FDA News Recap: Novel Drug Approvals in Q3 2026

Published on: October 1, 2026

FDA novel drug approvals in Q3 2026: 22 new therapies, including enlicitide, daraxonrasib, oveporexton, and first treatments for rare diseases.

Welcome back to our recap of news and updates from the US Food and Drug Administration (FDA)!

Q3 2026 delivered the busiest regulatory quarter of the year so far, spanning novel first-in-class approvals, first-ever treatments for ultra-rare diseases, and several long-anticipated decisions across oncology, neurology, cardiometabolic disease, nephrology, and rheumatology.

July opened with the accelerated approval of atacicept as the first dual BAFF/APRIL inhibitor for immunoglobulin A nephropathy (IgAN). Weeks later came enlicitide, the first oral PCSK9 inhibitor, and centanafadine, the first norepinephrine, dopamine, and serotonin reuptake inhibitor (NDSRI) for attention-deficit/hyperactivity disorder (ADHD). August brought oveporexton as the first orexin receptor 2 agonist for narcolepsy type 1 and brepocitinib as the first oral therapy for dermatomyositis. The month also brought daraxonrasib, an oral RAS(ON) inhibitor that doubled median overall survival vs chemotherapy in previously treated metastatic pancreatic cancer.

September closed the quarter with a wave of rare disease firsts. These included zilganersen for Alexander disease, apitegromab as the first muscle-targeted therapy for spinal muscular atrophy (SMA), and tiratricol for monocarboxylate transporter 8 (MCT8) deficiency. Zilurgisertib also became the third approved therapy for fibrodysplasia ossificans progressiva (FOP), following August's approval of garetosmab. The quarter also added efsitora, a once-weekly basal insulin for type 2 diabetes, and tavapadon, a first-in-class D1/D5 partial agonist for Parkinson disease.

Here is a look at the 22 novel drugs approved by the FDA in Q3 2026, listed in order of approval date:

1. Atacicept (Trutakna)

Company: Vera Therapeutics

Approval Date: July 7, 2026

Indication: Primary Immunoglobulin A Nephropathy (IgAN)

Summary: The FDA granted accelerated approval to atacicept to reduce proteinuria in adults with primary IgAN at risk for disease progression. Atacicept is the first dual inhibitor of B-cell activating factor (BAFF) and a proliferation-inducing ligand (APRIL) approved for the disease. The approval was supported by the prespecified interim analysis of the phase 3 ORIGIN 3 trial, in which atacicept reduced proteinuria at week 36 by 46% from baseline and by 42% compared with placebo (P <.0001).

2. Gedatolisib (Revtorpyk)

Company: Celcuity

Approval Date: July 14, 2026

Indication: Hormone Receptor–Positive, HER2-Negative Advanced Breast Cancer Without a PIK3CA Mutation

Summary: The FDA approved gedatolisib in combination with fulvestrant, with or without palbociclib, for adults whose disease progressed on or after at least one line of endocrine therapy in the metastatic setting. Gedatolisib is a multitarget PI3K/AKT/mTOR inhibitor designed to block multiple components of the pathway at once. In Study 1 of the phase 3 VIKTORIA-1 trial, median progression-free survival (PFS) reached 9.3 months with the gedatolisib triplet versus 2.0 months with fulvestrant alone (HR, 0.24).

3. Enlicitide (Lipfendra)

Company: Merck

Approval Date: July 15, 2026

Indication: Hypercholesterolemia, Including Heterozygous Familial Hypercholesterolemia (HeFH)

Summary: The FDA approved enlicitide as the first once-daily oral PCSK9 inhibitor, for use with diet and exercise to reduce low-density lipoprotein cholesterol (LDL-C) in adults. The macrocyclic peptide binds PCSK9 to preserve LDL receptor availability. In the phase 3 CORALreef Lipids and CORALreef HeFH trials, enlicitide reduced LDL-C by a placebo-adjusted 56% and 59%, respectively, at 24 weeks. The approval rests on lipid endpoints; CORALreef Outcomes, with more than 14,500 enrolled participants, is evaluating cardiovascular events.

4. Zidesamtinib (Jideytro)

Company: Nuvalent

Approval Date: July 22, 2026

Indication: ROS1-Positive Non–Small Cell Lung Cancer (NSCLC) After a Prior ROS1 Inhibitor

Summary: The FDA approved zidesamtinib for adults with locally advanced or metastatic ROS1-positive NSCLC who received at least one prior ROS1 tyrosine kinase inhibitor. In the phase 1/2 ARROS-1 trial, zidesamtinib produced an objective response rate (ORR) of 44% (95% CI, 34%-53%) among 117 treated patients. Among responders, 82% had responses lasting at least 6 months and 69% at least 12 months.

5. Bevacizumab-vikg (Lytenava)

Company: Outlook Therapeutics

Approval Date: July 24, 2026

Indication: Neovascular (Wet) Age-Related Macular Degeneration (AMD)

Summary: The FDA approved bevacizumab-vikg, the first FDA-approved ophthalmic formulation of bevacizumab for wet AMD in the US. The product was developed specifically for intravitreal administration, replacing reliance on repackaged intravenous formulations. The approval followed three prior complete response letters (CRLs) and a successful appeal through the FDA's formal dispute resolution process. Outlook expects to complete the US launch before year-end.

6. Centanafadine (Simtriyo)

Company: Otsuka Pharmaceutical

Approval Date: July 24, 2026

Indication: ADHD in Adults and Pediatric Patients Aged ≥6 Years

Summary: The FDA approved centanafadine, a once-daily extended-release capsule, for patients aged ≥6 years weighing ≥20 kg. Centanafadine is the first approved NDSRI, a mechanism distinct from existing stimulant and nonstimulant ADHD therapies. The approval was based on four phase 3 trials in adults, adolescents, and children. The label carries boxed warnings for suicidality and abuse potential, and commercial availability is pending scheduling by the US Drug Enforcement Administration.

7. Oveporexton (Orzeyful)

Company: Takeda

Approval Date: August 5, 2026

Indication: Narcolepsy Type 1 (NT1) in Adults

Summary: The FDA approved oveporexton, an oral orexin receptor 2 (OX2R)-selective agonist and the first therapy to directly restore the orexin signaling lost in NT1. The approval followed Breakthrough Therapy designation and Priority Review. It was based on the phase 3 FirstLight (n = 168) and RadiantLight (n = 105) trials, in which oveporexton 2 mg twice daily significantly improved Maintenance of Wakefulness Test performance vs placebo over 12 weeks (P <.001).

8. Iberdomide (Zenbexus)

Company: Bristol Myers Squibb

Approval Date: August 13, 2026

Indication: Relapsed or Refractory Multiple Myeloma After ≥1 Prior Line Including a Proteasome Inhibitor and an Immunomodulatory Agent

Summary: The FDA granted accelerated approval to iberdomide in combination with daratumumab and hyaluronidase-fihj and dexamethasone. Iberdomide is a cereblon E3 ligase modulator. In the phase 3 EXCALIBER-RRMM trial, the iberdomide regimen achieved a minimal residual disease (MRD)-negative complete response rate of 41% vs 21% with daratumumab, bortezomib, and dexamethasone (P < .0001). The label includes a boxed warning for embryo-fetal toxicity and thromboembolism, and iberdomide is available only through a Risk Evaluation and Mitigation Strategy (REMS) program.

9. Florquinitau F 18 (Tauklarify)

Company: Lantheus

Approval Date: August 13, 2026

Indication: Tau PET Imaging in Adults With Cognitive Impairment Being Evaluated for Alzheimer Disease

Summary: The FDA approved florquinitau F 18, previously known as MK-6240, to identify patients with tau neurofibrillary tangle pathology. It joins flortaucipir (Tauvid) as the second FDA-approved F-18 tau PET imaging agent. In 2 pivotal blinded-reader studies, positive percent agreement ranged from 68% to 88% and negative percent agreement from 93% to 99%. Safety and effectiveness have not been established for non-Alzheimer tauopathies.

10. Garetosmab-grts (Pasatru)

Company: Regeneron

Approval Date: August 19, 2026

Indication: FOP in Adults

Summary: The FDA approved garetosmab-grts, an intravenous activin A–neutralizing monoclonal antibody, to reduce new heterotopic ossification (HO) lesions and clinician-assessed flare-ups. In the phase 3 OPTIMA trial of 63 adults, garetosmab reduced new HO lesions by 90% to 94% vs placebo across 2 doses: 2 new lesions among 23 patients at 10 mg/kg and 1 among 19 patients at 3 mg/kg, vs 19 among 21 placebo recipients. Key risks include infection and epistaxis, and garetosmab is contraindicated in pregnancy.

11. Daraxonrasib (Rasonque)

Company: Revolution Medicines

Approval Date: August 26, 2026

Indication: Metastatic Pancreatic Adenocarcinoma After ≥1 Prior Systemic Therapy or in Patients Ineligible for Multi-Agent Therapy

Summary: The FDA approved daraxonrasib, an oral RAS(ON) multiselective inhibitor, 6.5 months ahead of its user fee goal date. In the phase 3 RASolute 302 trial of 500 patients, median overall survival was 13.2 months with daraxonrasib versus 6.7 months with chemotherapy (HR, 0.40; P < .0001). Median PFS was 7.2 versus 3.6 months (HR, 0.49).

12. Brepocitinib (Lisraya)

Company: Priovant Therapeutics

Approval Date: August 27, 2026

Indication: Dermatomyositis in Adults

Summary: The FDA approved brepocitinib, a once-daily oral TYK2/JAK1 inhibitor, as the first oral therapy specifically studied and approved for dermatomyositis. In the phase 3 VALOR trial of 241 adults, brepocitinib 30 mg produced a higher mean Total Improvement Score at week 52 versus placebo. Patients also showed improvements in physical function and skin disease activity and were more likely to reduce corticosteroid use. The label carries a boxed warning consistent with other JAK inhibitors.

13. Rusfertide (Mimrylo)

Company: Takeda

Approval Date: August 28, 2026

Indication: Erythrocytosis in Adults With Polycythemia Vera

Summary: The FDA approved rusfertide, the first polycythemia vera therapy to mimic hepcidin. It limits the iron available for red blood cell production. In the phase 3 VERIFY trial, 76.9% of rusfertide-treated patients achieved a clinical response, defined as no phlebotomy eligibility or phlebotomies from weeks 20 to 32, versus 32.9% with placebo (P <.0001). Serious adverse events occurred in 3.4% of rusfertide recipients versus 4.8% with placebo.

14. Zilganersen (Zanvastro)

Company: Ionis Pharmaceuticals

Approval Date: September 3, 2026

Indication: Alexander Disease in Pediatric and Adult Patients

Summary: The FDA approved zilganersen as the first treatment for Alexander disease. The intrathecal antisense oligonucleotide, dosed every 3 months, reduces production of glial fibrillary acidic protein (GFAP), whose accumulation drives the disease. In a phase 1-3 study of 54 participants, the 50-mg dose stabilized gait speed on the 10-Meter Walk Test at week 61 among participants aged ≥5 years (least-squares mean difference vs control, 33.3%; P = .041).

15. Camizestrant (Etcamah)

Company: AstraZeneca

Approval Date: September 4, 2026

Indication: HR-Positive, HER2-Negative Advanced Breast Cancer With an ESR1 Mutation Emerging During Aromatase Inhibitor Plus CDK4/6 Inhibitor Therapy

Summary: The FDA granted accelerated approval to camizestrant, an oral selective estrogen receptor degrader, in combination with a CDK4/6 inhibitor. The Guardant360 CDx assay was approved alongside it as a companion diagnostic. In the phase 3 SERENA-6 trial, switching to camizestrant upon detection of an ESR1 mutation improved median PFS to 16.0 months versus 9.2 months with continued aromatase inhibition (HR, 0.44; P <.00001).

16. Apitegromab-mstn (Isembyld)

Company: Scholar Rock

Approval Date: September 11, 2026

Indication: SMA in Patients Aged ≥2 Years Receiving an SMN2-Targeted Treatment

Summary: The FDA approved apitegromab-mstn, the first SMA therapy designed to directly target muscle loss, as a complement to SMN2-targeted therapies. The phase 3 SAPPHIRE trial randomized 188 nonambulatory patients aged 2 to 21 years. In the primary analysis of patients aged 2 to 12 years, 34.2% of patients receiving 10 mg/kg achieved at least a 3-point improvement on the Hammersmith Functional Motor Scale Expanded versus 13.5% with placebo. An increased risk of fractures was observed.

17. Floretyrosine F 18 (Pixclara)

Company: Telix Pharmaceuticals

Approval Date: September 11, 2026

Indication: PET Imaging to Differentiate Recurrent or Progressive Glioma From Treatment-Related Change

Summary: The FDA approved floretyrosine F 18, the first FDA-approved amino acid PET imaging agent for glioma. The agent targets L-type amino acid transporters 1 and 2 (LAT1 and LAT2) and is indicated for adults and pediatric patients aged ≥1 month, in conjunction with other diagnostic evaluations. FET-PET imaging is already recommended in international guidelines, including the NCCN Guidelines, but no FDA-approved agent previously existed for this use.

18. Insulin Efsitora Alfa-gobe (Onswik)

Company: Eli Lilly and Company

Approval Date: September 23, 2026

Indication: Glycemic Control in Adults With Type 2 Diabetes

Summary: The FDA approved insulin efsitora alfa-gobe, a once-weekly basal insulin that reduces basal injections from approximately 365 to 52 per year. Across the four phase 3 QWINT trials in more than 3,400 adults, efsitora achieved noninferior hemoglobin A1c reductions vs once-daily insulin glargine or degludec. In QWINT-1, A1c fell 1.19 percentage points with efsitora versus 1.16 with glargine at week 52, with fewer level 2 or 3 hypoglycemia events (0.50 vs 0.88 events per participant-year).

19. Lirafugratinib (Lyrfigtu)

Company: Elevar Therapeutics

Approval Date: September 23, 2026

Indication: Previously Treated FGFR2 Fusion– or Rearrangement-Positive Cholangiocarcinoma

Summary: The FDA approved lirafugratinib, a highly selective oral FGFR2 inhibitor. It is indicated for adults with unresectable, locally advanced or metastatic disease. In the REFOCUS trial of 116 FGFR inhibitor–naive patients, lirafugratinib produced an ORR of 46% (95% CI, 36%-55%) and a median duration of response of 11.8 months. The label includes warnings for ocular toxicity, hyperphosphatemia and soft tissue mineralization, and embryo-fetal toxicity.

20. Tavapadon (Juvmo)

Company: AbbVie

Approval Date: September 25, 2026

Indication: Parkinson Disease in Adults

Summary: The FDA approved tavapadon, a once-daily oral dopamine D1/D5 receptor partial agonist. It is mechanistically distinct from existing dopamine agonists, which primarily target D2/D3 receptors. The approval was based on the phase 3 TEMPO program, which evaluated tavapadon as monotherapy in early Parkinson disease and as an adjunct to levodopa in patients with motor fluctuations. In the TEMPO-4 open-label extension, 94% of patients taking tavapadon alone (257/273) had not started levodopa after 85 weeks. AbbVie expects US availability in October 2026.

21. Zilurgisertib (Atebrioz)

Company: Mirum Pharmaceuticals (under license from Incyte)

Approval Date: September 25, 2026

Indication: FOP in Patients Aged ≥12 Years

Summary: The FDA approved zilurgisertib, an oral activin receptor-like kinase 2 (ALK2) inhibitor. It became the third approved FOP therapy, following palovarotene and garetosmab. In cohort 1 of the phase 2 PROGRESS trial (n = 63), total new HO volume decreased by a mean of 3.2 cm³ with zilurgisertib vs a 24.6 cm³ increase with placebo at week 24 (nominal P = .004). The prespecified primary endpoint, the proportion of patients with new HO lesions, showed an 81% relative reduction but did not reach statistical significance (P = .0986).

22. Tiratricol (Emcitate)

Company: Egetis Therapeutics

Approval Date: September 28, 2026

Indication: Peripheral Thyrotoxicosis in Patients With MCT8 Deficiency

Summary: The FDA approved tiratricol as the first FDA-approved therapy for MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome. Tiratricol is a thyroid hormone receptor agonist able to enter cells independently of the MCT8 transporter, lowering the elevated serum T3 levels driving peripheral thyrotoxicosis. Its indication addresses peripheral thyrotoxicosis rather than the disorder's neurodevelopmental symptoms. Supporting data included the randomized, placebo-controlled withdrawal trial ReTRIACt.