The U.S. Food and Drug Administration recently approved acoramidis for the treatment of transthyretin cardiac amyloidosis (ATTR-CM), a life-threatening condition characterized by abnormal protein deposits in the heart, BridgeBio Pharma Inc. announced. BridgeBio markets acoramidis under the brand name Attruby.
The FDA “approved Attruby (acoramidis) to treat adults with cardiomyopathy (disorder that affects heart muscle) of wild-type or variant (hereditary) transthyretin-mediated amyloidosis (ATTR-CM) to reduce death and hospitalization related to heart problems,” the FDA announced last month.
What is ATTR-CM?
Transthyretin amyloidosis cardiomyopathy (ATTR-CM) is a rare progressive disease of the heart muscle that leads to congestive heart failure. It occurs when the transthyretin protein produced by the liver is unstable. Symptoms include fatigue; shortness of breath; irregular heart rate or palpitations; swelling of the legs, ankles and stomach; brain fog; wheezing; and dizziness. It often goes underdiagnosed because of a lack of awareness and knowledge of the disease. There is currently no cure for ATTR-CM.
Acoramidis is a first-of-its-kind therapy that achieved near-complete stabilization (greater than 90%) of transthyretin (TTR), the protein central to this disease, BridgeBio said. The stabilization significantly reduced cardiovascular death and hospitalizations, the company added.
Read more about ATTR-CM treatment and care
“Transthyretin cardiac amyloidosis is a progressive disease with a poor prognosis when left untreated,” Dr. Martha Grogan of the Mayo Clinic said in a BridgeBio news release. “Having a new first line treatment option which provides excellent TTR stabilization and improves outcomes in this disease gives patients more options.”
Grogan added: “Encouraging data suggests Attruby reduces all-cause mortality and cardiovascular hospitalization as early as three months after initiation of therapy. With continued advances in therapy, this previously fatal disease is becoming a manageable chronic cardiovascular condition.”
The phase 3 ATTRibute-CM trial involved 632 participants. Treatment with acoramidis led to a 42% reduction in the combined risk of death and cardiovascular-related hospitalizations compared to treatment with placebo over 30 months. The therapy also halved the frequency of cardiovascular hospitalizations. Patients in the study experienced improved quality of life as reflected in scores from the Kansas City Cardiomyopathy Questionnaire and performed better in a six-minute walk test.
Acoramidis works by preserving the function of TTR, a protein essential for transporting vitamin A and thyroxine. Designed to mimic a rare protective genetic mutation, it addresses the root cause of ATTR-CM by stabilizing the native structure of TTR, the company said.
