Aficamten Improves Exercise Capacity in Nonobstructive Hypertrophic Cardiomyopathy
Two studies tested aficamten, a drug that reduces cardiac myosin activity, in people with nonobstructive hypertrophic cardiomyopathy. In a 517-patient NEJM trial, those taking aficamten for 36 weeks showed greater gains in peak oxygen consumption and self-reported health than those on placebo. A companion Circulation study using heart ultrasound found less excessive contraction and better diastolic function with the treatment.
Aficamten targets cardiac myosin, the protein involved in heart muscle contraction. Nonobstructive hypertrophic cardiomyopathy is an inherited condition that thickens and stiffens the heart, causing breathlessness and limiting activity; it represents roughly 30% of hypertrophic cardiomyopathy cases. The NEJM trial enrolled 517 patients and compared aficamten with placebo for 36 weeks.
The Circulation analysis used ultrasound scans from the trial. It found reduced signs of overcontraction and better diastolic measures, including tissue Doppler relaxation readings, left-ventricular blood-volume changes, and lower cardiac stress markers. A reversible drop in one pumping measure was more common with aficamten. An extension study is tracking longer-term outcomes in both HCM forms.
Count? First para: Aficamten(1) targets2 cardiac3 myosin4, the5 protein6 involved7 in8 heart9 muscle10 contraction11. Nonobstructive12 hypertrophic13 cardiomyopathy14 is15 an16 inherited17 condition18 that19 thickens20 and21 stiffens22 the23 heart24, causing25 breathlessness26 and27 limiting28 activity29; it30 represents31 roughly32 30%33 of34 hypertrophic35 cardiomyopathy36 cases37. The38 NEJM39 trial40 enrolled41 51742 patients43 and44 compared45 aficamten46 with47 placebo48 for49 36 50 weeks51. Good 51. Second: The1 Circulation2 analysis3 used4 ultrasound5 scans6 from7 the8 trial9. It10 found11 reduced12 signs13 of14 overcontraction15 and16 better17 diastolic18 measures19, including20 tissue21 Doppl
If approved, aficamten could give people with nonobstructive hypertrophic cardiomyopathy a targeted option where none has existed, potentially improving exercise tolerance, symptoms, and daily functioning. Clinicians may gain another way to assess treatment response through exercise and ultrasound measures. Because a reversible decline in pumping ability appeared more often with the drug, patients and providers may need careful cardiac monitoring. Longer extension data could clarify whether benefits persist and how the therapy fits into care for this inherited condition. Count: If1 approved2, aficamten3 could4 give5 people6 with7 nonobstructive8 hypertrophic9 cardiomyopathy10 a11 targeted12 option13 where14 none15 has16 existed17, potentially18 improving19 exercise20 tolerance21, symptoms22, and23 daily24 functioning25. Clinicians26 may27 gain28 another29 way30 to31 assess32 treatment33 response34 through35 exercise36 and37 ultrasound38 measures39. Because40 a41 reversible42 decline43