Five April FDA approvals broaden options for HIV, type 1 diabetes, hives, genetic hearing loss and lupus

A flurry of U.S. approvals in April advanced care across rare and common conditions, with regulators clearing five therapies for HIV, type 1 diabetes, chronic hives, genetic-related hearing loss and lupus. The decisions span infectious disease, endocrinology, immunology and gene therapy, underscoring momentum in both early intervention and treatment simplification.

Among the headline moves, the FDA approved a once-daily fixed-dose combination of doravirine and islatravir (Idvysno; Merck) for adults with virologically suppressed HIV‑1 (HIV RNA under 50 copies/mL) who have no history of treatment failure or known resistance to doravirine.

Intended to replace an existing stable antiretroviral regimen, it is the first complete two‑drug option that is both non–integrase strand transfer inhibitor (non‑INSTI)‑based and tenofovir‑free. The decision drew on two phase 3 trials, MK‑8591A‑052 and MK‑8591A‑051.

In trial 052, which compared switching to doravirine/islatravir with continuing bictegravir/emtricitabine/tenofovir alafenamide, 1% of patients in both groups had HIV RNA of at least 50 copies/mL at week 48, meeting noninferiority, with viral suppression rates of 92% versus 94%, respectively.

Trial 051, which enrolled patients switching from various baseline regimens, found 1% of those receiving doravirine/islatravir versus 5% of those continuing baseline therapy had viral loads of at least 50 copies/mL. Across both studies, more than 700 adults aged 18 to over 80 years were enrolled, and safety was comparable to standard regimens with low discontinuation rates.

“People aging with HIV face additional health challenges, including managing multiple chronic conditions and medications at the same time,” said Carl Baloney Jr, president and CEO of AIDS United, adding that treatment choices should consider these factors alongside virologic control.

In endocrinology, the FDA on April 22 expanded the indication for teplizumab‑mzwv (Tzield; Sanofi) to children 1 year and older with stage 2 type 1 diabetes, a presymptomatic phase characterized by dysglycemia and diabetes-related autoantibodies. The therapy aims to delay progression to stage 3 disease, when lifelong insulin therapy becomes necessary.

Previously limited to children 8 years and older, the updated indication was supported by interim data from the phase 4 PETITE‑T1D study, which enrolled 23 participants with a mean age of 4.8 years for a 14‑day course of daily intravenous dosing.

All participants experienced treatment‑emergent adverse events, but no grade 4 or 5 events were reported; discontinuations were tied to manageable issues such as anemia, elevated liver enzymes and rash. Regulators also approved dupilumab (Dupixent; Sanofi and Regeneron) for young children with chronic spontaneous urticaria, adding a pediatric option for a condition marked by persistent hives without a clear trigger.

In gene therapy, lunsotogene parvec‑cwha (Otarmeni; Regeneron) became the first approved therapy targeting otoferlin (OTOF) gene–related hearing loss. And in lupus, a weekly anifrolumab‑fnia autoinjector (Saphnelo; AstraZeneca) was cleared, signaling progress in simplifying treatment for systemic lupus erythematosus.

Taken together, April’s decisions expand choices for patients and clinicians, from early disease modification in type 1 diabetes to streamlined HIV regimens and new modalities in immunology and gene therapy. Sponsors and clinicians now turn to implementation, including identifying eligible patients and integrating these options into care.