In January 2026, the FDA issued a series of approvals and regulatory updates spanning rare hematology, neurology, endocrinology, respiratory, and ophthalmology care, marking significant advances in previously untreated patient populations.
Pediatric aTTP and Rare Hematology
On January 5, 2026, the FDA approved caplacizumab-yhdp (Cablivi) for pediatric patients aged ≥12 years with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy. The approval, issued by Sanofi, expands the drug’s 2019 adult indication and represents the first approved therapy specifically for pediatric aTTP. A retrospective chart review of 30 patients aged 2–18 years showed 80% achieved clinical remission, defined by sustained normalization of platelet counts and LDH levels.
On January 7, 2026, the FDA approved a new 2-gram presentation of Octapharma’s fibrinogen (human) lyophilized powder for reconstitution (Fibryga) for fibrinogen replacement in patients with acquired fibrinogen deficiency. The expanded presentation builds on Fibryga’s 2024 approval as the first and only virus-inactivated, plasma-derived fibrinogen concentrate approved for this indication in the US and is designed to improve dosing flexibility and speed of administration in critical bleeding settings.
Mental Health and GLP-1 Safety Updates
The FDA approved ProlivRx on January 13, 2026, the first at-home, physician-directed neuromodulation therapy for adults with major depressive disorder who have not responded to at least one antidepressant. Approval was supported by the MOOD trial, in which ProlivRx significantly improved remission rates versus sham and demonstrated a favorable safety profile.
Also on January 13, 2026, the FDA requested removal of language describing a potential risk of suicidal ideation and behavior from labeling of select GLP-1 receptor agonists, including liraglutide (Saxenda), semaglutide (Wegovy), and tirzepatide (Zepbound). The request followed a comprehensive safety review based on meta-analyses of more than 100,000 patients and large real-world studies, finding no evidence of increased risk for suicidal ideation, self-harm, or other psychiatric adverse events associated with GLP-1 RA use.
Emergency Preparedness and Diagnostic Innovation
The FDA approved new discreet carrying-case packaging for OTC Narcan (naloxone) nasal spray to improve portability and encourage everyday carry. According to the agency, only 10% of the general population reports carrying naloxone despite its OTC availability.
The FDA granted 510(k) clearance to LEADOPTIK’s Last Inch Assessment™ system, a biopsy-integrated depth imaging technology designed to improve real-time tissue confirmation during lung biopsies. By embedding high-resolution imaging directly into standard biopsy tools, the system aims to improve biopsy accuracy, reported to exceed 95% in preclinical data.
Presbyopia Treatment
The FDA approved Tenpoint Therapeutics’ YUVEZZI (carbachol and brimonidine tartrate ophthalmic solution 2.75%/0.1%) as the first fixed-dose, dual-agent topical therapy for presbyopia in adults. Phase 3 BRIO I and II trials demonstrated ≥3-line improvement in binocular uncorrected near visual acuity for up to 8 hours without significant loss of distance vision, with a favorable safety profile including low rates of ocular hyperemia.
This article is an AI-assisted summary. All facts and figures are drawn from the original report: https://www.hcplive.com/view/fda-recap-january-2026