The European Commission approved the marketing of Vyndaqel, Pfizer’s treatment of the rare and progressive, and life-threatening disease transthyretin amyloidosis in adult patients with cardiomyopathy.
Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is characterized by the buildup of abnormal deposits of misfolded protein called amyloid in the heart and is defined by restrictive cardiomyopathy and progressive heart failure. ATTR amyloidosis can impact numerous organs and tissues in the body, including the peripheral nervous system, and organs such as the heart, kidneys, gastrointestinal tract and eyes. Once diagnosed, the median life expectancy in patients with ATTR-CM, dependent on sub-type, is approximately two to 3.5 years.
Vyndaqel is the first and only treatment approved in the European Union for patients with ATTR-CM. Prior to the approval of Vyndaqel, treatment options for patients with ATTR-CM in the European Union were restricted to symptom management, and, in rare cases, heart (or heart and liver) transplant.
The EC approval of Vyndaqel is based on results from the phase 3 ATTR-ACT study, the first and only completed global, double-blind, randomized, placebo-controlled clinical trial to investigate a pharmacologic therapy for the treatment of ATTR-CM. The study compared patients who received an oral daily dose of 20 mg or 80 mg of Vyndaqel compared to those who received placebo.
In the primary analysis of the study, Vyndaqel demonstrated a significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo over a 30-month period in patients with wild-type or hereditary ATTR-CM. The primary analysis demonstrated a relative reduction in the risk of all-cause mortality and frequency of cardiovascular-related hospitalization of 30 percent with the lower dose and 32 percent with the higher dose of Vyndaqel versus placebo.
Vyndaqel was well tolerated in the study, with an observed safety profile comparable to placebo. The frequency of adverse events in patients treated with Vyndaqel was generally similar and comparable to placebo.
In 2011, the European Union approved a 20 mg capsule formulation of Vyndaqel for transthyretin amyloidosis in adult patients with stage 1 symptomatic polyneuropathy (ATTR-PN) to delay peripheral neurologic impairment.
“Before today, the European transthyretin amyloidosis community had a dire need for new therapeutic options that can improve outcomes for patients with cardiomyopathy,” said Thibaud Damy, coordinator of the French Referral Centers for Cardiac Amyloidosis and past president of the French Heart Failure and Cardiomyopathy group, French Society of Cardiology. “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.”
Photo: Thibaud Damy, coordinator of the French Referral Centers for Cardiac Amyloidosis and past president of the French Heart Failure and Cardiomyopathy group, French Society of Cardiology.
Author: Rare Daily Staff
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