The US FDA New Drug Approvals in September 2026  

Shots:   The US FDA approved multiple novel drugs in September 2026 across rare diseases, oncology, neurology, diabetes, and precision medicine, with approvals from companies including Ionis, AstraZeneca, Scholar Rock, Lilly, AbbVie, and Egetis.   Key approvals included Zanvastro for Alexander disease, Etcamah for ESR1-mutated advanced breast cancer, Isembyld for spinal muscular atrophy, Onswik for type 2 diabetes, and Juvmo for Parkinson’s disease.   Other notable approvals included Pixclara for glioma imaging, Lyrfigtu for FGFR2-altered cholangiocarcinoma, Atebrioz for fibrodysplasia ossificans progressiva, and Emcitate for MCT8 deficiency, highlighting continued FDA activity in targeted and rare-disease therapies.  Ionis’ Zanvastro (Zilganersen) Receives US FDA Approval for Alexander Disease (AxD)           Company: Ionis                  Product: Zanvastro                 Active Ingredient: Zilganersen                 Disease: Alexander Disease                 Date: Sep 03, 2026                    Shots:   FDA has approved Zanvastro (50mg, Q3M, intrathecal injection) for the treatment of AxD in pediatric & adult pts, with launch expected in coming wks. FDA also granted Ionis a Rare Pediatric Disease PRV  Approval was backed by the Phase I-III trial assessing Zanvastro (25 or 50mg) vs control in 54 AxD pts (1.5-53yrs.) for 60wks., followed by an open label period & 120wk. open-label LTE, where 25mg cohort moved to the 50mg  Trial showed zilganersen (50mg) met the 1EP of gait speed as assessed by 10-Meter Walk Test at 61wks., plus showed improvement in gross motor function in pts (2-4yrs.) as assessed by GMFM-88. Benefit in key secondary & exploratory EPs was also observed   Editor’s Take: Beyond the Headlines   Zanvastro’s FDA approval gives Ionis a first-mover position in Alexander disease, establishing a targeted treatment option for a rare, underserved population. The approval strengthens Ionis’ neurology franchise and validates its antisense platform, potentially creating a high-value rare-disease opportunity with limited near-term competitive pressure.  AstraZeneca Receives the US FDA Approval for Etcamah (Camizestrant) to Treat Advanced Breast Cancer           Company: AstraZeneca                  Product: Etcamah                 Active Ingredient: Camizestrant                 Disease: ESR1-mutated Advanced Breast Cancer                 Date: Sep 04, 2026                    Shots:   FDA granted accelerated approval to Etcamah + a CDK4/6 inhibitor for HR+/HER2− LA/M breast cancer with an ESR1 mutation emerging during AI + CDK4/6 inhibitor therapy, alongside approval of a ctDNA-based CDx for detecting ESR1 resistance mutations  Approval was based on the P-III (SERENA-6) trial assessing Etcamah + CDK4/6 inhibitor vs AI (anastrozole/letrozole) + CDK4/6 inhibitor in 315 pts with locally advanced or metastatic HR+/HER2- breast cancer harboring ESR1 mutation  Trial showed a 56% reduction in the risk of disease progression or death (mPFS: 16 vs 9.2mos.), plus showed benefit in the time to second disease progression (25.7 vs 19.1mos.) & a favorable OS trend, which is under assessment. Data were published in The NEJM    Editor’s Take: Beyond the Headlines  Etcamah’s FDA approval strengthens AstraZeneca’s position in ESR1-mutated HR+/HER2− advanced breast cancer by introducing an oral endocrine therapy specifically targeting acquired resistance. Its 56% PFS risk reduction could shift treatment sequencing and intensify competition among endocrine and CDK4/6-based strategies.   Scholar Rock Reports the US FDA Approval of Isembyld (Apitegromab-mstn) to Treat Spinal Muscular Atrophy          Company: Scholar Rock                   Product: Isembyld                 Active Ingredient: Apitegromab-mstn                 Disease: Spinal Muscular Atrophy                 Date: Sep 11, 2026                    Shots:   The US FDA has approved Isembyld for the treatment of spinal muscular atrophy (SMA) in pts (≥2yrs.) who are currently receiving a SMN2-targeted treatment  Approval was backed by the P-III (SAPPHIRE), which met its 1EP of 2.2-point improvement in HFMSE with Isembyld + SMN2-targeted therapy vs PBO at 1yr. in main efficacy population (n=103); 34.2% pts showed a ≥3-point increase in HFMSE vs 13.5%  Isembyld’s US commercial launch is underway, with product shipments expected in the coming days; Scholar Rock also received a Rare Pediatric Disease Priority Review Voucher for a future marketing application    Editor’s Take: Beyond the Headlines  Isembyld’s approval gives Scholar Rock a differentiated position in SMA by targeting muscle function alongside existing SMN2 therapies rather than replacing them. Its add-on approach could expand the addressable market and drive combination use, intensifying competition for patients and treatment budgets among established SMA therapies.  Telix Gains the US FDA Approval for Pixclara for Brain Cancer Imaging           Company: Telix                   Product: Pixclara                 Active Ingredient: floretyrosine F 18                 Disease: FET-PET Imaging for Glioma                 Date: Sep 11, 2026                    Shots:   The US FDA has approved Pixclara (floretyrosine F 18) for pts (≥1mos.) as the FET-PET imaging drug for glioma, enabling PET imaging to help differentiate recurrent or progressive glioma from treatment-related changes  Pixclara comprises of a small-molecule targeting agent & fluorine-18; it binds with LAT1 & LAT2 transporters after IV administration, allowing the radioisotope’s emissions to be detected by PET imaging  The approval expands Telix’s Precision Medicine & theranostics portfolio, while Pixclara (TLX101-Px) is being evaluated in a P-III study for potential expansion into brain metastases diagnosis    Editor’s Take: Beyond the Headlines  Pixclara’s FDA approval strengthens Telix’s position in precision oncology imaging by adding an FET-PET option for glioma assessment. Its ability to distinguish recurrence from treatment-related changes could improve diagnostic decision-making, while Telix’s broader theranostics pipeline may support expansion into additional neuro-oncology imaging applications.  Eli Lilly Reports the US FDA Approval for Onswik to Treat Type 2 Diabetes           Company: Eli Lilly                   Product: Onswik                 Active Ingredient: Insulin efsitora alfa-gobe                Disease: Type 2 Diabetes                 Date: Sep 23, 2026                    Shots:   The US FDA has approved Onswik (insulin efsitora alfa-gobe) along with diet and exercise to control high blood sugar in adults with type 2 diabetes  Approval was based on the P-III (QWINT) trials, which evaluated Onswik (QW) in >3,400 adults with T2D; across each trial, Onswik met the 1EP, demonstrating non-inferior A1C reductions vs insulin glargine U-100 or insulin degludec U-100.  The Onswik KwikPen is expected to launch in the US in the coming mos. in 2 concentrations: 500 units/mL (1,500 units total) & 1,000 units/mL (3,000 units total)  Editor’s Take: Beyond the Headlines  Onswik’s approval strengthens Lilly’s position in diabetes by introducing a once-weekly basal insulin, potentially addressing adherence and injection-burden challenges versus daily insulin regimens. Its non-inferior glycemic control could accelerate adoption among appropriate patients and intensify competition with Novo Nordisk and Sanofi’s established basal insulin franchises.  Elevar Therapeutics Secures the US FDA Approval for Lyrfigtu (Lirafugratinib) to Treat Cholangiocarcinoma           Company: Elevar Therapeutics                    Product: Lyrfigtu                  Active Ingredient: Lirafugratinib                Disease: Cholangiocarcinoma with FGFR2 fusion or rearrangement                 Date: Sep 23, 2026                    Shots:   The US FDA has approved Lyrfigtu for the 2L treatment of cholangiocarcinoma (CCA) patients with FGFR2 fusion or rearrangement; US availability is expected by Q4’26  In the P-I/II (ReFocus) trial, Lyrfigtu showed a cORR of 46%, an mDoR of 11.8mos., & 12mos. PFS rate of 49.2% (mPFS: 11.3mos.), with a predictable and manageable safety profile  Lyrfigtu is being evaluated in a clinical trial enrolling pts with previously treated, advanced or metastatic non-CCA solid tumors harboring FGFR2 fusions or rearrangements who have not received prior FGFR inhibitors   Editor’s Take: Beyond the Headlines  Lyrfigtu’s approval strengthens Elevar’s position in the FGFR2-altered cholangiocarcinoma market, offering a targeted 2L option with durable response potential. Its differentiated profile could challenge existing FGFR inhibitors and expand treatment choice, while broader tumor-agnostic development may create additional growth opportunities for Elevar.   Mirum and Incyte Report FDA Approval of Atebrioz (Zilurgisertib) for Fibrodysplasia Ossificans Progressiva           Company: Mirum Pharmaceuticals and Incyte                 Product: Atebrioz                 Active Ingredient: Zilurgisertib                Disease: Fibrodysplasia Ossificans Progressiva                 Date: Sep 25, 2026                    Shots:   The US FDA has approved Atebrioz (100mg, PO, QD) tablets to reduce the volume of total new heterotopic ossification (HO) in pts (≥12yrs.) with FOP, & issued a Rare Pediatric Disease PRV to Incyte. MAA is under EMA’s review  Approval was based on Cohort 1 of the P-II (PROGRESS) study, where at Wk. 24, mean total new HO lesion volume decreased by 3.2 cm³ with zilurgisertib vs a 24.6 cm³ increase with PBO, with treatment effects maintained through Wk. 48 of the OLE study  PROGRESS program continues to advance, with enrollment completed in Cohort 2 of children (6 to <12yrs.) & underway in Cohort 3 (2 to <12yrs.). Atebrioz will be available through Mirum Access Plus, with US commercial availability expected in Oct 2026    Editor’s Take: Beyond the Headlines   Atebrioz’s approval gives Mirum and Incyte a first-mover position in FOP, addressing a rare disease with limited treatment options. Its demonstrated reduction in new HO formation could establish a new treatment standard, while pediatric expansion may broaden the addressable population and strengthen long-term franchise potential.  AbbVie Reports the US FDA Approval of Juvmo (Tavapadon) for Parkinson’s Disease           Company:  AbbVie                 Product:  Juvmo                 Active Ingredient:  Tavapadon                 Disease:  Parkinson’s Disease                 Date: Sep 25, 2026                   Shots:   FDA approval was based on the P-III (TEMPO) program, which evaluated Juvmo in early PD pts not taking oral levodopa (TEMPO-1 & 2) & as an adjunct to oral levodopa vs PBO + levodopa in PD pts with motor fluctuations (TEMPO-3)  At Wk. 26 in TEMPO-1, Juvmo showed MDS-UPDRS Part II scores of −1.6 (5mg) & −1.7 (15mg) vs +0.9 for PBO, reflecting 22–23% improvement vs 12% worsening; MDS-UPDRS Part II+III combined scores were also improved (−9.7 & −10.2 vs +1.8)   In TEMPO-2, Juvmo improved MDS-UPDRS Part II scores (−1.5 vs 0) & combined Part II+III scores (−10.3 vs −1.2). In TEMPO-3, Juvmo + levodopa increased daily “on” time without troublesome dyskinesia (1.7 vs 0.6hrs.) & reduced “off” time (1.9 vs 0.9hrs.). Efficacy was sustained through 85wks. (TEMPO-4); 93% pts on Juvmo + levodopa did not increase their levodopa dose, while 94% on Juvmo without levodopa did not initiate levodopa   Editor’s Take: Beyond the Headlines  Juvmo strengthens AbbVie’s Parkinson’s disease portfolio with a dopamine D1/D5 receptor agonist offering utility both before and alongside levodopa. Its sustained efficacy and impact on on/off time could differentiate it from established therapies, potentially delaying levodopa escalation and expanding AbbVie’s role across treatment stages.  Egetis Therapeutics Reports the US FDA Approval of Emcitate for MCT8 Deficiency           Company:  Egetis Therapeutics                 Product:  Emcitate                 Active Ingredient:  Tiratricol                 Disease:  Peripheral Thyrotoxicosis in MCT8 Deficiency                 Date: Sep 28, 2026                   Shots:   The US FDA has approved Emcitate (tiratricol) for the treatment of peripheral thyrotoxicosis in adults & pediatric pts with monocarboxylate transporter 8 (MCT8) deficiency (Allan–Herndon–Dudley syndrome)  Approval was supported by an extensive clinical development program assessing Emcitate in pts with MCT8 deficiency, incl. ReTRIACt, Triac Trial I, Triac Trial II, Erasmus Medical Center (EMC) Cohort Study, EMC Survival Study & the US EAP  Emcitate availability is expected in 8-10wks. post approval. Additionally, the US FDA has granted Egetis a Rare Pediatric Disease PRV, which the company expects to potentially monetize in Q4’26  Editor’s Take: Beyond the Headlines  Emcitate’s FDA approval establishes Egetis as a first mover in MCT8 deficiency, addressing a rare and underserved disorder with limited therapeutic options. Broad adult and pediatric use could support rapid adoption, while the Rare Pediatric Disease PRV adds strategic value and strengthens Egetis’ position in rare-disease development.  The post The US FDA New Drug Approvals in September 2026   appeared first on PharmaShots.