In a surprising twist, the FDA has slammed the brakes on AstraZeneca’s ambitious plans to expand the horizons of its revolutionary drug, Ultomiris (ravulizumab-cwvz). The company had high hopes for Ultomiris in the battle against neuromyelitis optica spectrum disorder (NMOSD), but the FDA’s complete response letter has sent shockwaves through the pharmaceutical world.
The FDA’s demand? A revamped Risk Evaluation and Mitigation Strategy (REMS) and a keen eye on patients’ meningococcal vaccination status or the need for prophylactic antibiotics before treatment. Despite no hiccups in Ultomiris’ performance, it’s clear that the FDA wants more than just efficacy and safety data.
AstraZeneca, via its rare disease wing, Alexion, is now in crisis management mode, working closely with the FDA to recalibrate the REMS program. Ultomiris, a long-acting monoclonal antibody, takes on the C5 complement protein, quelling the complement cascade and preventing the immune system from going haywire.
This remarkable mechanism of action earned Ultomiris its first FDA nod in 2018 for paroxysmal nocturnal hemoglobinuria. Since then, it has garnered approvals for atypical hemolytic uremic syndrome and generalized myasthenia gravis.
For its latest endeavor, AstraZeneca hinged its hopes on the Phase III CHAMPION-NMOSD trial, which enrolled nearly 60 patients worldwide. To qualify, NMOSD patients had to test positive for anti-APQ4 antibodies and have experienced at least one debilitating attack or relapse in the last year.
In October 2022, AstraZeneca dropped jaws by revealing that Ultomiris had achieved a staggering 98.6% reduction in the risk of relapse, compared to a placebo group, all thanks to CHAMPION-NMOSD. At 48 weeks, the Ultomiris group had an impeccable track record with zero relapses, while only 63% of their placebo counterparts could claim the same.
NMOSD, a rare and ruthless autoimmune ailment, unleashes an immune system frenzy, causing havoc in the central nervous system. Victims suffer vision disturbances, agonizing pain, strange skin sensations, and coordination woes. In severe cases, NMOSD can snatch away sight, inflict paralysis, and even spell untimely demise.
Should Ultomiris secure the FDA’s blessing in this arena, it will find itself in the same league as its sibling, Soliris (eculizumab), targeting the exact same quartet of diseases. Both are C5 complement inhibitors, armed with potent mechanisms of action and a shared boxed warning about the menace of serious meningococcal infections.