Drugs Xagena
The FDA ( U.S. Food and Drug Administration ) has granted Orphan Drug Designation for MYK-461 for treatment of symptomatic obstructive hypertrophic cardiomyopathy ( oHCM ), a subset of hypertrophic cardiomyopathy ( HCM ).
MYK-461 is the first therapy designed to target the underlying cause of hypertrophic cardiomyopathy. MyoKardia’s proposed initial indication for MYK-461 is obstructive hypertrophic cardiomyopathy.
MYK-461, is an orally administered small molecule designed to reduce excessive cardiac muscle contractility leading to hypertrophic cardiomyopathy.
Three phase 1 clinical trials have been initiated to assess MYK-461’s ability to modulate cardiac myosin by measuring reduction in cardiac muscle contractility via echocardiography.
Cardiac muscle contractility is an important biomarker of hypertrophic cardiomyopathy.
Data from two of the phase 1 trials have demonstrated clinical proof of mechanism in both HCM patients and healthy volunteers, and have indicated that MYK-461 is well tolerated with dose-proportional pharmacokinetics.
Symptomatic obstructive hypertrophic cardiomyopathy is a subset of a group of genetically-driven forms of heart failure that result from biomechanical defects in cardiac muscle contraction.
It is estimated that as many as 630,000 people in the United States have a form of hypertrophic cardiomyopathy.
Hypertrophic cardiomyopathy is defined as an otherwise unexplained thickening of the walls of the heart, known as hypertrophy. The consequences include reduced blood volumes and cardiac output, reduced ability of the left ventricle to expand, and high filling pressures.
These can all contribute to reduced effort tolerance and symptoms that include shortness of breath and chest pain.
Hypertrophic cardiomyopathy is a chronic disease, and for the majority of patients, the disease progresses slowly and can be extremely disabling.
Hypertrophic cardiomyopathy can also cause stroke or sudden cardiac death, and is the most common cause of sudden cardiac death in young people.
There are currently no approved therapeutic products indicated for the treatment of hypertrophic cardiomyopathy. Patients are typically prescribed one or more drugs ( including beta blockers, non-dihydropyridine calcium channel blockers and Disopyramide ) indicated for the treatment of hypertension, heart failure or other cardiovascular disorders more generally.
For a subset of HCM patients with more advanced disease progression or more pronounced symptoms, surgical or other invasive interventions may be appropriate. ( Xagena )
Source: MyoKardia, 2016
XagenaMedicine_2016
