Gepotidacin

Taylor Tieden for BioSpace The FDA’s docket in December includes decisions for two big biologic franchises: BMS’s Breyanzi and Amgen’s Uplizna. December is shaping up to be a loaded month for the FDA, with more than 15 decisions lined up. Over the next two weeks, the regulator is scheduled to render some big verdicts, including ones for a previously rejected nasal spray tachycardia drug and two gonorrhea treatments. Read below for more. BMS Seeks Fifth Indication for CAR T Blockbuster Breyanzi To kick off the year-end craze, the FDA will decide whether to authorize an expansion of Bristol Myers Squibb’s CAR T cell therapy Breyanzi into marginal zone lymphoma (MZL). The agency is set to release its verdict on Dec. 5. 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. Last year, the product surged 105% to bring in $747 million worldwide , with sales bolstered by additional indications picked up over the years, including chronic lymphocytic leukemia and small lymphocytic leukemia in March , and relapsed or refractory follicular lymphoma in May . To support its latest bid, BMS filed data from the MZL cohort of the Phase II TRANSCEND FL study, which found a 95.5% overall response rate after Breyanzi treatment, including a 62.1% complete response rate. Progression-free survival at 24 months was 85.7% while overall survival reached 90.4%. If approved, Breyanzi would be the first CAR T option for MZL. GSK Eyes Approval for Gonorrhea Antibiotic A few days later, GSK is looking at a Dec. 11 decision date for its oral antibiotic Blujepa for the treatment of uncomplicated urogenital gonorrhea. In particular, the pharma is targeting patients 12 years and up, in whom the drug elicited a 92.6% treatment success rate, according to an analysis of urogenital samples in the Phase III EAGLE-1 study . Comparators treated with intramuscular ceftriaxone plus oral azithromycin saw a success rate of 91.2%. Blujepa’s benefit, according to GSK, cleared the criteria for non-inferiority versus the current standard regimen for this disease. Caused by the bacteria Neisseria gonorrhoeae , gonorrhea is a common sexually transmitted infection that the CDC has deemed an “urgent” threat to public health, particularly given the rising rates of antimicrobial resistance. In March, the FDA approved Blujepa for uncomplicated urinary tract infections, marking the first new class of oral antibiotic for this disease in almost 30 years. BioCryst’s Pediatric Push for Orladeyo Nears Verdict Following Delay On or before Dec. 12, the FDA is expected to decide on BioCryst’s application to expand its hereditary angioedema (HAE) drug Orladeyo into a younger patient population. This verdict will come after a three-month delay the FDA handed down in June, citing the need for more time to review BioCryst’s additional submissions, which the regulator classified as a major amendment to the data package. Orladeyo, a plasma kallikrein blocker, is already approved for HAE, but is currently indicated only for adults and adolescents 12 years old and older. BioCryst is proposing to broaden the drug’s patient coverage to include children with HAE from 2 to 11 years of age. Interim data from the Phase III APeX-P study back this expansion bid, showing that the drug induced early and sustained improvements in monthly HAE attack rates, while also maintaining its safety pro this younger age group. 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. FDA To Decide on Milestone’s Response to Cardamyst Rejection In March, the FDA handed Milestone Pharmaceuticals a surprise rejection for its tachycardia nasal spray Cardamyst, citing chemistry, manufacturing and controls issues. The company has since met with the regulator and filed its response to address the problems raised. The FDA is currently reviewing Milestone’s answers and is expected to release a decision on Dec. 13. Cardamyst is a calcium channel blocker that achieves the efficacy of intravenous drugs despite being delivered via a nasal spray, CEO Joe Oliveto told BioSpace in an interview prior to the rejection. The company is proposing the drug for paroxysmal supraventricular tachycardia, a heart rhythm abnormality. Data from the pivotal Phase III RAPID study showed that 64% of patients treated with self-administered Cardamyst achieved normal heart rhythm within 30 minutes, as opposed to 31% of placebo controls. Cardamyst has had a difficult regulatory road so far, being hit with a refusal to in December 2023 after the FDA found Milestone’s data package for the drug insufficient for a full review. Milestone resubmitted in March 2024. Amgen Aims for Uplizna Expansion Into Myasthenia Gravis Amgen is gunning for another expansion for its anti-CD19 antibody Uplizna, this time targeting generalized myasthenia gravis (gMG). The FDA has a target action date of Dec. 14. Supporting the gMG push are data from the Phase III MINT study, which in September 2024 showed significant improvements in activities of daily living at 26 weeks as compared with placebo. Uplizna treatment also significantly eased disease severity. Follow-on data presented in March showed that Uplizna’s benefits on activities of daily living and disease activity are robust up to 1 year of follow-up , especially in patients positive for acetylcholine antibodies. William Blair analysts at the time said Uplizna had “strong efficacy with impressive durability” and noted that its dosing schedule—one injection every six months—could set it apart from other gMG therapies that have to be given weekly or monthly. Outside of gMG, Uplizna in April won the FDA’s blessing for IgG4-related disease, marking the regulator’s first approval in this indication. Innoviva Also Seeks Gonorrhea Greenlight With Oral Antibiotic Rounding out the front half of December is Innoviva, which is proposing its oral drug zoliflodacin for the treatment of uncomplicated gonorrhea in patients 12 years and older. The FDA’s decision is expected on Dec. 15. Zoliflodacin is a potentially first-in-class antibacterial agent that works by targeting and deactivating a bacterial enzyme called type II topoisomerase, which is crucial for their function and reproduction. According to Innoviva, in vitro studies have demonstrated zoliflodacin’s activity against N. gonorrhoeae, even when used on multidrug-resistant strains. Pivotal Phase III data presented at the European Society of Clinical Microbiology and Infectious Disease Global meeting in April 2024 showed that zoliflodacin elicited a 90.9% microbiological cure rate in patients with uncomplicated gonorrhea, as opposed to 96.2% with the current standard regimen of intramuscular ceftriaxone plus oral azithromycin. While the treatment difference favored the control, the results nevertheless satisfied statistical non-inferiority, Innoviva said at the time. Follow-up data presented in October additionally established zoliflodacin’s efficacy across key subgroups, including patients positive for infections at urogenital, rectal and pharyngeal sites. Cure rates for zoliflodacin likewise remained above 95% in patients with ciprofloxacin-resistant and -susceptible infections.