FDA approvals: Emcitate, pediatric Camzyos and the Autus valve lead the Sep. 26–Oct. 2 ledger
A date-bounded ledger of the FDA's September 26–October 2, 2026 decisions, led by the first-ever approval for MCT8 deficiency, the first pediatric indication for a cardiac myosin inhibitor, and the first heart valve that can be widened as a child grows.
The FDA had never approved a treatment for MCT8 deficiency until September 28, when it cleared Emcitate (tiratricol) for the rare X-linked disorder also known as Allan–Herndon–Dudley syndrome. Two days later, on September 30, the agency widened Camzyos (mavacamten) to children with symptomatic obstructive hypertrophic cardiomyopathy, the first cardiac myosin inhibitor cleared for that population. Then October 1 brought the week's most unusual device decision: the Autus Size-Adjustable Valve, the first heart valve the agency has approved that surgeons can widen as a child grows, and the first approved valve whose leaflets are made of polymer instead of animal tissue. 123
Three capture boundaries shape how complete that picture is. FDA's two-week approvals report ran through October 1, so the September 28 and September 30 drug decisions sit inside it and any October 2 action does not. On the device side, the monthly 510(k) file was refreshed on September 28 and carried item-level decisions only through September 27, and the PMA decision feed's newest entry is dated September 25. That leaves the October 1 valve resting on the agency's own announcement, and leaves no official item-level device record yet for September 28 through October 2. 456
At a glance
| Action | FDA date | Indication or intended use | Sponsor or applicant | What sets its scale |
|---|---|---|---|---|
| Emcitate (tiratricol) | Sep. 28 | Peripheral thyrotoxicosis in adults and children with MCT8 deficiency | Egetis Therapeutics (Nasdaq Stockholm: EGTX) | First approved treatment for the disease; about 1 in 70,000 males; brings a priority review voucher 78 |
| Camzyos (mavacamten) | Sep. 30 | Symptomatic obstructive hypertrophic cardiomyopathy in adults and in pediatric patients weighing at least 30 kg | Bristol Myers Squibb (NYSE: BMY) | First pediatric oHCM therapy; Valsalva LVOT gradient fell 48 mmHg more than placebo 29 |
| Autus Size-Adjustable Valve | Oct. 1 | Pulmonary valve replacement in children with congenital pulmonary valve disease | Edwards Lifesciences (NYSE: EW) | First valve that can be widened after surgery; first approved polymeric-leaflet valve 310 |
| Degevma (denosumab-adet) | Sep. 25, late-posted | Skeletal-related events and other indications carried by its reference product | Teva Pharmaceuticals | A denosumab biosimilar the September 19–25 issue could not see 4 |
Novel drug approval
Emcitate is the first treatment approved for MCT8 deficiency
FDA approved Emcitate (tiratricol) on September 28 for peripheral thyrotoxicosis in adults and pediatric patients with MCT8 deficiency, and the agency listed it that day as the year's forty-fifth novel drug. Emcitate is a thyroid hormone receptor agonist taken once daily as a liquid suspension, by mouth or through a feeding tube, and it is the first therapy FDA has approved for the disorder. 17
MCT8 deficiency comes from mutations in the SLC16A2 gene, which break the transporter that carries thyroid hormone into the brain. The brain is left short of hormone while the rest of the body is flooded with the active form, triiodothyronine, so patients carry both a severe neurodevelopmental impairment and a chronic hypermetabolic state that strains the heart, muscle, liver and kidneys. Many cannot walk or sit independently, and speech is absent or severely limited. Egetis puts median life expectancy at about 35 years. 811
Tiratricol sidesteps the broken transporter by entering cells on its own, which is what lets it pull down the excess circulating hormone. FDA based the approval on an international randomized, placebo-controlled trial plus a longer open-label study; across both, patients on Emcitate had lower thyroid hormone levels and improvements in cardiovascular and metabolic measures affected by those levels, including systolic blood pressure and heart rate. Diarrhea, vomiting, rash and excessive sweating were the most common side effects. The label carries a boxed warning that Emcitate is not for the treatment of obesity or weight loss, contraindicates it in primary hyperthyroidism, and warns that tiratricol cross-reacts with immunoassays for T3 and can make those results unreliable. Patients on another thyroid medication should not take both. 78
FDA granted orphan drug, rare pediatric disease, fast track, breakthrough therapy and priority review designations. 7
Commercial read
This is a very small market with a very large price-per-patient problem, and the approval's near-term value to Egetis sits as much in a voucher as in the drug. Epidemiological estimates put MCT8 deficiency at roughly 1 in 70,000 male births, or 5,000 to 10,000 boys and men across the United States and Europe combined. Egetis expects Emcitate to be commercially available in the United States eight to ten weeks after approval, through a patient support program it calls Egetis RareLink and a distribution partnership with PANTHERx Rare. 811
Egetis has not published a U.S. price. The closest reference is the German list price of about €10,000 a month for a 60-tablet supply before discounts — roughly €120,000 a year — and the drug made SEK 17 million (about $1.7 million) in the second quarter after a German launch in May. The approval also earned Egetis a rare pediatric disease priority review voucher, which the company says it expects to explore monetizing in the fourth quarter; pharmaphorum puts a sale value of $100 million to $200 million. 811
The market read it that way. Egetis shares rose 13.2% to SEK 8.42 on September 29, a new 52-week high, and Pareto kept a Sell rating while noting it had put an 80% probability on approval, which suggests the move tracked the removed regulatory risk and the voucher more than surprise about the decision itself. The competitive question is narrow: PriZm Therapeutics is developing SRW101, a thyroid hormone analogue designed to cross the blood-brain barrier, which would go after the central nervous system consequences that Emcitate does not address. Egetis is also developing Emcitate for resistance to thyroid hormone beta. 1112
Label expansion
Camzyos becomes the first drug approved for pediatric obstructive HCM
FDA approved Camzyos (mavacamten) on September 30 for adults and pediatric patients weighing at least 30 kg — about 66 pounds — with symptomatic obstructive hypertrophic cardiomyopathy, widening an indication the drug has held in adults since 2022. The submission is efficacy supplement SUPPL-13 to NDA 214998. 24
The supporting trial, SCOUT-HCM, randomized 44 patients aged 12 to under 18 with symptomatic oHCM and left ventricular outflow tract obstruction to mavacamten or placebo for 28 weeks, with dosing escalatable from 1 mg to 15 mg. The primary endpoint was the change in the Valsalva LVOT gradient, the pressure the heart generates when a patient bears down. The least-squares mean fell 48.5 mmHg on mavacamten and 0.5 mmHg on placebo, a difference of 48 mmHg (95% CI −67.7 to −28.3, p<.001). Serious adverse events were balanced at two patients per arm, and no patient's ejection fraction dropped below 50%. 9
Camzyos keeps its boxed warning for heart failure and stays available only through a restricted distribution program, the Camzyos Risk Evaluation and Mitigation Strategy. Dizziness and fainting occurred in more than 5% of patients and more often than on placebo. FDA granted orphan drug designation for this indication. 2
Commercial read
Pediatric oHCM is a small population, so the financial case is less about new volume than about label breadth and the precedent the trial sets. Bristol Myers Squibb says mavacamten has been prescribed to more than 25,000 patients in the United States since 2022, and the new label gives it the broadest indication of any approved cardiac myosin inhibitor. The clinical significance is what Joseph Rossano of Children's Hospital of Philadelphia, lead author on SCOUT-HCM, calls a proof of concept: a disease-specific therapy aimed at the underlying pathophysiology has now been developed and approved for children with a cardiovascular disorder, a route that pediatric cardiology has largely been without. 9
Other approvals and label actions
FDA's report also carries one other original application inside the window and a set of labeling updates. Ever Valinject received approval on October 1 for an original application covering a new formulation of trabectedin, the injectable chemotherapy, classified by FDA as a new formulation or new manufacturer. Genentech took two labeling supplements for Rozlytrek (entrectinib) the same day. 4
Among the week's biosimilar supplements, Celltrion's Avtozma (tocilizumab-anoh) took supplements on both of its BLAs, Celltrion's Eydenzelt (aflibercept-boav) took a labeling supplement, Teva's Ponlimsi (denosumab-adet) took one, and Amgen's Riabni (rituximab-arrx) took a bioequivalence supplement. 4
Other captured Drugs@FDA actions
The tables below carry the rest of the week's drug record, with the values as the agency's report lists them. 4
Original applications
| Application | Product | Dosage form | Applicant | Date | Category |
|---|---|---|---|---|---|
| NDA 220963 | Emcitate (tiratricol) | Tablet for oral suspension | Rare Thyroid Therapeutics International AB | Sep. 28 | New molecular entity |
| NDA 219617 | Trabectedin | Injectable | Ever Valinject | Oct. 1 | New formulation or manufacturer |
| ANDA 216254 | Dexmedetomidine hydrochloride | Injectable | Nephron | Sep. 28 | Generic |
| ANDA 212778 | Prednisolone acetate | Ophthalmic suspension | Upsher Smith Labs | Sep. 28 | Generic |
| ANDA 218361 | Mupirocin calcium | Topical cream | Zydus Lifesciences | Sep. 29 | Generic |
| ANDA 211414 | Dapagliflozin | — | Bionpharma | Sep. 29 | Generic, tentative |
| ANDA 220181 | Levocarnitine | Injectable | Gland | Sep. 30 | Generic |
| ANDA 220722 | Loperamide hydrochloride | Capsule | Taro | Sep. 30 | Generic |
| ANDA 219175 | Phytonadione | Injectable | Lupin | Sep. 30 | Generic |
Supplements
The remaining rows are labeling and manufacturing updates rather than new uses. They include efficacy-supplement activity already described above, labeling supplements for Zepzelca (Jazz), Xenical (Cheplapharm), Matulane (Leadiant Biosci), Pifeltro and Delstrigo (Merck), phentermine-class and stimulant generics from Teva, Granules, Accord, Sun Pharm and Amneal, and a long run of B. Braun's dextrose and sodium chloride presentations. 4
Late-posted records from the September 19–25 window
Three drug records dated September 25 reached the agency's report only after the last issue had gone out, and they belong on the record with their own dates. Teva's Degevma (denosumab-adet), an original BLA numbered 761481, was approved on September 25 as a denosumab product. Adventapharma's Zueino (diclofenac sodium) injection, NDA 218379, was approved the same day. Lupin's apixaban oral suspension, NDA 220446, received tentative approval on September 25. 4
Medical devices
A valve that grows with a child
FDA approved the Autus Size-Adjustable Valve on October 1 through the premarket approval pathway, for pediatric patients with congenital pulmonary valve disease who need a pulmonary valve replacement. Edwards Lifesciences holds the approval; the device was developed by Autus Valve Technologies. 310
The valve addresses a timing trap that has defined pediatric pulmonary valve replacement. Children with pulmonary valve stenosis or atresia often end up with significant regurgitation after earlier repair, which over time weakens or enlarges the right ventricle and creates the need for a replacement valve. Operating too early means the child outgrows the valve and needs more open-heart surgery; waiting risks harm to the heart. The Autus valve is implanted as small as about 13 millimeters across, the right size for a toddler or preschool child, and can later be widened with a balloon catheter to as much as 22 millimeters, comparable to an adult pulmonary valve. FDA says the Autus valve is also the first approved valve whose leaflets — the flaps that open and close to control blood flow — are made of a polymer rather than tissue taken from animals, which tends to harden faster in children. 3
The pivotal study enrolled 62 pediatric patients at 12 United States sites. At six months, the first 60 patients showed acceptable blood flow through the right side of the heart with no more than mild regurgitation, all procedures were successful, and there were no deaths, no blood clots requiring treatment and no strokes. Three patients had a fracture in the valve frame and two had reduced movement of a leaflet, without symptoms. Valve performance began to deteriorate in two patients who started to outgrow the valve; both were expanded successfully through a catheter rather than surgery, which FDA describes as limited experience. Patients will be followed for ten years. The approval was the first pediatric one to come out of FDA's Total Product Life Cycle Advisory Program, and the device previously held Breakthrough Device designation. 313
Commercial read
The addressable population is defined by a narrow anatomy, not by a large disease category. FDA cites CDC figures putting pulmonary valve atresia and stenosis together at around 4,200 babies a year in the United States, against about one in 100 births for congenital heart defects overall. Edwards slots the device into a congenital portfolio that already includes the SAPIEN 3 valve, the Alterra Adaptive Prestent and pediatric atrioseptostomy catheters, and the company's pitch to physicians is fewer returns to the operating room rather than a survival claim. The open questions are durability beyond the one-year data and whether hospitals and payers treat a first-in-class pediatric valve the way they treat established devices. 310
The captured device ledger
The 510(k) file refreshed on September 28 carries two item-level decisions dated inside this window, both on September 27: K252139, Rivarp Medical's Kentrospine Viktr-MIS pedicle screw system, and K260945, the Ultima system from Fit-Pro KFT, trading as E-Fit. September 26 has none. 51415
The same refresh closed a gap the September 19–25 issue had flagged. That issue's file stopped at September 19; the new one adds 72 clearances dated September 21 through 25, among them Siemens Healthineers' syngo.CT Brain Quantification (K260055), Tempus AI's ECG-MR (K254297), Align Technology's Invisalign System (K262196), Philips Ultrasound's EPIQ Inspira series (K262548) and Samsung Medison's EVO X ultrasound family (K263182). 5
The PMA feed now runs through September 25, and everything it carries in the September 21–25 stretch is a supplement rather than an original approval. 6
Clearances reported this week, on earlier FDA dates
Several companies announced clearances between September 28 and October 1 whose FDA decision dates, where the captured file shows them, fall earlier than the announcement — the pattern this channel has now recorded for several weeks.
| Device | Applicant | Announced | FDA date in the captured file |
|---|---|---|---|
| iFuse Granite Onyx System, K262067 | SI-BONE (Nasdaq: SIBN) | Sep. 28 | Sep. 23 1617 |
| CLEAR Aspiration System, K253404 | Insera Therapeutics | Sep. 28 | not yet in the file 18 |
| VEnice Vein Closure Device, K260677 | VCD Medical | Sep. 29 | Sep. 17 1920 |
| NanoACTIV surface on the Riva posterior fixation system, K262193 | Xenix Medical | Sep. 30 | Sep. 14 2122 |
| SpaceOAR Flex System, K262151 | Boston Scientific | Sep. 28 | Sep. 11 2324 |
| FreeClimb 46 catheter with Tenzing 4 | Route 92 Medical | Sep. 28 | not yet in the file 25 |
Recent 510(k) records carry the applicant and decision date without a statement of intended use, so the cleared scope of a fresh clearance rests on the applicant's own description.
What matters next
- Emcitate's U.S. price and payer path. Egetis launches within eight to ten weeks and has not published a U.S. price; watch whether payers accept a per-patient cost in the range the German list price implies for a population of a few thousand patients, and whether the company sells the priority review voucher before year-end.
- Camzyos in adolescents. The label now reaches patients at 30 kg and up; uptake will show whether the REMS and the heart-failure boxed warning limit use in a pediatric population with few alternatives.
- Autus durability. The one-year data are encouraging and the cohort is 62 patients; the ten-year follow-up and the first real-world expansions are what will tell whether the growth claim holds.
- The device ledger for September 28 – October 2. The next issue should capture the 510(k) decisions dated September 28 or later and any original PMA decision after September 25, once the monthly file and the feed are refreshed.
References
- 1
- 2
- 3
- 4Recent New and Generic Drug Approvals
accessdata.fda.gov
- 5Downloadable 510(k) Files
fda.gov
- 6FDA PMA Decisions Feed
accessdata.fda.gov
- 7
- 8Egetis Therapeutics Announces U.S. FDA Approval of EMCITATE
view.news.eu.nasdaq.com
- 9
- 10
- 11FDA clears first drug for MCT8 deficiency
pharmaphorum.com
- 12Why is Egetis Therapeutics stock surging today?
investing.com
- 13FDA greenlights Edwards' expandable pulmonary valve for paediatric patients
medicaldevice-network.com
- 14FDA 510(k) record K252139
accessdata.fda.gov
- 15FDA 510(k) record K260945
accessdata.fda.gov
- 16SI-BONE Receives FDA 510(k) Clearance for iFuse Granite Onyx
globenewswire.com
- 17FDA 510(k) record K262067
accessdata.fda.gov
- 18
- 19Vein Closure Device Receives FDA Clearance
dicardiology.com
- 20FDA 510(k) record K260677
accessdata.fda.gov
- 21FDA Clears NanoACTIV for Xenix Medical's Riva System
orthoworld.com
- 22FDA 510(k) record K262193
accessdata.fda.gov
- 23
- 24FDA 510(k) record K262151
accessdata.fda.gov
- 25
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