FDA approvals: Fayuvi, Inluriyo and Bexlutry lead the Sep. 12–18 ledger
A date-bounded review of Fayuvi's first-ever gene therapy for Sanfilippo syndrome type A, Lilly's Inluriyo-Verzenio combination, Curium's radioligand equivalent Bexlutry, and the captured CDRH device ledger for September 12–18, 2026.
The FDA approval window from September 12 through September 18, 2026 produced three therapeutic approvals worth acting on: the first-ever treatment for a fatal childhood brain disease, an oral combination for an ESR1-mutated breast cancer subgroup, and the first competing copy of a radiopharmaceutical cancer therapy. A fourth record, an imaging drug carrying a September 11 decision date, reached the agency's public tables after last week's issue went out. 12
Two boundaries shape this issue. The agency's two-week approvals report carried drug actions dated September 4 through September 16, so the September 17 and September 18 approvals rest on FDA's own announcements. The monthly 510(k) file was refreshed on September 14 with item-level decisions through September 13, so the clearances granted from September 14 onward appear below from the applicants and still need re-checking against the file. 34
At a glance
Eight items, sourced from the FDA's own Sanfilippo and imlunestrant approval notices and from the approval or clearance announcements made by Curium, Telix, Medtronic, Microstructure Imaging, Beta Bionics and Pace Surgical. 56789101112
| Action | FDA date | Indication or intended use | Sponsor or applicant | Commercial and clinical read |
|---|---|---|---|---|
| Fayuvi (rebisufligene etisparvovec-hopf) | Sep. 17 | Neurologic manifestations of MPS IIIA in pediatric patients with preserved neurodevelopmental function | Ultragenyx (NASDAQ: RARE) | First approved treatment for the disease; one-time intravenous gene therapy at $3.95 million; ships to treatment centers within 30–60 days. |
| Inluriyo (imlunestrant) with Verzenio (abemaciclib) | Sep. 18 | ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer after one endocrine therapy line | Eli Lilly (NYSE: LLY) | Median progression-free survival 11.1 months against 5.5; Guardant360 CDx is the companion test; competes with AstraZeneca's camizestrant. |
| Bexlutry (lutetium Lu 177 dotatate) | Sep. 14 | Adults with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors | Curium | First radioligand equivalent to Novartis's Lutathera, whose first-half 2026 sales were $436 million; no price announced. |
| Pixclara (floretyrosine F 18) | Sep. 11 | Differentiating recurrent or progressive glioma from treatment-related change, age 1 month and older | Telix Pharmaceuticals | First approved FET-PET imaging drug for glioma in the United States, against about 24,000 new U.S. glioma cases a year. |
| LigaSure RAS Maryland instrument on the Hugo RAS system | Sep. 16 | Vessel sealing and cutting up to 7 mm during robotic-assisted urologic surgery | Medtronic (NYSE: MDT) | Puts Medtronic's laparoscopic vessel sealer on Medtronic's own surgical robot, whose U.S. label still covers urology alone. |
| MICSI-PET | Sep. 16 | MRI-guided enhancement and quantification of neurological PET scans | Microstructure Imaging | First FDA-cleared MRI-guided PET software per the company; cleared across FDG, amyloid and tau tracers. |
| Mint patch pump | Sep. 14 | Automated insulin delivery for people with diabetes | Beta Bionics (NASDAQ: BBNX) | Launch pulled forward to the first quarter of 2027; 2026 revenue guidance cut to $121–126 million. |
| Tempo variable locking plating system | Sep. 17 | Internal fixation of fractures, osteotomies and fusions in the foot, ankle, hand and wrist | Pace Surgical | New entrant in foot and ankle fixation, with an alpha launch planned for early 2027. |
Drug and biologic approvals
Fayuvi becomes the first treatment for Sanfilippo syndrome type A
The FDA approved Fayuvi (rebisufligene etisparvovec-hopf) on September 17 for pediatric patients with mucopolysaccharidosis type IIIA, also called Sanfilippo syndrome type A. The disease is inherited and progressively destroys cognitive, language and motor function, and until now treatment meant managing symptoms. 5
The therapy is given once, intravenously. A modified, non-infectious adeno-associated virus serotype 9 vector carries a working copy of the SGSH gene into the patient's cells, which then produce sulfamidase, the enzyme missing in this disease. The label covers the neurologic manifestations of MPS IIIA in pediatric patients with preserved neurodevelopmental function, a narrower population than the disease itself. 513
The evidence comes from the single-arm Transpher A trial, which followed 17 treated patients aged 2 to 5 against 27 untreated patients from a comparable natural-history cohort for nearly eight years. Treated patients scored 23.5 points higher on the change in Bayley-III Cognitive raw score between 24 and 60 months of age, and the result supported a standard full approval. 513
Monitoring sits with the treatment center. Liver enzyme increase was the most frequent adverse reaction at 85%, ahead of vomiting at 67%, with a long tail of blood count, metabolic and neurological reactions. Warnings cover hepatotoxicity, falling platelet counts, infusion and hypersensitivity reactions, and the risk of malignancy from vector DNA integrating into the genome; thrombotic microangiopathy has appeared with other AAV gene therapies, so the label asks for monitoring for it. Every patient receives corticosteroids from one day before the infusion through at least eight weeks after, platelets are checked weekly for four weeks and then monthly for six months, and vaccines are avoided around the corticosteroid course. FDA granted Orphan Drug, Fast Track and Breakthrough Therapy designations. 513
Commercial read
Ultragenyx set a list price of $3.95 million, and the approval landed slightly ahead of a September 19, 2026 PDUFA date. The company estimates the disease affects roughly 3,000 to 5,000 patients across commercially accessible geographies, with a median life expectancy of about 15 years, and expects commercial product to ship to Qualified Treatment Centers within 30 to 60 days, supported by an UltraCare access program. 1314
Investors took the approval as a win sized against the rest of the pipeline. Ultragenyx shares rose 12.6% on September 17, and Canaccord raised its price target to $39 from $37 while keeping a Buy rating, with attention staying on a pending cost-cutting update and on monetizing the company's two priority review vouchers. 1415
The asset carries a royalty: Ultragenyx in-licensed the program from Abeona Therapeutics in 2022, with Abeona taking tiered royalties from the mid-single digits up to 8% of net sales and up to $30 million in commercial milestones. Abeona shares rose 4.1% that day. One offsetting item sits beside the approval: the Phase 3 Aspire trial of apazunersen (GTX-102) in Angelman syndrome missed its endpoints, after which H.C. Wainwright, Canaccord and Wells Fargo cut their Ultragenyx price targets. 1415
Inluriyo plus Verzenio, approved September 18
The FDA approved imlunestrant (Inluriyo) with abemaciclib (Verzenio) on September 18 for adults with estrogen receptor-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test, after progression on at least one line of endocrine therapy. Both drugs are Eli Lilly products, and the agency approved the Guardant360 CDx assay as the companion diagnostic that identifies the qualifying ESR1 mutations. 6
The evidence comes from EMBER-3, which enrolled 874 adults previously treated with an aromatase inhibitor alone or with a CDK4/6 inhibitor and randomized them 1:1:1 to imlunestrant, to an investigator's choice of fulvestrant or exemestane, or to the combination, with ESR1 status read from blood circulating tumor DNA and limited to specific ligand-binding-domain mutations. In an exploratory analysis of 159 patients with ESR1-mutated tumors, the combination cut the risk of progression at a hazard ratio of 0.53, with a 95% confidence interval of 0.35 to 0.80. Median progression-free survival was 11.1 months against 5.5 months for imlunestrant alone, and objective response rates were 35% against 15%. Overall survival was immature, with 35% of that subgroup having died at the interim analysis. 6
The FDA's review states the limit of the finding: imlunestrant monotherapy showed no progression-free survival improvement over investigator's choice of endocrine therapy in either the overall population or the ESR1-mutation-not-detected population, which places the benefit in the ESR1-mutated group alone. Dosing is 400 mg of imlunestrant once daily on an empty stomach with 150 mg of abemaciclib twice daily, continued until progression or unacceptable toxicity, and the warnings cover embryo-fetal toxicity for imlunestrant and, for abemaciclib, diarrhea, neutropenia, interstitial lung disease or pneumonitis, hepatotoxicity, venous thromboembolism and embryo-fetal toxicity. 6
Commercial read
This is Lilly's second approval for Inluriyo in under a year; the drug was first approved in September 2025 as a monotherapy in the same population, and the combination is now available in the United States. 16
The competitive field formed two weeks earlier. AstraZeneca's camizestrant (Etcamah) took accelerated approval on September 4 for hormone receptor-positive, HER2-negative, locally advanced or metastatic breast cancer when an ESR1 mutation is detected during aromatase inhibitor and CDK4/6 inhibitor therapy. Two oral endocrine approaches now target the same post-aromatase-inhibitor, ESR1-mutated population, and both depend on a blood-based ESR1 result before a patient qualifies. 1
Bexlutry, the first radioligand equivalent
Curium's Bexlutry (lutetium Lu 177 dotatate) was approved on September 14 for adults with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors, including foregut, midgut and hindgut tumors, and the company says it is available for prescribing. The therapy binds somatostatin receptors on those tumors and delivers targeted radiation; GEP-NETs make up 60% to 70% of all neuroendocrine tumors. 7
The approval ran through the FDA's 505(b)(2) pathway as a radioligand equivalent to Novartis's Lutathera, supported by published evidence and bridging data showing a comparable biological and chemical profile; the agency's report files NDA 218525 as a new formulation or new manufacturer and renders the ingredient as "lutetium Lu 177 donatate," while the label and the company use "dotatate." Efficacy and safety both rest on the reference product, whose label warns of radiation exposure, myelosuppression, secondary myelodysplastic syndrome and leukemia, renal and hepatic toxicity, hypersensitivity, neuroendocrine hormonal crisis, embryo-fetal toxicity and infertility. In NETTER-1, anemia, thrombocytopenia and neutropenia all ran several times higher on the therapy than on high-dose octreotide alone, and MDS was reported in 2.3% of the treated arm over a median 76 months. Patients stop long-acting somatostatin analogs four weeks and short-acting octreotide 24 hours before each dose and receive an amino acid solution around the infusion to cut the radiation dose to the kidneys. 27
Commercial read
The reference product sets the size of the market: Lutathera, approved in January 2018 as the first radioligand therapy, generated $436 million in sales for Novartis in the first half of 2026, and Curium's announcement came without a price. 17
Novartis has told investors to expect substitution to behave differently here. On the company's second-quarter earnings call, chief executive Vas Narasimhan said the radioligand copycat market will work differently from generics and biosimilars given the logistical complexity, and that Novartis's supply network had mitigated the impact of a generic launch even at a lower price. Curium describes itself as the only vertically integrated manufacturer of lutetium-based therapies for neuroendocrine tumors. 717
A second entrant is already queued: Lantheus holds a tentative approval from March for PNT2003, a Lutathera generic filed under the abbreviated new drug application pathway, and Curium is acquiring Lantheus in a deal worth up to $8 billion announced on August 3. Curium's own application, filed in July 2024, waited on litigation that a June court decision resolved in its favor. 17
Pixclara, a September 11 record that reached the tables late
Pixclara (floretyrosine F 18) carries a September 11 approval date, one day outside this issue's window. It appears here because FDA's novel-drug table now lists it as the year's fortieth novel approval and last week's issue did not cover it; Telix announced the approval on September 13 and 14. 18
The drug is an intravenous PET imaging agent used to differentiate recurrent or progressive glioma from treatment-related change in adults and pediatric patients 1 month and older, and its fluorine-18-labelled small molecule targets L-type amino acid transporters 1 and 2, which tumor tissue overexpresses. Safety was evaluated in 382 patients with gliomas, among whom headache was the only adverse reaction reaching 0.5%. Gliomas account for roughly 30% of all central nervous system tumors and 80% of malignant brain tumors, with about 24,000 new U.S. cases a year, and Telix describes Pixclara as the only approved radiopharmaceutical imaging drug for glioma. The FDA granted fast-track designation in April 2024 and later required additional confirmatory evidence, which Telix supplied in a resubmitted application; a Phase 3 study is running for a possible brain-metastases indication. 818
Other captured Drugs@FDA actions
The two tables below carry the rest of the week's drug record, and the field values are exactly what the agency's report lists. 2
Original applications
| Application | Product | Dosage form | Applicant | Date | Action |
|---|---|---|---|---|---|
| BLA 761497 | Garzulys (insulin aspart-fsan) | Pen, prefilled; vial, multidose | Emerge Bioscience Pte. Ltd. | Sep. 15 | Original, ORIG-2 |
| ANDA 220657 | Epinephrine | Solution, intramuscular, intravenous, subcutaneous | Difgen Pharms | Sep. 15 | Original, ORIG-1 |
| ANDA 219832 | Epinephrine | Solution, intramuscular, intravenous, subcutaneous | Zydus Pharms | Sep. 14 | Original, ORIG-1 |
| ANDA 207399 | Milnacipran hydrochloride | Tablet, oral | Strides Pharma | Sep. 15 | Original, ORIG-1 |
| ANDA 210944 | Milnacipran hydrochloride | Tablet, oral | Macleods Pharms | Sep. 15 | Original, ORIG-1 |
| ANDA 218560 | Tretinoin | Cream, topical | Zydus Lifesciences | Sep. 15 | Original, ORIG-1 |
| ANDA 214418 | Beclomethasone dipropionate | Aerosol, metered, inhalation | Aurolife Pharma | Sep. 14 | Original, ORIG-1 |
| ANDA 218210 | Haloperidol | Tablet, oral | Ajanta Pharma | Sep. 14 | Original, ORIG-1 |
| ANDA 220438 | Letrozole | Tablet, oral | Zydus Lifesciences | Sep. 14 | Original, ORIG-1 |
Commercial read: These add supply rather than new mechanisms. Two more epinephrine injection suppliers join a category short of redundancy, and the tretinoin cream, milnacipran and letrozole approvals widen multi-source competition in dermatology, chronic pain and breast cancer endocrine therapy. Garzulys is a subsequent original application for insulin aspart from a Singapore-based applicant.
Efficacy and labeling supplements
| Application | Product | Active ingredient | Sponsor | Submission | Scope | Date |
|---|---|---|---|---|---|---|
| NDA 219713 | Ibtrozi | Taletrectinib adipate | Nuvation | SUPPL-4 | Efficacy | Sep. 16 |
| NDA 218197 | Truqap | Capivasertib | Astrazeneca | SUPPL-6 | Efficacy | Sep. 16 |
| NDA 208065 | Tagrisso | Osimertinib mesylate | Astrazeneca | SUPPL-35 | Efficacy | Sep. 14 |
| NDA 203214 | Xeljanz | Tofacitinib citrate | Pfizer | SUPPL-42 | Labeling | Sep. 16 |
| NDA 213082 | Xeljanz | Tofacitinib citrate | Pfizer | SUPPL-15 | Labeling | Sep. 16 |
| NDA 208246 | Xeljanz XR | Tofacitinib citrate | Pfizer | SUPPL-28 | Labeling | Sep. 16 |
| ANDA 202862 | Olanzapine | Olanzapine | Macleods Pharms Ltd | SUPPL-56 | Labeling | Sep. 16 |
| NDA 204441 | Jynarque | Tolvaptan | Otsuka | SUPPL-16 | Labeling | Sep. 15 |
| ANDA 075945 | Amnesteem | Isotretinoin | Mylan Pharms Inc | SUPPL-45 | Labeling | Sep. 15 |
| ANDA 078592 | Carbamazepine | Carbamazepine | Teva Pharms | SUPPL-6 | Labeling | Sep. 15 |
| ANDA 211715 | Dextroamphetamine mixed salts | Amphetamine salts, extended release | Sun Pharm Industries | SUPPL-12 | Labeling | Sep. 15 |
| ANDA 090241 | Pramipexole dihydrochloride | Pramipexole dihydrochloride | Chartwell Rx | SUPPL-6 | Labeling | Sep. 15 |
| ANDA 209390 | Testosterone | Testosterone | TWi Pharms | SUPPL-11 | Labeling | Sep. 15 |
| ANDA 091620 | Tigecycline | Tigecycline | Sandoz | SUPPL-14 | Labeling | Sep. 15 |
| ANDA 214020 | Tigecycline | Tigecycline | Meitheal | SUPPL-6 | Labeling | Sep. 15 |
| NDA 213535 | Evrysdi | Risdiplam | Genentech Inc | SUPPL-18 | Labeling | Sep. 14 |
| NDA 219285 | Evrysdi | Risdiplam | Genentech Inc | SUPPL-3 | Labeling | Sep. 14 |
| NDA 208065 | Tagrisso | Osimertinib mesylate | Astrazeneca | SUPPL-36 | Labeling | Sep. 14 |
| ANDA 205080 | Itraconazole | Itraconazole | Amneal Pharms | SUPPL-20 | Labeling | Sep. 14 |
| ANDA 206741 | Itraconazole | Itraconazole | Alembic | SUPPL-22 | Labeling | Sep. 14 |
| ANDA 040088 | Methadone hydrochloride | Methadone hydrochloride | Vistapharm Llc | SUPPL-33 | Labeling | Sep. 14 |
| ANDA 212968 | Neostigmine methylsulfate | Neostigmine methylsulfate | Gland | SUPPL-2 | Labeling | Sep. 14 |
| ANDA 203924 | Primaquine phosphate | Primaquine phosphate | Alvogen | SUPPL-2 | Labeling | Sep. 14 |
| ANDA 204476 | Primaquine phosphate | Primaquine phosphate | Unichem | SUPPL-7 | Labeling | Sep. 14 |
| ANDA 211711 | Clobazam | Clobazam | Micro Labs | SUPPL-7 | Manufacturing (CMC) | Sep. 14 |
The three efficacy supplements are the ones that change what a drug is for, and for all three the revised prescribing information has to be the source for the changed indication, population or dosing, because the submission type records only that the sponsor filed for an efficacy change.
Medical devices
The device ledger splits by evidence type. Three clearances carry item-level FDA records dated September 12 and 13, along with one premarket approval supplement dated September 13. Four more were announced by their applicants between September 14 and September 18, after the monthly file was refreshed, and those records have to be re-checked next issue. 34
Clearances announced September 14–18
Medtronic LigaSure RAS Maryland jaw was cleared on September 16 for use with the Hugo robotic-assisted surgery system. It seals and cuts vessels, thick tissue and lymphatics up to 7 mm, sealing in about two seconds, and works with the Valleylab FT10 energy platform. Medtronic describes it as the first robotic-driven LigaSure instrument; Hugo is cleared in the United States for urologic procedures, with general and gynecologic surgery still to come. 9
MICSI-PET, reported September 16, is software that uses structural MRI to guide enhancement and automated quantification of neurological PET, cleared across FDG, the amyloid tracers florbetapir, florbetaben and flutemetamol, and the tau tracer flortaucipir. Its cleared functions include MRI-guided super-resolution and denoising for diagnostic use, automated Centiloid and CenTauR quantification, regional standardized uptake value ratios against normative controls, and quality control across the pipeline. Microstructure Imaging, a spinoff of NYU Langone Health, describes it as the first FDA-cleared MRI-guided PET software and the first FDA-cleared CenTauR implementation. 10
Beta Bionics Mint, announced September 14, is a patch pump with a reusable and disposable two-piece architecture, smartphone control and compatibility with the Dexcom G7, Dexcom G7 15 Day and Abbott FreeStyle Libre 3 Plus monitors, running three days with a 12-hour grace period on a 200-unit reservoir. Full U.S. commercialization is expected in the first quarter of 2027, earlier than planned, through the pharmacy channel, with about 40% of insured U.S. lives covered at launch. Beta Bionics cut its 2026 revenue guidance to $121–126 million from $131–136 million for up to $10 million of fourth-quarter deferral as prospective users wait for Mint, and the shares rose 2.2% that day. 11
Pace Surgical Tempo, cleared September 17, is an internal fixation platform for the foot, ankle, hand and wrist that lets surgeons set screw trajectory during the procedure while keeping the stability of a fixed-angle construct, covering fractures, osteotomies, joint fusion and revision. Pace Surgical plans an alpha launch in early 2027. 12
The captured CDRH ledger, September 12–13
The table comes from the official CDRH decision records for each 510(k) and for PMA supplement P150031/S084; the monthly 510(k) file and the PMA decision feed set the file boundaries. 19202122
| Number | Device | Applicant | Decision date | Classification |
|---|---|---|---|---|
| K260133 | CADDIE | Odin Medical Limited, London | Sep. 12 | Gastrointestinal lesion software detection system; 21 CFR 876.1520; product code QNP; Special review; Gastroenterology and Urology panel. Substantially equivalent. |
| K261287 | Digistat Care | Ascom Ums Srl Unipersonale, Scandicci, Italy | Sep. 12 | Network and communication system for physiological monitors; 21 CFR 870.2300; product code MSX; Traditional review; Cardiovascular panel. Substantially equivalent. |
| K262526 | Safety lancet (XL) | Tianjin Huahong Technology Co., Ltd., Tianjin | Sep. 13 | Single-use blood lancet with integral sharps injury prevention; 21 CFR 878.4850; product code FMK; Traditional review; General and Plastic Surgery panel. Substantially equivalent. |
| P150031/S084 | Vercise deep brain stimulation system | Boston Scientific Corp | Sep. 13 | Normal 180-day track, labeling change. Adds the Vercise Cartesia X and HX 16-contact directional leads, the 16-contact lead extensions and the 8x2-contact lead extensions of the Vercise Genus DBS system to the configurations approved for 1.5 T MRI scans. Neurology panel. |
The FDA detail pages for these three 510(k) records carry no summary text or statement of intended use, so the cleared scope of CADDIE, Digistat Care and the XL safety lancet rests on the classification entry alone. The PMA feed showed no other decision dated between September 12 and September 18. 419
What matters next
- Fayuvi launch execution. Watch when the Qualified Treatment Center network opens, how payers handle a $3.95 million one-time therapy, and whether Ultragenyx sells, transfers or holds its two priority review vouchers.
- ESR1-mutated breast cancer turns competitive. Inluriyo with Verzenio and AstraZeneca's camizestrant now chase the same post-aromatase-inhibitor population, and both need a blood ESR1 result before a patient qualifies, so testing volume and coverage of the Guardant360 CDx assay will move before prescribing does.
- Bexlutry pricing and the Lantheus combination. Curium has published no price for the first radioligand equivalent, and its up-to-$8 billion acquisition of Lantheus would consolidate the second entrant, PNT2003, under the same owner. How Novartis defends Lutathera's $436 million first-half run rate is the number to track.
- Device ledger recheck. The next issue should confirm item-level records for the LigaSure RAS, MICSI-PET, Mint and Tempo clearances, and fill in any other 510(k) decisions FDA dated from September 14 onward.
References
- 1
- 2Recent New and Generic Drug Approvals
accessdata.fda.gov
- 3Downloadable 510(k) Files
fda.gov
- 4FDA PMA Decisions Feed
accessdata.fda.gov
- 5
- 6
- 7Curium announces FDA approval of BEXLUTRY
globenewswire.com
- 8FDA Approves Telix's Brain Cancer Imaging Drug Pixclara
telixpharma.com
- 9Medtronic announces FDA clearance for LigaSure RAS Maryland instrument
news.medtronic.com
- 10MICSI secures FDA clearance for MR-guided PET platform
auntminnie.com
- 11
- 12
- 13Ultragenyx Announces Approval of FAYUVI
ir.ultragenyx.com
- 14
- 15
- 16FDA approves Lilly's Inluriyo-Verzenio combo for breast cancer
ca.investing.com
- 17
- 18FDA clears new PET agent for brain cancer imaging
auntminnie.com
- 19FDA 510(k) record K260133
accessdata.fda.gov
- 20FDA 510(k) record K261287
accessdata.fda.gov
- 21FDA 510(k) record K262526
accessdata.fda.gov
- 22FDA PMA supplement record P150031/S084
accessdata.fda.gov
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