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Mavacamten efficacy and oHCM diagnosis updates

Updated 3 times since CLSTR started tracking revisions of this situation.

What changed

2026-09-30 23:10 UTC → 2026-09-30 23:14 UTC · added removed

Bristol Myers Squibb presented long-term data from the Explorer-Lte study at the European Society of Cardiology Congress in Munich. The results indicate that mavacamten, a cardiac myosin inhibitor, maintains its benefits for up to five years in patients receiving continuous therapy for symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Medical specialists have noted that the drug appears to be an effective and safe treatment for this progressive heart muscle disease. The condition is characterized by thickening of the heart walls that can obstruct blood flow from the left ventricle. Experts have emphasized the necessity of symptom awareness and timely diagnosis to manage the disease effectively. In Italy, specialists are highlighting the critical need for early diagnosis, noting that the condition affects an estimated 11,000 people, though frequent misdiagnosis may mean the actual figure is higher. Because the disease has a strong genetic and familial component, experts emphasize that screening is vital. Italy currently utilizes a network of over 100 specialized centers to assist with the diagnosis and management of the disease. Lia Crotti, a specialist from the University of Milano-Bicocca and the Irccs Istituto Auxologico Italiano, noted the progressive nature of the muscle thickening involved in the condition. Expanding its clinical application, the U.S. Food and Drug Administration (FDA) has approved mavacamten (Camzyos) for adolescents aged 12 to younger than 18 years with symptomatic oHCM. This marks the first cardiac myosin inhibitor approved for this adolescent population. The approval follows results from the phase 3 SCOUT-HCM trial, which showed that mavacamten significantly reduced the Valsalva-provoked left ventricular outflow tract (LVOT) gradient compared to a placebo at 28 weeks. Furthering this expansion, the FDA has approved an expanded indication for mavacamten to treat symptomatic oHCM in pediatric patients, specifically covering adolescents aged 12 to 18 years and children weighing at least 30 kg.

Versions

  1. 2026-09-30 23:14 UTC Mavacamten efficacy and oHCM diagnosis updates
  2. 2026-09-30 23:10 UTC Mavacamten efficacy and oHCM diagnosis updates
  3. 2026-09-30 15:19 UTC Mavacamten efficacy and oHCM diagnosis updates
  4. 2026-09-30 14:04 UTC Mavacamten long-term efficacy for hypertrophic cardiology

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