The Mavacamten is the first approved drug of a new class of drugs, which reduce the activity of "cardiac myosin", a protein fundamental for the contraction of heart cells, which plays a determining role in the creation of left ventricular outflow obstruction in most patients with obstructive hypertrophic cardiomyopathy.

The positive results of efficacy and safety of the Phase 3 experimental studies EXPLORER-HCM (2020) and VALOR-HCM (2022) have allowed the approval of "Mavacamten" by the FDA (Federal Drug Administration), and in July this year also by EMA (European Medical Agency) and subsequently by AIFA (Italian Medicines Agency) for the treatment of adult patients with Obstructive hypertrophic cardiomyopathy.

A few weeks ago, the activation of the Compassionate Use Program was officially published on the AIFA website, which makes Mavacamten (CAMZYOS) available free of charge, indicated for adult patients (over 18 years of age), with symptomatic hypertrophic obstructive cardiomyopathy ( class II-III according to the New York Heart Association, NYHA classification), who have exhausted or have not tolerated the available therapeutic alternatives.

For patients suffering from hypertrophic cardiomyopathy, but who do not meet these criteria, the drug cannot be prescribed, although the studies currently underway could extend the indications substantially in the coming years.

Treatment with Mavacamten must be requested and supervised by a cardiologist experienced in the management of patients with hypertrophic obstructive cardiomyopathy as, to have access to the treatment, some preliminary assessments and frequent clinical monitoring are necessary, especially in the first three months.  

The technical data sheet approved by AIFA shows the characteristics of the patients who can receive the treatment: Determine 131-2023 CAMZYOS.

Below is some important information reported in the technical data sheet:

  • Before starting treatment, the left ventricular ejection fraction (EF VS) of patients should be evaluated by echocardiography. If LVEF is < 55%, treatment should not be started.
  • Before starting treatment, women of childbearing age must have a negative pregnancy test
  • The treatment should be taken orally once daily, with or without meals, at approximately the same time each day.

L' "compassionate use" it is an important practice in medicine. It allows patients with particular characteristics to access drugs that have not yet been authorized for distribution in Italy.

In the specific case of patients with obstructive hypertrophic cardiomyopathy, the compassionate use of Mavacamten allows us to start using the drug in some selected patients and verify the therapeutic efficacy, such as reduction of symptoms, outflow obstruction and proBNP.