The US Food and Drug Administration (FDA) is set to review Bristol Myers Squibb’s (BMS) investigational candidate mavacamten for patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
Hypertrophic cardiomyopathy (HCM) is a chronic and progressive disease which causes excessive contraction of the heart muscles and reduces the ability of the left ventricle to fill.
In both obstructive or non-obstructive HCM patients, physical exertion can cause fatigue or shortness of breath, and has also been associated with increased risks of atrial fibrillation, stroke, heart failure and sudden cardiac death.
BMS’ new drug application (NDA) for mavacamten in this indication is based on results from the phase 3 EXPLORER-HCM trial in patients with symptomatic oHCM versus placebo.
In this study, mavacamten met the primary endpoint of a composite functional score, designed to assess symptoms and cardiac function. When compared to placebo, patients on mavacamten had greater reductions in post-exercise LVOT gradient as well as improved symptom scores.
The investigational drug candidate met all primary and secondary endpoints in the phase 3 study, BMS said in a statement.
“Today’s acceptance from the FDA puts us one step closer to having a highly targeted therapeutic approach for oHCM, as mavacamten is a first-in-class myosin inhibitor developed to address the underlying molecular defect of the disease,” said Roland Chen, senior vice president, cardiovascular development, BMS.
“We are committed to supporting patients in need of HCM treatment and look forward to working with the FDA,” he added.
BMS picked up mavacamten as part of its $13.1bn acquisition of MyoKardia, announced last year. This experimental candidate is a potentially first-in-class allosteric modulator of cardia myosin, believed to work by reducing cardiac muscle contractility.
In addition to oHCM, BMS is also set to explore the potential of mavacamten in additional indications, including non-obstructive HCM.
On top of mavacamten, BMS is developing MyoKardia’s pipeline of novel compounds, which comprises two clinical-stage therapeutics, danicamtiv and MYK-224, as well as two pre-clinical assets, ACT-1 and LUS-1.
The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date for a decision on mavacamten in oHCM on 28 January 2022.