Stabilizing the underlying cause of a rare heart condition
For patients and physicians grappling with ATTR-cardiac amyloidosis (ATTR-CM), Attruby® (acoramidis) brings the possibility of hope.
Please see Important Safety Information for Attruby below. For the Full Prescribing Information and Patient Information for Attruby, visit Attruby.com/PI.
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What does it mean to live with a chronic heart condition?
Day-to-day life with a chronic heart condition is about much more than increased risk of heart attack or stroke. Proper circulation of blood is essential to every part of our overall health, meaning that any issues in the heart may cause ripple effects throughout the body. From swelling in the legs to shortness of breath to persistent fatigue, heart disease can present symptoms that can be difficult to diagnose. And when the condition is rare, diagnosis and treatment become even more challenging.
For much of Dr. Andrew Darlington’s career, he saw patients suffering from ATTR-CM — complaining of shortness of breath and chronic fatigue — and found himself with limited options to treat the underlying disease. As his patients’ health declined, they would find themselves in and out of the hospital, losing not only their health but their quality of life. Often, the condition would progress until they succumbed to the disease.
“From diagnosis to death, it could be as short as two to three years,” said Darlington, a heart failure cardiologist with Piedmont Heart Institute in Atlanta. “We were desperately in need of options.”
Over the last decade, those options have arrived. Although ATTR-CM remains incurable, Darlington said that over the last ten years an “explosion of innovation” has changed how medical providers can approach this disease.
“We’ve known about this disease for quite a long time. Only recently have we been able to make a difference for patients.”
Last year the U.S. Food and Drug Administration approved Attruby, a prescription medication that treats adults with ATTR-CM, working to help slow the progression of the disease. A 30-month clinical study that compared the effectiveness of Attruby to a placebo found that Attruby was shown to impact survival, hospitalizations due to heart issues, and health-related quality of life, so that people living with ATTR-CM may have more opportunities to experience key moments in their lives. Possible side effects include diarrhea and abdominal pain; for most people, these were mild and went away so they could continue taking Attruby.
The importance of a protein
Attruby works by targeting the disease at its source. One of the most abundant proteins in the body is transthyretin, also known as prealbumin or TTR, which helps transport the metabolism-regulating hormone thyroxine and retinol (vitamin A) through the bloodstream. This vital protein provides essential support for organs ranging from the heart to the eyes to the skeletal system. People develop ATTR-CM when their TTR breaks down and reforms into abnormal proteins called amyloid fibrils. Over time, the amyloid fibrils build up throughout the body and stop things from working properly, including the heart.
Attruby treats the condition by helping to keep the TTR proteins together. By stabilizing TTR proteins, Attruby helps prevent abnormal amyloid fibrils from forming. The result means less buildup of amyloid fibril deposits that could otherwise thicken the walls of the heart and cause the symptoms associated with ATTR-CM, including heart failure.
Lab results
have shown that
Attruby stabilized over 90% of TTR proteins
in as little as 28 days
A follow-up model-based study of people taking Attruby in the clinical trial found that higher TTR levels were linked to better survival in people living with ATTR-CM, regardless of other factors. This means that their heart stayed healthy for longer, they had fewer hospital visits, and they saw an easing of their ATTR-CM symptoms. In the words of Dr. Darlington, "without stable TTR, we see progression (of ATTR-CM).”
One patient’s story
For adults living with ATTR-CM, Attruby’s effects can be meaningful. For Ken, an 80-year-old patient who was diagnosed with ATTR-CM after suffering a stroke, the condition threatened not only to shorten his life but to make it harder for him to enjoy the time he had left. Attruby helped give him and his family hope.
Ken’s journey from diagnosis to treatment took a winding path. When his doctors told him the condition was untreatable, he found himself losing hope. “Hearing doctors say, ‘There is no treatment,’ is much more devastating than saying, ‘I don’t know how to treat it,’” he explained. Seeking solace and understanding, he began attending a support group for patients with ATTR-CM, and it was there that he learned about Attruby’s clinical trial. When a doctor gave a presentation on the clinical trial to evaluate Attruby, Ken found him so deeply knowledgeable and excited about the possibilities of this potential treatment that afterwards he asked to become his patient. It is important to always talk to a physician about all other medication and any possible side effects. Although individual results may vary, Ken believes that Attruby had made a meaningful difference to his health-related quality of life.
After he started taking Attruby, Ken noticed an impact on his symptoms. Before the treatment, he struggled with shortness of breath so severe that he couldn’t push his lawn mower more than 50 feet. Now with Attruby, he finds himself able to enjoy yard work once again. He’s been able to travel, spend time with his family, and walk his dogs. He feels optimistic again — and that means a lot.
“What can I say? I'm thankful for it.”
Raising awareness for both people living with ATTR-CM and clinicians alike will be key moving forward, Darlington says, so that people not only recognize the symptoms but also know that treatment is available. “We've come a long way,” Darlington said, adding that timing is everything. “We're diagnosing it earlier than we ever have. There are more treatments than ever, but we still see people succumb to this disease because they didn’t start treatment early enough.”
With advances in research and treatment options like Attruby, Darlington is happy to see progress being made in addressing this rare heart condition. “Things are changing quickly,” he added. “I’m looking forward to seeing how this progress will affect the lives of people living with ATTR-CM.”