Heart Pill for Obstructive HCM from About 66 Pounds

Camzyos is an oral cardiac myosin inhibitor (mavacamten). The United States prescribing information revised 9/2026 expanded the indication to adults and pediatric patients weighing 30 kg or more with symptomatic obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms. FDA public materials describe this as the first approved drug to improve functional capacity and symptoms in children with this rare inherited heart condition. It is a once-daily capsule available only through the restricted CAMZYOS REMS program because of the risk of heart failure from reduced pumping strength. It is not approved under 30 kg, not a cure, and not interchangeable with other myosin inhibitors by brand name. Marketed by Bristol-Myers Squibb Company.

Distinct path — not this use (fact-only): Aficamten (Myqorzo) is a different cardiac myosin inhibitor covered separately on Medcelerator. It is not Camzyos. Non-obstructive HCM, phenocopy diseases that thicken the heart for other reasons (for example Fabry disease, amyloidosis, or Noonan-related hypertrophy in the SCOUT exclusions), and septal reduction surgery pathways are not this labelled population.

Where this is taking place

Commercial door today: United States. USPI Recent Major Changes list Indications and Usage and Dosage and Administration updates in 9/2026. Bristol Myers Squibb announced the pediatric expansion on 30 September 2026. An FDA drugs news page describes approval for children weighing at least 30 kg (~66 lb) with symptomatic obstructive HCM.

Who gives it: A cardiology or pediatric cardiology clinician certified in the CAMZYOS REMS Program, with echocardiography available before and during treatment. Patients must enroll in REMS and use a certified pharmacy (not a routine local retail fill). REMS information on the USPI: www.CAMZYOSREMS.com or 1-833-628-7367. Suspected side effects: Bristol-Myers Squibb 1-800-721-5072 or FDA MedWatch 1-800-FDA-1088.

Trial that supported the pediatric expansion: SCOUT-HCM — NCT06253221. Phase 3, randomised, double-blind, placebo-controlled. Sponsor: Bristol-Myers Squibb. Status checked 1 October 2026: active, not recruiting (primary completion 25 November 2025). Forty-four adolescents aged 12 to <18 years were randomised. Trial sites included the United States and several other countries; a completed trial site is not a paediatric marketing authorisation there.

Outside the United States: Health Canada, EMA/EC, MHRA, TGA, PMDA, and Swissmedic authorisation of Camzyos for pediatric obstructive HCM is not confirmed on sources used here. Company materials say pediatric data are being discussed with other regulators. Adult Camzyos authorisation elsewhere is a different question — do not assume the pediatric US wording applies. Do not import on your own.

Approval matrix

Access by country

• United States: Ask whether a REMS-certified HCM cardiology clinic will evaluate symptomatic obstructive HCM, confirm weight ≥30 kg, confirm LVEF is high enough to start, enroll the patient in REMS, and arrange certified pharmacy dispensing with scheduled echoes. No list price or copay dollars in this article.
• Canada / EU / UK / other countries: Pediatric authorisation not confirmed. Do not import on your own.
• Adult Camzyos use outside the US, if authorised locally, does not automatically include this pediatric population.

Who is eligible

United States (prescribing information revised 9/2026):

• Indication: Treatment of adults and pediatric patients weighing 30 kg or more with symptomatic obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms.
• Pediatrics: Safety and effectiveness established in patients weighing ≥30 kg, supported by adult trials plus SCOUT-HCM in ages 12 to <18. Not established under 30 kg.
• Do not start or up-titrate if LVEF is <55%. Interrupt if LVEF is <50% or if heart-failure symptoms / worsening status appear (see dosing algorithms on the USPI).
• Contraindications: Strong CYP2C19 inhibitors; moderate to strong CYP2C19 inducers or moderate to strong CYP3A4 inducers.
• REMS: Prescriber certification, patient enrollment, certified pharmacy only.
• Pregnancy: May cause fetal harm. Confirm absence of pregnancy before start. Effective contraception during treatment and for 4 months after the last dose. Some combined hormonal contraceptives need a backup method — see USPI.
• SCOUT population notes (label): Symptomatic NYHA II–III obstructive HCM; LVEF ≥60% and LVOT peak gradient ≥50 mmHg at rest or with provocation in the trial; background HCM medicines allowed. Phenocopy / multi-system malformation syndromes causing hypertrophy were excluded.

Not this indication, on the sources used here:

• Weight under 30 kg.
• Non-obstructive HCM as the labelled claim.
• Starting when LVEF is already under 55%.
• Filling outside REMS.

What the pivotal study showed (SCOUT-HCM / NCT06253221)

Use USPI section 14 (Pediatric Patients — SCOUT-HCM) for a prescription conversation. Adult EXPLORER-HCM and VALOR-HCM remain on the same label for adult evidence.

Design: 44 pediatric patients (23 Camzyos, 21 placebo), ages 12 to <18, weight ≥35 kg in the trial, symptomatic obstructive HCM, randomised 1:1 for 28 weeks. Starting dose 2.5 mg if weight 35 to <45 kg, or 5 mg if ≥45 kg, then titrated using echo (allowable doses 1–15 mg). Mean age 15 years; 84% NYHA II; 84% on a beta blocker at baseline.

Primary endpoint: Change from baseline in Valsalva LVOT gradient at Week 28.

Result (USPI): Mean (SD) change −49.4 (42.6) mmHg with Camzyos versus −1.8 (37.8) mmHg with placebo. Least-squares mean difference −48.0 mmHg (95% CI −67.7 to −28.3); p < 0.0001.

The label states Camzyos reduced resting and Valsalva LVOT gradients in these pediatric patients and, based on adult functional results plus these gradient and pharmacology data, is expected to improve functional capacity and symptoms in pediatric patients ≥30 kg.

Pediatric safety (USPI §6.1): No new adverse reactions compared with adult studies in the SCOUT safety narrative. Adult trials most often reported dizziness and syncope more than placebo.

How it is taken

The prescribing information (§2.1–2.2), not a press release, sets dose and monitoring.

Safety (what the label puts first)

Boxed warning — risk of heart failure:

• Camzyos reduces LVEF and can cause heart failure from systolic dysfunction.
• Echo LVEF assessments are required before and during use.
• Do not initiate if LVEF <55%. Interrupt if LVEF <50% or clinical status worsens.
• Certain CYP450 drug interactions raise heart-failure risk; some combinations are contraindicated.
• Available only through CAMZYOS REMS.

Other USPI warnings: drug interactions leading to heart failure or loss of effect (including some OTC acid reducers); embryo-fetal toxicity.

Common reactions (>5% and more than placebo in adults): dizziness (27% vs 18%), syncope (6% vs 2%).

How to talk to a doctor

Bring: weight in kg, symptom history (shortness of breath, chest pain, syncope), current HCM medicines (beta blockers, calcium-channel blockers, disopyramide), echo reports (LVEF and LVOT gradients), infection or arrhythmia history, pregnancy plans, and a full medicine list including OTC omeprazole/esomeprazole/cimetidine.

Ask: Are you REMS-certified? Do we meet the ≥30 kg symptomatic obstructive HCM population? What echo schedule will we follow? Which drug interactions would stop or cut the dose? How does this compare with aficamten or with septal reduction options if those are on the table?

Research and regulatory team (from sources only — no invented contacts)

• Sponsor / marketer: Bristol-Myers Squibb Company (USPI; ClinicalTrials.gov NCT06253221).
• SCOUT-HCM publication (reference on CT.gov): Rossano et al., N Engl J Med 2026 (PMID 41910394 on the trial record) — cite the USPI numbers for labelled claims.
• Named individual PI is not presented as a single contact on the ClinicalTrials.gov record used here (study director: Bristol-Myers Squibb). Do not invent a clinician phone.

Canada zoom: pediatric expansion not confirmed

Camzyos for pediatric obstructive HCM is not confirmed as a Health Canada authorisation on sources used here. US REMS enrollment is not a Canadian paediatric door. Do not import on your own.

EU zoom: pediatric authorisation not confirmed

Camzyos for this pediatric population is not confirmed as an EMA or European Commission authorisation. Company materials describe discussions with global authorities. SCOUT sites in Europe are not an EC decision. Do not import on your own.

Bottom line

Camzyos is a US-labelled once-daily oral cardiac myosin inhibitor for symptomatic obstructive HCM in people who weigh at least 30 kg, including eligible adolescents, with the same REMS, echo, and interaction rules that already bind adult use. SCOUT-HCM showed a large drop in Valsalva LVOT gradient at week 28 versus placebo. The near-term path is a REMS-certified cardiology clinic in the United States — not a local pharmacy shortcut, not a sub-30 kg approval, and not an assumption that another country has authorised the pediatric wording.

Primary sources

• Camzyos (mavacamten) US Prescribing Information, BMS PDF, Revised 9/2026
• FDA news: first drug to improve functional capacity and symptoms in children with rare inherited heart condition
• ClinicalTrials.gov NCT06253221 (SCOUT-HCM)
• Bristol Myers Squibb company announcement, 30 Sep 2026 (company — not the regulator)