5 Notable FDA Approvals From the First Half of 2026

The first half of 2026 brought a steady stream of FDA approvals across obesity, oncology, nephrology, and rare genetic disease, several of them first-in-class or first-in-indication therapies.

Among the most closely watched were the first oral glucagon-like peptide-1 (GLP-1) receptor agonist for obesity without food or water restrictions, the first all-oral frontline regimen for acute myeloid leukemia (AML), the first therapy FDA-approved for focal segmental glomerulosclerosis (FSGS), the first gene therapy for hearing loss tied to OTOF mutations, and the first new treatment for high-risk non–muscle-invasive bladder cancer (NMIBC) in more than 30 years.

Learn more about 5 of the most notable approvals from the first half of 2026 below:

1. FDA Approves Orforglipron as First Oral GLP-1 for Obesity

The FDA approved orforglipron (Foundayo; Eli Lilly) on April 1 for chronic weight management in adults with obesity, or in adults who are overweight with at least 1 weight-related comorbidity, used alongside a reduced-calorie diet and increased physical activity.¹ The once-daily pill is the first oral GLP-1 receptor agonist that can be taken at any time of day without restrictions on food or water intake.

In clinical trials, orforglipron produced an average of 12.4% weight loss at its highest dose. However, company leadership and clinicians have acknowledged that the pill does not match the efficacy of injectable GLP-1 agents. As a small molecule rather than a peptide, orforglipron is easier to manufacture than oral semaglutide, which was the first GLP-1 weight-loss pill approved in December 2025.² 

2. FDA Approves First All-Oral Regimen for AML

On May 13, the FDA approved decitabine and cedazuridine (Inqovi; Taiho) plus venetoclax for adults 75 years and older, or otherwise ineligible for intensive induction chemotherapy, who have newly diagnosed AML. Notably, the approval is the first all-oral combination therapy for the indication.³

The decision was supported by the phase 2 ASCERTAIN-V trial (NCT04657081), which enrolled 101 patients and demonstrated a complete response rate of 41.6%, a complete response plus a complete remission with incomplete count recovery rate of 63.4%, and a median overall survival of 15.5 months. More than 75% of responders maintained remission at 12 months. Grade 3 or higher adverse events were common and dominated by myelosuppression. Because standard care in this setting has relied on parenteral hypomethylating agents requiring monthly, multiday clinic visits, the all-oral regimen allows eligible patients to be treated at home.

3. FDA Approves Sparsentan for Focal Segmental Glomerulosclerosis

On April 14, the FDA approved sparsentan (Filspari; Travere Therapeutics) for FSGS, marking the first FDA approval for the rare, progressive kidney disease.⁴ FSGS causes scarring in the kidney's filtering units and drives protein leakage into the urine, and it had previously been managed with supportive and off-label care rather than any agent approved for the condition itself. The approval extends sparsentan, a dual endothelin and angiotensin II receptor antagonist, into a disease with no prior on-label option.

4. FDA Approves First Gene Therapy for OTOF-Related Hearing Loss

On April 23, lunsotogene parvec (Otarmeni; Regeneron) became the first FDA-approved gene therapy for hearing loss caused by mutations in the OTOF gene, delivering durable improvements from a single dose.⁵ The manufacturer said it would supply the therapy free of charge in the US, an unusual launch approach for a one-time gene therapy.

5. FDA Approves Durvalumab Plus BCG for High-Risk NMIBC

The FDA closed out the half in May by approving durvalumab (Imfinzi; AstraZeneca) with bacillus Calmette-Guérin (BCG) induction and maintenance for adults with BCG-naive, high-risk NMIBC. This is the first immunotherapy combination approved for the disease and the first new treatment for the population in more than 3 decades.⁶

The approval was based on the phase 3 POTOMAC trial (NCT03528694). After a median follow-up of 60.7 months, adding durvalumab to BCG reduced the risk of high-risk disease recurrence or death by 32% compared with BCG alone (HR, 0.68; 95% CI, 0.50-0.93; P = .015). Trial investigator Neal D. Shore, MD, told The American Journal of Managed Care^® (AJMC^®) the regimen "takes the brakes off of the immune system," helping T cells recognize antigens on cancer cells.⁶

References

1. Hohmann E. FDA approves Lilly's oral GLP-1 orforglipron for obesity. AJMC. Published April 1, 2026. Accessed July 6, 2026. https://www.ajmc.com/view/fda-approves-lilly-s-oral-glp-1-orforglipron-for-obesity
2. Joszt L. FDA approves oral semaglutide as first GLP-1 pill for weight loss. AJMC. Published December 15, 2025. Accessed July 6, 2026. https://www.ajmc.com/view/fda-approves-oral-semaglutide-as-first-glp-1-pill-for-weight-loss
3. Shaw ML. FDA approves first all-oral regimen for AML. AJMC. Published May 13, 2026. Accessed July 6, 2026. https://www.ajmc.com/view/fda-approves-first-all-oral-regimen-for-aml
4. Shaw ML. FDA approves sparsentan for focal segmental glomerulosclerosis. AJMC. Published April 13, 2026. Accessed July 6, 2026. https://www.ajmc.com/view/fda-approves-sparsentan-for-focal-segmental-glomerulosclerosis
5. McNulty R. Lunsotogene parvec becomes first FDA-approved gene therapy for OTOF-related hearing loss. AJMC. Published April 23, 2026. Accessed July 6, 2026. https://www.ajmc.com/view/lunsotogene-parvec-becomes-first-fda-approved-gene-therapy-for-otof-related-hearing-loss
6. McCormick B. FDA approves durvalumab plus BCG as first immunotherapy combo regimen for high-risk NMIBC. AJMC. Published May 28, 2026. Accessed July 6, 2026. https://www.ajmc.com/view/fda-approves-durvalumab-plus-bcg-as-first-immunotherapy-combo-regimen-for-high-risk-nmibc