FDA approves Bristol-Myers drug for inherited heart disease known to kill young athletes

May 2, 2022

U.S. health regulators have approved a pill from Bristol-Myers Squibb  that is the first to treat people with the most common genetic heart disease, reports The Wall Street Journal.

.The Food and Drug Administration cleared the drug for people showing symptoms of the condition, which can lead to blood clots, strokes and in rare cases, death, Bristol said April 28.

The disease, called hypertrophic cardiomyopathy (HCM), causes the heart muscles to thicken excessively and make it difficult for the organ to pump blood. The condition affects about 1 in 500 people. It is the most common cause of sudden cardiac death in young people, including athletes, although it is rare.

However, since young athletes rarely are diagnosed with the condition, the pills might not be prescribed for them.

Possible symptoms of HCM include shortness of breath, rapid heartbeat, chest pain, dizziness, unexplained tiredness, and fainting.

The new drug, which carries the chemical name mavacamten and which Bristol will market as Camzyos, was the centerpiece of the company’s $13.1 billion acquisition of MyoKardia in 2020. Bristol is counting on sales to help offset several upcoming patent expirations for key products.

Camzyos could generate more than $2 billion in sales by 2026, according to analysts at Bernstein. Bristol said it priced Camzyos at $89,500 per year.

“This is a humongous step in the direction of trying to find transformative medicines for patients with obstructive hypertrophic cardiomyopathy,” Samit Hirawat, Bristol’s chief medical officer, said in an interview.

In HCM patients, heart walls are thicker and the organ contracts more forcefully, decreasing the flow of blood out of the heart. Camzyos blocks a protein, called myosin, that is believed to play a key role in causing the contractions.

The drug’s approval is for a common form of the disease, known as obstructive HCM. In that form, blood flow is partially blocked from the left ventricle, the heart’s main pumping chamber, to the aorta.

The FDA based its decision on a 251-subject late-stage study from Bristol that found Camzyos significantly improved heart function, quality of life and oxygen consumption in patients with HCM after 30 weeks.

No serious safety concerns were observed, the researchers said.

Research contact: @WSJ