There’s no cure for obstructive hypertrophic cardiomyopathy (oHCM) — treatments focus on symptom management and preventing complications. But the landscape is changing, and more cutting-edge therapies are on the way.
Mavacamten (Camzyos), a cardiac myosin inhibitor and the first medication to target the underlying cause of oHCM, was approved in 2022. Now we’re on the cusp of many more therapies joining the oHCM arsenal.
“This is a hopeful time to be a patient who has limiting symptoms. People have so many more options on the table now, and so many more that are coming down the pike that are really worthwhile,” says Sara Saberi, MD, an assistant professor in the division of cardiovascular medicine at the University of Michigan in Ann Arbor.
Here’s what you may be able to expect in the next 5 to 10 years, plus what you can do to access cutting-edge oHCM treatments today.
In December 2025, the U.S. Food and Drug Administration (FDA) approved aficamten (Myqorzo), a second cardiac myosin inhibitor for the treatment of oHCM. Both mavacamten and aficamten work by slowing the interaction of the proteins actin and myosin, which are involved in muscle function. That helps the heart muscle calm down, so it doesn’t contract or squeeze so hard. “You just get a more normal contraction,” explains Anjali Tiku Owens, MD, the medical director of the Center for Inherited Cardiac Disease at Penn Medicine in Philadelphia.Both medications have been shown to be about equally effective in reducing symptoms like shortness of breath and chest pain.A third cardiac myosin inhibitor could be available in the coming years. Delocamten, which was designed to have a more flexible dosing schedule than its predecessors, is currently in phase 2 clinical trials.
Sodium-glucose cotransporter 2 (SGLT2) inhibitors have been shown to be effective for many types of heart failure. Now researchers are looking into whether this class of medications, which includes sotagliflozin, can help oHCM. The drugs work along metabolic pathways that remove excess sugar from the body. “That seems to have beneficial effects on cardiac function, though we don’t yet quite understand how the beneficial effects occur,” Dr. Owens says.A phase 3 clinical trial looking at sotagliflozin is expected to wrap up in August 2026. If the drug proves effective, it could be submitted to the FDA for approval for this use within months.
While cardiac myosin inhibitors encourage the heart to squeeze out blood or contract normally, a sarcomere modulator helps the heart fill with blood the way that it’s supposed to. “It basically acts to improve the heart muscle’s relaxation phase,” explains Dr. Saberi.
The investigational sarcomere modulator EDG-7500 is currently in a phase 2 clinical trial, and so far, it has shown promising results both for safety and improving heart function. If the drug gets good results in phase 3 clinical trials, experts like Saberi expect that it could get FDA approval sometime around 2030.
Surgery for oHCM has become much less common since cardiac myosin inhibitors came onto the scene. But it’s still needed in some cases, and emerging procedures could make surgery easier and lower risk.
Transapical beating heart septal myectomy is a less-invasive version of traditional septal myectomy, an open-heart surgery that removes a portion of the thickened heart muscle. “The approach makes a much smaller incision and doesn’t require the breastbone to be cracked and then healed. It’s a real game changer,” Saberi says. It was shown to be safe and effective in Chinese clinical trials, and may eventually be approved in the United States.Minimally invasive electro septal myectomy is another in-the-works alternative to traditional septal myectomy that small studies have shown is safe and effective. “It uses a tool that works sort of like an apple corer to take out a piece of the heart muscle. You can target the tool based on how much muscle you want to take out,” explains Saberi.Scientists have identified nearly 30 genes that could play a role in HCM development, most of which are involved in how the heart muscle contracts. In the future, gene editing technologies like CRISPR could be used to silence the faulty genes or replace them with normal ones, allowing a person’s heart muscle to contract the way it’s supposed to.
While these are exciting treatments that could potentially cure oHCM, “We’re at the very beginning with gene therapy, with the first few patients participating in phase I clinical trials,” says Owens. In order for gene therapy to become an approved treatment, many more years of research will be needed.
You may be a candidate for a clinical trial if advanced therapies like mavacamten or aficamten haven’t done enough to control your oHCM symptoms. To learn more about your options, you can research ongoing trials at ClinicalTrials.gov and talk with your cardiologist about which ones may be a good fit for you.
New medications for oHCM — cardiac myosin inhibitors like mavacamten and aficamten — target the disease more directly than ever before, and additional drugs in this class are in development.
Several other promising drug classes are in the pipeline, including SGLT2 inhibitors and sarcomere modulators, which may improve heart function in different ways and could expand treatment options in the coming years.
Less-invasive surgical techniques to septal myectomy are being developed to avoid open-heart surgery while still effectively removing excess heart muscle, which may reduce risk and recovery time.
Gene therapy, which could one day address the root genetic causes of oHCM, holds long-term potential but remains in the earliest stages of research.
