other symptoms. Most patients are diagnosed when they’re younger than 4 years old, according to St. Jude Children’s Research Hospital, though it’s extremely rare — only 1 or 2 cases out of a million children are reported each year.
BMS evaluated Vidaza in an open-label study with 18 JMML patients prior to hematopoietic stem cell transplantation (HSCT). The patients received the intravenous drug daily on days 1 through 7 of a 28-day cycle, which was repeated a minimum of three times and a maximum of six times.
Half of the children saw a confirmed response to the drug, three of whom achieved clinical complete remission and six of whom achieved partial remission. The median time to response was 1.2 months, according to the FDA. Nearly all of the patients (94%) received HSCT, the median time to which was 4.6 months.
The most common side effects were fever, rash, upper respiratory tract infection, and anemia.
Celgene acquired Vidaza in its $2.9 billion buyout of Pharmion back in 2007. At its peak, the drug brought Celgene $600 million per year, before it ended up in BMS’ pipeline in 2019. A year later, BMS snagged a win for an oral version of the drug — known as Onureg — in adults with acute myeloid leukemia.
While that remains Onureg’s only other approved indication, the drug is in ongoing studies for angioimmunoblastic T-cell lymphoma and lower-risk MDS, according to BMS’ website.
Gilead ran into trouble earlier this year when the FDA slammed the brakes on enrollment in trials that combined the CD47 drug magrolimab with Vidaza. The company said regulators flagged an “apparent imbalance” in the suspected unexpected serious adverse reactions between study arms.
A month later, Gilead revealed that the agency had expanded the hold to its diffuse large B-cell lymphoma and multiple myeloma studies.
The FDA lifted its partial hold on studies for magrolimab and azacitidine in April, and Gilead said it’s on track for an interim readout in 2023.