December is set to be a busy month for the U.S. Food and Drug Administration (FDA), with more than 15 decisions expected, including key verdicts on treatments for lymphoma, gonorrhea, hereditary angioedema, tachycardia, and myasthenia gravis.
Bristol Myers Squibb (BMS) is awaiting an FDA decision on December 5 regarding the expansion of its CAR T cell therapy Breyanzi into marginal zone lymphoma (MZL). If approved, Breyanzi would become the first CAR T option for MZL. BMS has submitted data from the Phase II TRANSCEND FL study showing a 95.5% overall response rate and a 62.1% complete response rate among patients treated with Breyanzi. Progression-free survival at 24 months was reported at 85.7%, with overall survival at 90.4%. "First approved in 2021 for relapsed or refractory large B cell lymphoma, Breyanzi has become one of the industry’s biggest and fastest-growing CAR T brands," according to the company.
GlaxoSmithKline (GSK) is seeking approval for its oral antibiotic Blujepa to treat uncomplicated urogenital gonorrhea in patients aged 12 years and older, with an FDA decision expected on December 11. In the Phase III EAGLE-1 study, Blujepa achieved a treatment success rate of 92.6%, compared to a 91.2% success rate with standard therapy using intramuscular ceftriaxone plus oral azithromycin. GSK noted that "Blujepa’s benefit... cleared the criteria for non-inferiority versus the current standard regimen." The Centers for Disease Control and Prevention (CDC) considers gonorrhea an urgent public health threat due to rising antimicrobial resistance.
BioCryst Pharmaceuticals expects an FDA decision by December 12 on expanding its hereditary angioedema drug Orladeyo to include children aged two to eleven years old. The review follows a three-month delay requested by the agency for additional data submissions from BioCryst. Interim results from the Phase III APeX-P study indicated early and sustained improvements in monthly HAE attack rates among younger patients while maintaining safety standards. "Follow-on findings from APeX-P last month showed that 65.5% of patients were attack-free at one month after Orladeyo initiation, growing to 70.4% at 12 months," according to BioCryst.
Milestone Pharmaceuticals awaits an FDA verdict on December 13 regarding its nasal spray Cardamyst for paroxysmal supraventricular tachycardia following a previous rejection in March over chemistry, manufacturing, and controls issues. The company has since addressed these concerns in discussions with regulators and resubmitted its application earlier this year after initially receiving a refusal-to-file letter in late 2023.
Amgen is pursuing an expansion of its anti-CD19 antibody Uplizna into generalized myasthenia gravis (gMG), with an FDA action date set for December 14. Data from Amgen's Phase III MINT study demonstrated significant improvement in daily living activities at week 26 compared with placebo; benefits persisted up to one year in follow-up studies presented this March.
Innoviva is also seeking approval this month—on December 15—for zoliflodacin as an oral treatment for uncomplicated gonorrhea in individuals aged twelve years or older. Zoliflodacin targets bacterial type II topoisomerase enzymes essential for bacterial function and reproduction; it demonstrated activity against multidrug-resistant strains of Neisseria gonorrhoeae during laboratory testing. In pivotal clinical trials presented earlier this year, zoliflodacin achieved microbiological cure rates comparable to current standard therapies while meeting statistical non-inferiority requirements.
These upcoming decisions reflect ongoing efforts by pharmaceutical companies to expand indications or introduce new treatments across several therapeutic areas as regulatory reviews conclude before year's end.
