CAMZYOS
Pronunciation: mav-a-KAM-ten


What it isCardiac myosin inhibitor reducing actin-myosin cross-bridging; obstructive hypertrophic cardiomyopathy in adults and children ≥30 kg.
Why this oneThe first oral therapy to target HCM hypercontractility directly, it can defer septal reduction.
What limits itSystolic dysfunction requires echocardiographic monitoring under a REMS. CYP2C19 variation changes exposure severalfold.
Legacy pregnancy categoryNot assigned — PLLR-era drug
Defining liabilityGenerally tolerated at therapeutic doses; clinically important risks are agent-specific and increase with interactions or organ impairment.
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| FDA dosing | Population | Start | Target | Max |
|---|---|---|---|---|
| Symptomatic obstructive HCM | Adult | 5 mg QD | individualized 2.5–15 mg QD | 15 mg QD |
| Obstructive HCM + moderate CYP2C19 or strong CYP3A4 inhibitor | Adult | 2.5 mg QD (one dose below recommended start) | individualized | 15 mg QD |
| Symptomatic obstructive HCM | Pediatric ≥45 kg | 5 mg QD | individualized 1–15 mg QD | 15 mg QD |
| Symptomatic obstructive HCM | Pediatric 30 kg to <45 kg | 2.5 mg QD | individualized 1–15 mg QD | 15 mg QD |
| Obstructive HCM + moderate CYP2C19 or strong CYP3A4 inhibitor | Pediatric ≥30 kg | one dose below weight-band starting dose | individualized | 15 mg QD |
Titration Assess LVEF and Valsalva LVOT gradient before each up-titration (weeks to steady state); when on an interacting inhibitor, do not up-titrate until 12 weeks after inhibitor initiation.
Renal No clinically significant PK difference in mild–moderate impairment (eGFR 30 to 89); severe (eGFR 15 to 30) and kidney failure unknown
Hepatic No adjustment for mild–moderate (Child-Pugh A/B) with the standard titration/monitoring plan; severe (Child-Pugh C) unknown.
Available only through the CAMZYOS REMS Program.
Available strengths
Pregnancy May cause fetal harm (animal data); confirm absence of pregnancy before starting and use contraception during and 4 months after last dose.
Lactation No data on presence in milk or effects on infant/production; weigh benefits.
| Strength | #/mo | generic $/mo | CostPlus | |
|---|---|---|---|---|
| 2.5 mg | cap | 30 qd | not offered | |
| 5 mg | cap | 30 qd | not offered | |
| 10 mg | cap | 30 qd | not offered | |
| 15 mg | cap | 30 qd | not offered |
Generic entry2034 est.
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