From the first targeted therapy for chronic spontaneous urticaria and a new treatment for immunoglobulin G4–related disease, to the first oral Janus kinase inhibitor for giant cell arteritis and a 6-month continuous glucose monitor, April’s U.S. Food and Drug Administration approvals highlighted progress in immunology, endocrinology, rheumatology, and more. Here's a comprehensive overview of the month’s key decisions.
Allergy and Immunology
FDA Approves FcRn Blocker for Generalized Myasthenia Gravis
The U.S. Food and Drug Administration (FDA) approved nipocalimab-aahu (Imaavy) for adult and adolescent patients aged 12 years and older with anti-acetylcholine receptor- or anti-muscle-specific kinase-positive generalized myasthenia gravis. The neonatal Fc receptor-blocking antibody demonstrated significant MG-ADL improvements and up to 75% immunoglobulin G reduction in the phase III Vivacity-MG3 trial. Pediatric data from the Vibrance-MG study showed similar efficacy and safety. Common adverse events included upper respiratory infection, peripheral edema, and muscle spasms.
Source: Johnson & Johnson
FDA Approves First Targeted Therapy for Chronic Spontaneous Urticaria
The FDA approved dupilumab as the first targeted therapy for chronic spontaneous urticaria in patients aged 12 years and older whose symptoms are inadequately controlled with histamine-1 antihistamines. The fully human monoclonal antibody inhibits interleukin (IL)-4 and IL-13 signaling and is administered subcutaneously every 2 weeks following a loading dose. Approval was supported by phase III trials showing significant improvements in itch severity and urticaria activity scores. The most common adverse event was injection-site reaction. Dupilumab is not classified as an immunosuppressant and is approved for multiple type 2 inflammatory conditions.
Source: Regeneron Pharmaceuticals
FDA Approves First Therapy for IgG4-Related Disease
The FDA approved inebilizumab-cdon (Uplizna), a humanized monoclonal antibody targeting CD19-positive B cells, as a first-line treatment option for immunoglobulin G4–related disease in adult patients. In the MITIGATE trial, inebilizumab reduced flare risk by 87% and lowered glucocorticoid use compared with placebo. The infusion is given every 6 months following two loading doses. Common adverse events included lymphopenia and urinary tract infection. Inebilizumab is also approved for neuromyelitis optica spectrum disorder and is under review for generalized myasthenia gravis.
Source: Amgen
Endocrinology
FDA Clears Longest-Lasting Continuous Glucose Monitor
The FDA cleared Dexcom G7 15 Day, a continuous glucose monitoring (CGM) system among adult patients with diabetes, offering up to 15.5 days of wear—the longest of any CGM system approved in the United States. The device features a mean absolute relative difference of 8.0%, real-time data sharing, Apple Watch compatibility, and automated tracking of meals, activity, and medications. It includes a 12-hour sensor replacement grace period and remains water resistant. Dexcom expects the system to launch in the second half of 2025.
Source: Dexcom
Gastroenterology
FDA Approves New Option for IgA Nephropathy
The FDA granted accelerated approval to atrasentan (Vanrafia), a selective endothelin A receptor antagonist, to reduce proteinuria in adult patients with primary immunoglobulin A nephropathy at risk of rapid progression. Indicated for use with standard care, including renin-angiotensin system and optional sodium-glucose cotransporter-2 inhibition, atrasentan reduced proteinuria by 36.1% at 36 weeks in the phase III ALIGN trial. Common adverse events included peripheral edema, anemia, and elevated liver enzymes. Liver function monitoring is recommended. Continued approval is contingent on confirmatory data from ongoing studies.
Source: Novartis
Infectious Diseases
Phase III Trial Supports Oral Gepotidacin for Gonorrhea
Gepotidacin, an investigational oral antibiotic, demonstrated noninferiority to ceftriaxone plus azithromycin for treating uncomplicated urogenital gonorrhea in a phase III trial. Microbiological success was achieved in 92.6% of patients receiving gepotidacin compared with 91.2% receiving standard therapy. The oral regimen consists of two 3,000-mg doses administered 10 to 12 hours apart. Adverse events were primarily gastrointestinal and mild to moderate. Gepotidacin offers a potential oral alternative amid growing concern over antimicrobial resistance.
Source: The Lancet
Neurology
FDA Expands Intranasal Diazepam Use in Pediatric Patients
The FDA expanded approval of diazepam nasal spray (Valtoco) for the acute treatment of seizure clusters in pediatric patients aged 2 years and older. The proprietary formulation enables rapid intranasal delivery using absorption enhancement technology. Approval was supported by pharmacokinetic and safety data presented at recent neurology conferences. Common adverse events included somnolence, headache, and nasal discomfort. The novel nasal spray carries a boxed warning for risks associated with opioid co-use, abuse, and dependence, and is contraindicated in patients with central nervous system depression, acute narrow-angle glaucoma, or diazepam hypersensitivity.
Source: Neurelis
Oncology
FDA Approves Dual Immunotherapy for Advanced Colorectal Cancer
The FDA approved nivolumab in combination with ipilimumab for patients aged 12 years and older with unresectable or metastatic microsatellite instability–high or mismatch repair–deficient colorectal cancer. In the CheckMate-8HW trial, median progression-free survival was not reached in the combination group vs 5.8 months with chemotherapy. Objective response rates were 71% for the combination and 58% for nivolumab monotherapy. Common adverse events included fatigue, diarrhea, abdominal pain, and pruritus. The FDA also granted standard approval to single-agent nivolumab in this setting following prior chemotherapy.
Source: FDA
FDA Approves Penpulimab-kcqx for Nasopharyngeal Carcinoma
The FDA approved penpulimab-kcqx, a humanized IgG1 anti–PD-1 monoclonal antibody, for adult patients with recurrent or metastatic nonkeratinizing nasopharyngeal carcinoma. It's indicated in combination with platinum-based chemotherapy for first-line treatment and as monotherapy following progression on prior therapies. In a randomized trial, median progression-free survival was 9.6 months with the combination vs 7.0 months with chemotherapy alone. Common adverse events included nausea, hypothyroidism, and fatigue. Penpulimab-kcqx received fast track, breakthrough therapy, and orphan drug designations.
Source: FDA
Rheumatology
FDA Approves First Oral JAK Inhibitor for Giant Cell Arteritis
The FDA approved upadacitinib (Rinvoq) as the first oral Janus kinase inhibitor for the treatment of giant cell arteritis in adults. Approval was based on phase III data showing 46.4% of patients achieved sustained remission with upadacitinib plus a 26-week corticosteroid taper compared with 29.0% with placebo and a 52-week taper. The oral agent offers a steroid-sparing option for this large-vessel vasculitis. Safety risks include serious infections, malignancy, cardiovascular events, thrombosis, and gastrointestinal perforation. Common adverse events include upper respiratory tract infections and elevated liver enzymes. Patients should be monitored for hematologic and cardiovascular abnormalities during treatment.
Source: AbbVie
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