As originally reported in Pfizer, the European Commission has just approved the first treatment option ever available to treat transthyretin amyloidosis cardiomyopathy (ATTR-CM). The medication, VYNDAQEL, taken orally once daily, is additionally novel because of the drug’s two formulas enabling it to treat two variations of the disease: ATTR-CM and stage 1 polyneuropathy.
Transthyretin Amyloidosis
Transthyretin amyloidosis is a disease resulting from a misfolded protein that creates an excess of amyloid. Transthyretin amylodis can occur in any number of the organs or tissues, in turn causing damage. However, this drug addresses the heart-related subtypes of the disease, ATTR-CM, which specifically affects the cardiovascular system. The drug impacts two specfic ATTR-CM subtypes: the hereditary version which most often appears in the 50s, and the “wild-type” which affects men almost exclusively. The wild-type is a disease usually seen later in life. The disease is progressive, meaning before this treatment option, the the median life expectancy following diagnosis was two to three and a half years.
The Trial Results
The drug significantly improved various symptoms, including reducing death for any reason by 30% and hospitalization due to heart health by 32% in comparison to the placebo. The drug additionally assisted patients with further functional capacity measured with walking tests and their measures showed it delayed declines in health status.
The only treatment options previously available addressed symptomatic effects or necessitated undergoing a heart transplant with no chance of a complete cure. Disease expert Dr. Thibaud Damy explained the momentous impact of this new option, which addresses a lethal and painful progression of the disease which rapidly robs patients of their lives. Dr. Damy had this to say:
“VYNDAQEL represents a major advance for patients, as it can significantly reduce all-cause mortality and the frequency of cardiovascular-related hospitalizations in patients with wild-type or hereditary ATTR-CM.”
For patients with the disease, the step to one option from none is a world apart.
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