¡New FDA approval for rare genetic disease: Amyloidosis (ATTR), to reduce cardiovascular death and hospitalizations for related causes!
First and only product approved in this field
November 26 2024
¿What is ATTR Amyloidosis?
Amyloidosis is a disease caused by the buildup of abnormal proteins, called amyloid, in the body’s peripheral organs and nerves. These protein deposits can cause organs to not function properly (for example, the heart) and cause nerve damage.
Transthyretin amyloid cardiomyopathy (ATTR-CM: Transthyretin amyloid cardiomyopathy) it is a progressive heart disease caused by the extracellular deposition of transthyretin (TTR) amyloid fibrils, either in its inherited form (ATTRv) or the wild-type form (ATTRwt).
Diagnosing the hereditary nature of ATTR-CM allows for genetic counseling, familial screening (facilitating early diagnosis of relatives), and, if polyneuropathy is present, allows for treatment with certain TTR-specific medications. Unfortunately, ATTR-CM is often assumed to be of wild-type origin, and genetic testing for TTR is not performed when ATTR-CM is diagnosed in older individuals.
PharmaBridgeBio Inc., is a new type of biopharmaceutical company focused on genetic diseases. The U.S. Food and Drug Administration (FDA) approved Attruby™ (acoramidis), an orally administered near-complete (≥90%) transthyretin (TTR) stabilizer, for the treatment of adults with wild-type or variant transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM) to reduce cardiovascular death and cardiovascular-related hospitalization.
FDA approval is based on positive results seen in the Phase 3 ATTRibute-CM study, where Attruby significantly reduced cardiovascular-related death and hospitalization and improved quality of life. Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR.