New Heart Drug Gives Hope to 1 in 700 People Worldwide

Sarah Efron couldn't walk her corgi up the hills in her Portland neighborhood without chest pain stopping her in her tracks. The 39-year-old had watched hypertrophic cardiomyopathy kill four family members, and her own heart was failing so badly that doctors predicted she'd need a transplant within a decade.

Today, she's hiking again. A new drug called aficamten has given her something she thought she'd lost forever: a future.

Researchers just announced results from a major clinical trial testing the medication on 517 people with nonobstructive hypertrophic cardiomyopathy. This inherited condition causes the heart muscle to thicken, making even simple activities exhausting and painful. About 1 in 700 people worldwide live with this version of the disease, and until now, they had no medications to help.

The trial, led by Dr. Ahmad Masri at Oregon Health & Science University, showed dramatic improvements. After 36 weeks, patients taking aficamten could exercise longer, reported better quality of life, and showed improved clinical outcomes compared to those receiving a placebo. The study followed participants for up to 72 weeks across 160 health centers in 19 countries.

"This is the first-ever successful therapy for nonobstructive hypertrophic cardiomyopathy," said Masri, who reported the findings in the New England Journal of Medicine and at a major cardiology conference in Munich. "These results are highly encouraging for patients with this condition and for cardiologists who struggle to help them."

The medication works by relaxing the heart muscle, essentially giving the heart room to breathe. For patients like Efron, who became the first person in the world enrolled in this trial three years ago, the difference has been life-changing.

"I was dealing with chest pain daily, and that has almost disappeared with the drug," Efron said. She's been taking aficamten since March 2025 and can now walk stairs and hills without problems. "I've been able to go out and do more things, whether it's taking a hike or spending the whole day out in the world like a normal person."

Many people don't know they have this condition unless it runs in their family, making it frequently underdiagnosed and mismanaged. Without proper care, it can cause sudden death from cardiac arrest. Until now, treatment focused solely on lifestyle changes like managing blood pressure, weight, and sleep apnea.

Why This Inspires

This breakthrough represents hope for hundreds of thousands of people worldwide who've been told to simply live with their limitations. Efron's cardiologist can now offer something he never could before: a pill that actually makes patients feel better.

The trial recruited participants from 19 countries, with OHSU enrolling the most patients of any single site. That global collaboration means the results apply across diverse populations, bringing this hope to communities everywhere.

For Efron, who lost loved ones to this disease throughout her life, the medication means more than just physical relief. "This drug gives me hope for the future," she said.