Upcoming FDA decisions span expanded indications and investigational therapies across multiple specialties. April 2026 could bring several high-impact regulatory decisions, including potential label expansions and novel therapies in neurology, nephrology, endocrinology, and infectious diseases.
Neurology
GTx-104
The FDA has accepted GTx-104 for review for the treatment of patients with aneurysmal subarachnoid hemorrhage, with a PDUFA target action date of April 23, 2026. GTx-104 is an intravenous formulation of nimodipine designed for continuous infusion. In the Phase 3 STRIVE-ON safety trial, GTx-104 reduced the incidence of clinically significant hypotension compared with oral nimodipine, with adverse events reported as comparable between treatment groups.
Source: Grace Therapeutics
AXS-05 (dextromethorphan HBr and bupropion HCl)
The FDA has accepted AXS-05 (dextromethorphan HBr and bupropion HCl) for priority review for the treatment of Alzheimer’s disease agitation, with a PDUFA target action date of April 30, 2026. AXS-05 is an oral combination of dextromethorphan and bupropion that acts as an NMDA receptor antagonist and sigma-1 receptor agonist, with bupropion increasing dextromethorphan bioavailability via CYP2D6 inhibition. The application is supported by a clinical development program that includes four Phase 3 randomized, controlled trials and a long-term safety study.
Source: Axsome Therapeutics
Nephrology
The FDA has extended its review of sparsentan (FILSPARI) for focal segmental glomerulosclerosis (FSGS), with a PDUFA target action date of April 13, 2026. Sparsentan is an oral, non-immunosuppressive dual endothelin type A receptor and angiotensin II type 1 receptor antagonist. In the Phase 3 DUPLEX and Phase 2 DUET studies, sparsentan reduced proteinuria and was associated with higher remission rates compared with irbesartan, with a safety profile comparable to the active control; however, the DUPLEX study did not meet its primary eGFR slope endpoint.
Source: Travere Therapeutics
Endocrinology
The FDA has accepted teplizumab (TZIELD) for priority review to expand its indication to children aged 1 year and older with stage 2 type 1 diabetes to delay progression to stage 3 disease, with a PDUFA target action date of April 29, 2026. Teplizumab is a CD3-directed monoclonal antibody that modulates immune activity involved in beta cell destruction. The application is supported by interim one-year data from the Phase 4 PETITE-T1D study evaluating safety and pharmacokinetics in children younger than 8 years.
Source: Sanofi
Infectious Disease
Doravirine/Islatravir (DOR/ISL)
The FDA is reviewing doravirine/islatravir (DOR/ISL) for the treatment of HIV-1 infection in adults who are virologically suppressed on a stable antiretroviral regimen, with a PDUFA target action date of April 28, 2026. DOR/ISL is an investigational, once-daily, oral two-drug regimen. In a Phase 3 trial in treatment-naive adults, DOR/ISL was non-inferior to bictegravir/emtricitabine/tenofovir alafenamide at Week 48. In separate Phase 3 switch studies in virologically suppressed adults, DOR/ISL maintained high rates of viral suppression through Week 96, with a safety profile similar to comparator regimens.
Source: Merck & Co., Inc
