LONDON (dpa-AFX) - The U.S. Food and Drug Administration (FDA) approved 13 drugs in September 2026, spanning first-in-class therapies, treatments for rare diseases, and new options for patients with serious conditions.
With the month yielding a mix of new therapies and expanded treatment options, let's take a closer look at the drugs approved, the conditions they target, and the key pricing details behind them.
1. Zanvastro (Zilganersen)
Developed by: Ionis Pharmaceuticals Inc. (IONS)
Approved on: September 3, 2026
Indication: Alexander disease
Alexander disease (AxD) is a rare, progressive, and ultimately fatal neurological disorder that affects one in every 1 to 3 million individuals. The disorder attacks patients' astrocytes, which support neurons in the brain, by introducing variations in the GFAP genes. The disease currently has no cure.
Zanvastro (zilganersen) is an antisense, oligonucleotide RNA- targeting therapy. The drug inhibits excess production of the glial fibrillary acidic protein (GFAP), to restore the function of astrocytes in patients of AxD. The drug was formulated as an intrathecal solution.
The drug was granted a Breakthrough Therapy and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA), in addition to an Orphan Drug designation from both the FDA and European Medicines Agency (EMA).
Positive data from the Phase 3 pivotal study for zilganersen in AxD was reported by the company. The trial assessed 49 pediatric and adult patients of AxD, who showed difficulty walking at baseline. Following treatment, patients demonstrated a meaningful stabilization of gait speed as measured by the 10 metre walk test (10MWT), showcasing improvements in gross motor function at week 61.
Ionis previously announced entering a license agreement with Recordati for the international commercialization of Zilganersen in treating Alexander disease. The drug is now the first and only treatment for AxD.
According to the company, Zanvastro will be available at a wholesale acquisition cost (WAC) of $285,000 per dose.
Stock Quote: IONS has been trading in a range of $42.08 ?to $86.74 in the last 1 year. The stock closed yesterday's trading at $44.12, up 3.33%.
2. Omcazio (cabozantinib)
Developed by: Handa Pharmaceuticals Inc. (6620.TWO)
Approved on: September 3, 2026
Indication: Carcinoma of the kidney and liver, extra-pancreatic neuroendocrine tumors
Carcinomas arise from epithelial tissues of various organs and are the most common forms of malignancies, accounting for 80% to 90% of all cancer diagnoses.
Neuroendocrine tumors, however, originate from cells that regulate hormone release, and can impair signaling between nerve cells as well.
Omcazio is a laurylsulfate salt formulation of the kinase inhibitor cabozantinib, which has previously been approved in other formulations for treating cancers of the thyroid, kidney, liver and pancreas. In this most recently approved composition, Omcazio can be used to treat renal cell carcinoma (RCC) in adults as a monotherapy or in combination with nivolumab.
Additionally, it may be utilized to treat hepatocellular carcinoma (HCC) in adults who have received prior treatment with sorafenib. It is also approved for adult and pediatric patients with previously treated, unresectable, advanced, or metastatic extra-pancreatic neuroendocrine tumors (epNET).
The drug is to be offered in doses of 34.5 mg, 23 mg, and 11.5 mg. CABOMETYX, developed by Exelixis, is the original approved formulation of cabozantinib that generated a U.S. revenue of $2.11 billion in 2025.
Competitors:
DRUG | DEVELOPER | INDICATION | U.S. WAC |
|---|---|---|---|
CABOMETYX | Exelixis Inc. (EXEL) | Neuroendocrine tumors; advanced cancer of the thyroid, kidney, and liver | $26,740 for a 30-day daily dose regimen |
Yervoy | Bristol-Myers Squibb Co. (BMY) | Renal cell carcinoma (RCC); solid tumors | $9,210 per 50mg/10mL vial |
Stock Quote: 6620.TWO has been trading on the Taipei Exchange between TWD 64.10 and TWD 155 over the past 1 year. The stock closed yesterday's trade at TWD 143.90, up 9.96%
3. Etcamah (camizestrant)
Developed by: AstraZeneca PLC (AZN)
Approved on: September 4, 2026
Indication: First line HR+/HER2- breast cancer
Locally advanced or metastatic breast cancers that are Hormone Receptor (HR) positive, Human Epidermal growth factor receptor 2 negative (HER2-) show expression of either estrogen or progesterone receptors, or both, without the overexpression of the HER2 protein. The growth of the breast cancer cells is driven by estrogen receptors like ESR1, which can show resistance to treatment by aromatase inhibitors.
Etcamah (camizestrant) acts as a selective estrogen receptor degrader (SERD) by binding to and breaking down the estrogen receptor to stop tumor resistance. When administered alongside a CDK4/6 inhibitor like abemaciclib, palbociclib, or ribociclib, the cancer growth may be prevented. The drug is designed as a once-daily, oral dose.
The approval was based on an interim analysis of the Phase 3 SERENA-6 trial, which showed the drug's ability to decrease risk of disease progression or death by 56%. Secondary progression-free survival (PFS2) was observed to be 25.7 months, compared to 19.1 months on a standard-of-care treatment.
The full results of the Phase 3 SERENA-6 study are yet to be released, but initial data suggests improved PFS and overall survival (OS) rates.
Competitors:
DRUG | DEVELOPER | INDICATION | U.S. WAC |
|---|---|---|---|
Faslodex (fulvestrant) | Eli Lilly & Co. (LLY); AstraZeneca (AZN) | HR+/HER- advanced or metastatic breast cancer | $1,902 for 10 ml vial |
Inluriyo (imlunestrant) | Eli Lilly & Co. (LLY) | ER+/HER-, ESR-1 mutated advanced or metastatic breast cancer | $10,997 for 28-day daily dose regimen |
Stock Quote: For the past 1 year, shares have traded between $153.41 and $212.71. AZN closed yesterday at $166.68, up 2.87%.
4. Pixclara (floretyrosine F 18)
Developed by: Telix Pharmaceuticals Limited (TLX)
Approved on: September 11, 2026
Indication: Radiodiagnostic FET-PET agent for Glioma
Gliomas constitute the most common type of nervous system cancers, accounting for 80% of all malignant brain tumors. The malignancies grow from the tissues supporting neuronal networks in the brain, and can often increase in size rapidly over a short period of time. Early diagnosis and treatment are thus key to determining a patient's survival.?
Pixclara (floretyrosine F 18 or TLX101-Px) comprises a small molecule labeled with the radioactive isotope fluorine-18, which targets membrane transport proteins called L-type amino acid transporters 1 and 2 (LAT1 and LAT2). These bound compounds can then be detected and visualized on a positron emission tomography (PET) scanning machine.?
The diagnostic agent was previously granted a Fast Track designation by the FDA. With the approval, the drug can now be used to differentiate recurrent and progressive gliomas in adult and pediatric patients aged one month or older.?
A Phase 3 registrational study is currently underway to determine a potential application of Pixclara in detecting brain metastases.?
Competitors:
DRUG | DEVELOPER | INDICATION | U.S. WAC |
|---|---|---|---|
FDOPA (Fluorodopa F18) | Cardinal Health Inc. (CAH) | PET visualiser for Parkinson's disease | $2,605 per batch (single use) |
Tauklarify ( florquinitau F18 injection) | Lantheus Holdings Inc. (LNTH) | PET visualiser for Tau neurofibrillary tangles in Alzheimer's disease | NA |
Stock Quote: Shares have ranged between $6.28 and $13.11 over the past year. TLX closed yesterday at $10.57, down 0.75%.
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5. ISEMBLYD (apitegromab-mstn)
Developed by: Scholar Rock Holding Corp (SRRK)
Approved on: September 11, 2026
Indication: Spinal Muscular Atrophy (SMA)
Spinal Muscular Atrophy (SMA) is a rare genetic neuromuscular disease characterized by irreversible loss of motor neurons, progressive muscle wasting and declining motor function. Existing treatments target the aberrant SMN2 gene to address motor neuron loss.
ISEMBLYD is a recombinant, fully human monoclonal IgG4 antibody designed to target pro-forms of myostatin to prevent the activation and signaling of the protein, thus addressing the muscle component of atrophy. It is administered by intravenous infusion once every four weeks, with a recommended dose of 10 mg/kg.
The approval was based on the Phase 3 SAPPHIRE study, a randomized, double-blind, placebo-controlled trial involving 188 patients of spinal muscular atrophy currently receiving a survival motor neuron 2 (SMN2)-targeted treatment.
The study met its primary endpoint. In the main efficacy population, the drug produced a 2.2-point improvement in the Hammersmith Functional Motor Scale Expanded (HFMSE) at one year compared with placebo. In addition, 34.2% of patients receiving ISEMBYLD achieved a gain of at least three points on HFMSE, compared with 13.5% with placebo.
ISEMBYLD has a wholesale acquisition cost (WAC) of $11,659 for a single-use 150 mg/3 mL vial.
Competitors:
DRUG | DEVELOPER | INDICATION | U.S. WAC |
|---|---|---|---|
SPINRAZA (nusinersen) | Biogen Inc. (BIIB) | SMA | $152,000 for 28 mg vial; $271,000 for 50 mg vial |
EVRYSDI (risdiplam) | Roche Holdings Inc. (RHHBY) | SMA in patients from birth | $35,335.70 per 30-tablet bottle; $14,134.28 per 80 mL |
ZOLGENSMA (onasemnogene abeparvovec) | Novartis AG (NVS) | SMA in pediatric patients with biallelic SMN1 mutations | $2.125 million |
ITVISMA ((onasemnogene abeparvovec-brve) | Novartis AG (NVS) | SMA in adults and pediatric patients with SMN1 mutation | $2.59 million |
Stock Quote: SRRK has traded between $27.07 and $61.76 in the past 1 year. Shares closed yesterday up 2.63% at $40.64.
6. Fayuvi (rebisufligene etisparvovec-hopf)
Developed by: Ultragenyx Pharmaceutical Inc. (RARE)
Approved on: September 17, 2026
Indication: Pediatric patients with Mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome Type A)
Mucopolysaccharidosis type IIIA (MPS IIIA) is an ultra-rare, inherited lysosomal storage disorder caused by mutations in the SGSH gene, resulting in deficiency of the sulfamidase enzyme and accumulation of hepran sulfate. The buildup primarily damages the central nervous system, leading to progressive loss of cognitive, language and motor function and, ultimately, early death.
Fayuvi is a single-dose, intravenous AAV9 gene therapy designed to deliver a functional copy of the SGSH gene to cells. This enables production of the missing sulfamidase enzyme and promotes breakdown of accumulated heparan sulfate. The FDA-recommended dose is 30,000 billion vector genomes/kg, administered as a one-time IV infusion.
The approval was supported by the pivotal Transpher A study and long-term follow-up data. The study enrolled 28 patients across three dose cohorts, with 17 patients in the modified intent-to-treat (mITT) population receiving the highest dose.
In the mITT population, FAYUVI-treated patients had a 23.5-point higher mean Bayley-III cognitive score than an external natural-history cohort of 27 untreated patients.
The U.S. Wholesale acquisition cost for the drug candidate was listed as $3.95 million for the one-time treatment.
There are no other FDA-approved drugs for Sanfilippo syndrome, so the drug has no other comparators in the market.
Stock Quote: RARE ranged between $12.73 to $39.89 for the past one year. The stock closed yesterday at $15.17, up 1.81%.
7. BRAVNETSA (lutetium Lu 177 dotatate)
Developed by: Lantheus Holdings, Inc. (LNTH)
Approved on: September 22, 2026
Indication: Somatostatin receptor-positive (SSTR+) gastroenteropancreatic neuroendocrine tumors (GEP-NETs)
Gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are rare, often slow-growing cancers that develop in the gastrointestinal tract and pancreas. The company estimates that approximately 200,000 people in the U.S. are living with GEP-NETs.
BRAVNESTA is a radiopharmaceutical and radioligand therapy that targets somatostatin receptors expressed on GEP-NET cells and delivers radiation directly to the tumors.
The drug was approved through the FDA's Abbreviated New Drug Application (ANDA) pathway. It is the first radioligand therapy approved through the ANDA pathway and the only radiopharmaceutical that the FDA has determined to be bioequivalent and therapeutically equivalent to LUTATHERA, developed by Novartis.
BRAVNETSA is administered intravenously at 7.4 GBq (200 mCi) every eight weeks for a total of four doses. Long-acting octreotide is administered between four and 24 hours after each BRAVNETSA dose.
Competitors:
Drug | Developer | Indication | U.S. WAC |
|---|---|---|---|
LUTATHERA (lutetium Lu 177 dotatate) | Novartis (NVS) | Adult patients with SSTR-positive GEP-NETs | $55,896 per dose |
BEXLUTRY (lutetium Lu 177 dotatate) | Curium Pharma | Adult patients with SSTR-positive GEP-NETs | NA |
Stock Quote: LNTH has traded over the past year in the range of $50.15 to $111.86. Shares closed down 0.01% yesterday at $99.99.
8. LYRFIGTU (lirafugratinib)
Developed by: Elevar Therapeutics, Inc.
Approved on: September 23, 2026
Indication: Metastatic Cholangiocarcinoma
Cholangiocarcinoma, also known as bile duct cancer, is a rare cancer that develops in the bile ducts. Approximately 8,000 people in the U.S. are newly diagnosed each year, according to the American Cancer Society (ACS).
Lyrfigtu, or lirafugratinib, is an oral, selective FGFR2 kinase inhibitor that blocks FGFR2 signaling involved in the growth and survival of cancer cells. The drug is administered at a recommended dose of 70 mg once daily.
The FDA approval was based on results from the Phase 1/2 ReFocus trial, which evaluated the drug candidate in 116 patients with unresectable or metastatic cholangiocarcinoma who had received prior chemotherapy or chemoimmunotherapy and had not previously received an FGFR inhibitor.
The study reported a 46% confirmed objective response rate (ORR) and a median duration of response of 11.8 months. Median progression-free survival was 11.3 months, with 49.2% of patients remaining progression-free at 12 months.
Lyrfigtu's U.S. wholesale acquisition cost (WAC) has not yet been publicly disclosed by Elevar.
Competitors:
DRUG | DEVELOPER | INDICATION | U.S. WAC |
|---|---|---|---|
PEMAZYRE (pemigatinib) | Incyte Corp (INCY) | Metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement | $18,000 per 28-day cycle |
LYTGOBI (futibatinib) | Taiho Oncology | Metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement | $19,000 per 28-day cycle |
9. Onswik (insulin efsitora alfa-gobe)
Developed by: Eli Lilly and Company (LLY)
Approved On: September 23, 2026
Indication: Improve glycemic control in adults with Type 2 diabetes mellitus as an adjunct to diet and exercise
Type 2 diabetes is a chronic condition in which blood glucose levels become elevated because the body does not produce enough insulin or does not use insulin effectively. Insulin helps regulate blood glucose by facilitating glucose uptake into cells and reducing glucose production by the liver.
Onswik, or insulin efsitora alfa-gobe, is a long-acting insulin analog designed to provide glycemic control with once-weekly subcutaneous administration. It is administered under the skin on the same day each week and is available in 500 units/mL and 1000 units/mL strengths in prefilled pens.
The approval was supported by four randomized, open-label, active-controlled Phase 3 studies that evaluated Onswik in adults who were either new to insulin treatment or previously treated with basal insulin, with or without mealtime insulin. Across the studies, once-weekly Onswik reduced HbA1c to a similar extent as once-daily insulin glargine or insulin degludec.
Onswik is the second once-weekly insulin approved in the U.S. for adults with type 2 diabetes.
Competitors:
Drug | Developer | Indication | U.S. WAC |
|---|---|---|---|
Awiqli (insulin icodec-abae) | Novo Nordisk (NVO) | Adults with type 2 diabetes to improve glycemic control as an adjunct to diet and exercise | NA |
Stock Quote: Eli Lilly has been trading in the range of $783.85 to $1,292.65 in the past 1 year. The stock closed yesterday up 3.55% at $1,251.61.
10. JUVMO (tavapadon)
Developed by: Abbvie Inc. (ABBV)
Approved on: September 25, 2026
Indication: Parkinson's disease in adults
Parkinson's disease is a progressive neurological disorder characterized by tremor, muscle rigidity, slowness of movement, and balance problems. More than 11 million people worldwide are living with the disease. As Parkinson's progresses, patients may experience motor fluctuations, including periods when symptoms are controlled 'on' and when symptoms turn 'off', as well as dyskinesia.
JUVMO (tavapadon) is a once-daily oral therapy that can be used with or without levodopa. It is the first and only selective D1/D5 receptor agonist approved for adults with Parkinson's disease.
Tavapadon is a selective D1/D5 dopamine receptor partial agonist designed to activate dopamine pathways involved in motor control. Unlike currently available agonists that primarily target D2/D3 receptors, JUVMO selectively targets D1/D5 receptors.
The approval was supported by the Phase 3 TEMPO program, in which JUVMO improved daily functioning in early Parkinson's disease and increased daily 'on' time without troublesome dyskinesia when added to levodopa in patients with motor fluctuations.
Abbvie has not publicly announced a WAC for the drug candidate as of the FDA approval announcement. The company expects the drug to become available in the U.S. in October 2026.
Competitors:
Drug | Developer | Indication | U.S. WAC |
|---|---|---|---|
Mirapex (pramipexole) | Boehringer Ingelheim | Parkinson's disease | NA |
Requip (ropinirole) | GlaxoSmithKline (GLAXO.NS) | Parkinson's disease | NA |
Neupro (rotigotine) | UCB SA (UCB.BR) | Parkinson's disease | NA |
Stock Quote: ABBV spanned $190.75 to $291.33 per share in the past year. Shares closed yesterday at $283.40, up 0.59%.
11. Atebrioz (zilurgisertib)
Developed by: Mirium Pharmaceuticals Inc. (MIRM) and Incyte Corporation (INCY)
Approved on: September 25, 2026
Indication: Heterotopic ossification associated with fibrodysplasia ossificans progressiva
Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disorder that causes bone formations in the middle of soft tissues, which is also known as heterotopic ossification (HO).
Atebrioz acts by inhibiting the ALK2 receptor, which is unusually active in individuals with FOP, leading to HO lesions through the body. The drug was formulated as a once-daily oral therapy.
The approval was validated by results from Cohort 1 of the PROGRESS study that evaluated zilurgisertib in adults and children aged 12 years or older. The trial determined that dosed patients saw the mean total HO lesion volume decrease by 3.2 cubic centimetres, whereas those on a placebo therapy experienced a lesion expansion of 24.6 cubic centimetres.
The PROGRESS study is currently ongoing for two pediatric cohorts.
Atebrioz may carry an estimated annual cost of $750,000, according to a Citizens analyst.
Competitors:
DRUG | DEVELOPER | INDICATION | U.S. WAC |
|---|---|---|---|
Sohonos (palovarotene) | Ipsen Biopharmaceuticals Inc. (IPSN) | Fibrodysplasia ossificans progressiva (FOP) | $5,051 for 14 capsules |
Stock Quote: MIRM has traded between $63.23 to $130 per share in the past year. Shares closed yesterday up 3.04% at $82.39. INCY has ranged between $84.02 and $132.60 in the past year. The stock closed yesterday at $113.27, up 1.52%.
12. Degevma (denosumab-adet)
Developed by: Teva Pharmaceuticals USA Inc. (TEVA)
Approved on: September 25, 2026
Indication: Skeletal-related events linked to bone cancers and hypercalcemia
Primary bone cancer or cancer that spreads to the bones can have severe, life-altering consequences for patients. Multiple myeloma that metastasizes can cause osteolytic bone lesions, while giant bone tumors can impact mobility and the structural strength of the skeletal region.?
Degevma (denosumab-adet) is a RANK ligand (RANKL) inhibitor that can be prescribed to treat metastases to the bone or bone tumors in cases where surgical resection is impossible or likely to cause morbidity. The drug is a biosimilar to Xgeva, a composition of denosumab that received approval in 2010, and already has nine other approved biosimilars on the market.?
In addition to malignancies, Degevma can also be used to treat hypercalcemia that has shown resistance to biphosphonate therapy.?
Competitors:
DRUG | DEVELOPER | INDICATION | U.S. WAC |
|---|---|---|---|
WYOST | Sandoz Group AZ (SDZ) | Bone metastases; multiple myeloma | $3,142 for 1.7 mL of subcutaneous solution |
Xtrenbo | Hikma Pharmaceuticals PLC (HIIK) | Giant cell tumor of bone; hypercalcemia | $477 for 1.7 mL of subcutaneous solution |
Bilprevda (denosumab-nxxp) | Organon & Co. (OGN) | Bone metastases; multiple myeloma | $1,688 per dose |
Stock Quote: Shares have ranged between $18.83 and $42.02 per share in the past 1 year. TEVA closed yesterday up 0.96% at $41.11.
13. Emcitate (tiratricol)
Developed by: Egetis Therapeutics AB (EGTX)
Approved on: September 28, 2026
Indication: Peripheral thyrotoxicosis MCT8 deficiency
Monocarboxylate transporter 8 (MCT8) is a membrane protein that facilitates the transport of thyroid hormones, particularly triiodothyronine (T3), across cell membranes and the blood-brain barrier. MCT8 deficiency, also known as Allan-Hernon-Dudley Syndrome, is an X-linked disorder that leads to mutations in the SLC16A2 gene.
The deficiency thus causes hypothyroidism or peripheral thyrotoxicosis, which may result in neurodevelopmental delays and cardiac arrhythmia. ? Emcitate was designed as a thyroid hormone receptor agonist which can enter cells regardless of the absence of MCT8 proteins. The drug enters cells with the aberrant gene and restores thyroid hormone signaling. It can?be administered orally or through an enteral feeding tube.?
The FDA approval was supported by positive results gained from the ReTRIACt trial, in addition to a Cohort and Survival study conducted under a U.S. Expanded Access Program (EAP).?
Competitors:
DRUG | DEVELOPER | INDICATION | U.S. WAC |
|---|---|---|---|
Teatrois | Cenexi/DB Pharma | Thyroid dysfunction, including MCT8 deficiency | EUR 0.35 to EUR 0.65 per dose |
Stock Quote: EGTX.ST has traded on the Stockholm Exchange between SEK 3.62 and SEK 10.14 in the past year. Shares closed yesterday up 1.95% at SEK 9.39.
Did You Know?
This month marked 36 years since the first administration of a gene therapy. On September 14, 1990, four-year-old Ashanthi DeSilva became the first patient to receive gene therapy for a genetic disease. She had adenosine deaminase deficiency leading to severe combined immunodeficiency (ADA-SCID), a rare inherited immune disorder. Researchers introduced a functional copy of the ADA gene into her immune cells, helping improve her T-cell counts and overall health. The landmark treatment helped pave the way for decades of gene therapy research and the development of treatments for genetic diseases.?
Related Reading:
New FDA Drug Approvals - August 2026
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