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The US Food and Drug Administration (FDA) has approved GlaxoSmithKline's (NYSE:GSK) novel treatment, Ojjaara (momelotinib), for adults with intermediate- or high-risk myelofibrosis and anemia. This announcement comes after a three-month delay due to the need for additional data review.
Myelofibrosis is a rare blood cancer characterized by scar tissue formation in the bone marrow, leading to severe anemia among other health issues. Ojjaara, a once-daily oral JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor, is the only FDA-approved medicine addressing the key manifestations of the disease: anemia, constitutional symptoms, and splenomegaly (enlarged spleen). It is applicable to both newly diagnosed and previously treated patients.
GSK acquired Ojjaara through a $1.9 billion purchase of Sierra Oncology (NASDAQ:SRRA) in April 2022. The drug already had positive phase 3 trial data at the time of acquisition. By August 2022, the FDA had accepted its New Drug Application (NDA), initially assigning a Prescription Drug User Fee Act (PDUFA) date of June 16, 2023. However, the agency extended this deadline by three months to review recently submitted data.
Ojjaara's approval was based on two phase 3 trials, MOMENTUM and SIMPLIFY-1. The latter was designed to evaluate the drug's efficacy and safety against Incyte (NASDAQ:INCY)'s Jakafi in myelofibrosis patients who had not received prior JAK inhibitor treatment. Regulators evaluated safety and efficacy results based on a subset of patients with anemia.
Despite having similar action mechanisms to Incyte’s Jakafi and Bristol Myers’ Inrebic, Ojjaara differentiates itself by inhibiting the ACVR1 protein, which GSK believes leads to its effect on anemia. A late-stage clinical trial revealed that a significantly higher percentage of myelofibrosis patients receiving Ojjaara experienced a 50% or greater reduction in disease-related symptoms. The drug also appeared to improve other health measures such as reducing spleen size and the need for blood transfusions.
“With momelotinib we have the potential to establish a new standard of care for myelofibrosis patients with anemia,” said Ruben Mesa, executive director at the Atrium Health Wake Forest Baptist Comprehensive Cancer Center.
The broad labeling and apparent effects on anemia could make Ojjaara a meaningful competitor to Jakafi, according to Andrew Berens, an analyst at Leerink Partners. Last year, Incyte reported $2.4 billion in net revenue from Jakafi, which is also approved to treat two other diseases.
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