New Pill When the Heart Muscle Is Too Thick

For people living with obstructive hypertrophic cardiomyopathy, the practical question is not “is there a myosin inhibitor?” Camzyos (mavacamten) already exists in several markets. It is “can my cardiologist prescribe this one, and who pays?”

Aficamten (Myqorzo) is a once-daily cardiac myosin inhibitor. The FDA labelled it on 19 December 2025 for adults with symptomatic obstructive HCM, to improve functional capacity and symptoms. The European Commission authorised it on 12 February 2026. China’s NMPA authorised a local product (阿夫凯泰片, brand 星舒平) in December 2025. The UK MHRA authorised it on 29 July 2026, and NICE published TA1181 the next day — England’s NHS has a 30-day funding clock from that publication, not the usual 90.

Health Canada accepted a new-drug submission in December 2025. As of the SUR list dated 31 July 2026, it is still under review. That is not a no.

Non-obstructive HCM is not on any of those labels. ACACIA-HCM reported positive topline results; that is a later filing, not today’s prescription.

Where this is taking place

SEQUOIA-HCM (NCT05186818) is completed. Phase 3, 24 weeks, echo-titrated 5–20 mg once daily vs placebo. 282 adults randomized (142 aficamten, 140 placebo). Countries on the FDA Drug Trials Snapshot: China, Czechia, Germany, Denmark, Spain, France, the United Kingdom, Hungary, Israel, Italy, the Netherlands, Poland, Portugal, the United States. Canada is not on that list. Site-count sources disagree (82 / 101 / 114); the country list is what a reader can act on.

ACACIA-HCM (NCT06081894) — symptomatic non-obstructive HCM — is ACTIVE_NOT_RECRUITING. Not labelled. Collaborators on CT.gov: Sanofi, Bayer. Do not write it as an enrolment path.

FOREST-HCM (NCT04848506) is the open-label extension, ENROLLING_BY_INVITATION. CEDAR-HCM (NCT06412666) is paediatric oHCM, ACTIVE_NOT_RECRUITING for 12–<18; 6–<12 not yet recruiting. MAPLE-HCM (NCT05767346) vs metoprolol is COMPLETED; an FDA sNDA has a PDUFA date of 14 Nov 2026 — not yet approved.

Commercial supply: US (REMS), EU (centralised MA), UK (MHRA + NICE), China (Sanofi Greater China). Canada: no marketed product.

Approval matrix (obstructive HCM in adults)

Do not collapse NYHA on the US line with the EU/UK/China II–III wording. Use the label of the country that will prescribe. Do not write non-obstructive HCM as labelled.

Access by country

• United States: Cardiologist experienced in HCM. FDA-labelled 19 Dec 2025. Three numbers, do not mix them:
• MYQORZO REMS (prescriber, patient, and pharmacy enroll because of heart-failure / systolic-dysfunction risk): myqorzorems.com and 1-844-285-7367.
• MYQORZO & You navigator (savings and support, not REMS): 833-697-6796 (833-MYQORZO), myqorzo.com/savings-and-support/myqorzo-and-you/. No copay dollar amount locked from that page.
• Adverse events / pregnancy exposure: 1-833-633-2986 (US PI). Echo titration 5–20 mg. Prior authorization still applies. List price not on DailyMed.
• United Kingdom (England): MHRA authorised 29 Jul 2026. NICE TA1181: option for adults with NYHA 2–3, as add-on to individually optimised beta-blocker / non-dihydropyridine calcium-channel blocker / disopyramide, or alone if those are contraindicated, and if the company commercial arrangement is in place. Use the least expensive of aficamten and mavacamten. NHS England must fund within 30 days of 30 Jul 2026. That is a funding clock, not a proven first-dispense date. Ask the HCM clinic; do not DIY-import.
• Wales: NICE TA usually funded within 60 days of first publication of final draft guidance. Ask NHS Wales / the HCM clinic.
• Scotland: SMC not found. Do not assume NICE = Scotland.
• European Union: Centralised MA 12 Feb 2026. Prescription; start/supervise by a doctor experienced in cardiomyopathy. Educational pack and patient card (heart failure + embryo-fetal risk). National reimbursement unknown here unless a local HTA is cited. Travelling from Canada for an EU script is a medical/legal plan with both specialists, not a DIY import.
• China: In-country commercial: 星舒平 (阿夫凯泰片), Sanofi Greater China. NMPA-authorised for NYHA II–III obstructive HCM in adults. NRDL not found on an official NHSA source — do not write it as insured. This is a China in-country path, not a Canadian travel path.
• Canada (zoom, not the Access Level): No NOC. SUR under review (accepted Dec 2025; list current 31 Jul 2026). That is not a no. Specialist can ask Health Canada SAP for a non-marketed drug when conventional therapy has failed, is unsuitable, or is unavailable. Manufacturer must agree to supply. SAP unknown — not available and not refused. SEQUOIA had no Canadian sites on the FDA country list. This zoom is not a Compassionate Access tag on the piece.
• Australia / Japan / Switzerland: Authorisation not found. Specialist + local special-access / trial if those programs apply. Do not invent a compassionate-use ID.

If your regulator has not authorised it: do not import on your own. Ask about travelling to a labelled market (US REMS, UK NHS, EU, China) only as a plan with a local specialist.

What the pivotal trial showed

SEQUOIA-HCM randomized 282 adults with NYHA II–III obstructive HCM (screening LVEF ≥60%; resting LVOT-G ≥30 mmHg and post-Valsalva ≥50). Mean age 59.1; 59.2% men; mean resting gradient 55.1 mmHg; mean LVEF 74.8%.

At week 24, DailyMed/USPI Table 4 least-squares mean peak VO2 change was 1.7 vs 0.0 mL/kg/min (difference 1.7; 95% CI 1.0–2.4). That is the FDA number. NEJM reported 1.8 vs 0.0 (same difference 1.7; P<0.001). All 10 hierarchical secondary endpoints improved. On the FDA label: NYHA class improved ≥1 in 58.5% vs 24.3%; KCCQ-CSS difference 7.3 points; Valsalva gradient −48.8 mmHg vs placebo; Valsalva <30 mmHg in 49.3% vs 3.6%.

Hypertension was the only adverse event >5% and more common on drug (8% vs 2%). LVEF fell below 50% in 4% vs 1%; those drops reversed and were not tied to clinical heart failure in SEQUOIA. Mean LVEF change −7% vs −2%. That is why the US label carries a boxed warning and a REMS, and why the EU pack includes a patient card.

This is not a cure. It is a titrated myosin inhibitor that, in one 24-week trial, moved exercise capacity, symptoms, and the obstruction gradient.

How it is taken (labelled)

Start 5 mg once daily (US and EU). Swallow whole. Increase by 5 mg every 2–8 weeks to a maintenance dose or a maximum of 20 mg, using echo (LVEF and Valsalva LVOT-G). Do not start or up-titrate if LVEF is <55%. Interrupt if LVEF <40% or heart-failure symptoms appear.

The EU SmPC lets clinicians consider a 10 mg start if LVOT-G is ≥100 mmHg. The US PI does not. Use the label of the country that will prescribe.

US contraindication: rifampin only. EU also contraindicates adagrasib, fluconazole more than a single dose, rifampicin, and St John’s wort. Main enzyme: CYP2C9.

Pregnancy is not a contraindication on either label. There are no human data. Effective contraception is required on the EU SmPC. US: report pregnancy to 1-833-633-2986 (AE / pregnancy line, not the MYQORZO & You navigator and not REMS) if exposed on drug or within 3 weeks of the last dose. Pediatrics: not established.

Research team (Who)

Names below are as they appear on primary sources.

• Martin S. Maron, MD — SEQUOIA-HCM principal investigator; first author, NEJM. Hypertrophic Cardiomyopathy Center, Lahey Hospital and Medical Center (Burlington, MA).
• Cytokinetics, Incorporated — sponsor (SEQUOIA, MAPLE, ACACIA, FOREST, CEDAR). US/EU/UK MAH family: Cytokinetics / Cytokinetics (Ireland) Limited.
• Corxel Pharmaceuticals — collaborator on SEQUOIA CT.gov; prior Greater China rights holder.
• Sanofi — Greater China commercial rights (星舒平). Collaborator on ACACIA CT.gov.
• Bayer — collaborator on ACACIA CT.gov.

No Canadian PI is named because SEQUOIA’s FDA country list does not include Canada.

How to talk to a doctor

Bring the NCT and the regulator that applies:

1. “I have symptomatic obstructive HCM (NYHA II–III). Is aficamten labelled where we are, or is mavacamten the funded option?”
2. If US: “Are you REMS-certified for Myqorzo?”
3. If England/Wales: “Does TA1181 apply to me — add-on to optimised standard care, or monotherapy because those drugs are contraindicated?”
4. If Canada: “The NDS is under review as of 31 Jul 2026. Is SAP appropriate, or do we wait for the NOC?”
5. If non-obstructive: “ACACIA is not a labelled indication. Do not write me aficamten off-label from a topline press release.”

Canada zoom: under review / SAP unknown

Health Canada is under review. SAP for a non-marketed drug is unknown — not a listing, not a refusal. That zoom is not the Access Level. US: labelled + REMS, so expanded access is the wrong door for obstructive HCM in adults. UK/EU/China: labelled commercial paths.

Bottom line

Aficamten is labelled for symptomatic obstructive HCM in adults in the United States, the European Union, the United Kingdom, and China. England’s NHS has a 30-day NICE clock from 30 July 2026. Health Canada is under review, not refused. Non-obstructive disease is not labelled. Authorized is not funded, except where NICE has already said it must be. Canada is a zoom.

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Primary sources

1. FDA news, 19 Dec 2025 — https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-improve-functional-capacity-and-symptoms-adults-rare-inherited-heart-condition
2. FDA Novel Drug Approvals 2025 (NDA 219083) — https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025
3. US PI / DailyMed — https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=fd778507-1274-4d1a-a659-5431d55c543a
4. FDA label PDF — https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/219083s000lbl.pdf
5. FDA Drug Trials Snapshot — https://www.fda.gov/drugs/drug-trials-snapshots/drug-trials-snapshots-myqorzo
6. MYQORZO REMS — https://www.myqorzorems.com/ (REMS phone 1-844-285-7367) 6a. MYQORZO & You navigator — https://www.myqorzo.com/savings-and-support/myqorzo-and-you/ (833-697-6796 / 833-MYQORZO). No copay dollars locked from this page. 6b. US PI AE / pregnancy — 1-833-633-2986
7. EMA EPAR Myqorzo — https://www.ema.europa.eu/en/medicines/human/EPAR/myqorzo
8. EMA SmPC — https://www.ema.europa.eu/en/documents/product-information/myqorzo-epar-product-information_en.pdf
9. GOV.UK MHRA/NICE news, 30 Jul 2026 (MA 29 Jul 2026) — https://www.gov.uk/government/news/thousands-of-patients-to-benefit-from-new-daily-pill-for-serious-heart-condition-following-co-ordinated-mhra-and-nice-decisions
10. NICE TA1181 — https://www.nice.org.uk/guidance/ta1181
11. NICE TA1181 recommendations — https://www.nice.org.uk/guidance/ta1181/chapter/1-Recommendations
12. NICE TA1181 implementation (30-day England / 60-day Wales) — https://www.nice.org.uk/guidance/ta1181/chapter/3-Implementation
13. Health Canada SUR (current as of 2026-07-31) — https://www.canada.ca/en/health-canada/services/drug-health-product-review-approval/submissions-under-review/new-drug-submissions-under-review.html
14. Health Canada SAP — https://www.canada.ca/en/health-canada/services/drugs-health-products/special-access/drugs.html
15. SEQUOIA-HCM CT.gov NCT05186818 — https://clinicaltrials.gov/study/NCT05186818
16. Maron MS et al. NEJM 2024;390:1849–1861. DOI 10.1056/NEJMoa2401424 — https://www.nejm.org/doi/full/10.1056/NEJMoa2401424
17. NMPA-affiliated CPI reprint, 23 Dec 2025 — https://www.cpi.ac.cn/zx/xyzl/202512/t20251223_429060.html
18. Beijing News reprint of NMPA text — https://www.bjnews.com.cn/detail/1766388465129356.html
19. ACACIA-HCM NCT06081894 (ACTIVE_NOT_RECRUITING; not labelled) — https://clinicaltrials.gov/study/NCT06081894
20. FOREST-HCM NCT04848506 — https://clinicaltrials.gov/study/NCT04848506
21. CEDAR-HCM NCT06412666 — https://clinicaltrials.gov/study/NCT06412666
22. MAPLE-HCM NCT05767346 — https://clinicaltrials.gov/study/NCT05767346