Pfizer Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of VYNDAQEL (tafamidis), a once-daily 61 mg oral capsule, for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
“The CHMP positive opinion of VYNDAQEL for ATTR-CM reflects our steadfast commitment to improving outcomes for patients living with this rare and fatal disease,” said Brenda Cooperstone, MD, Senior Vice President and Chief Development Officer, Rare Disease, Pfizer Global Product Development. “In ATTR-ACT, VYNDAQEL reduced mortality and the frequency of cardiovascular-related hospitalizations in patients with wild-type or hereditary forms of the disease. If approved, VYNDAQEL would represent a real breakthrough for patients.”
ATTR-CM is a rare, life-threatening disease 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. On average, patients live only 2 to 3.5 years following diagnosis.
“For those living with ATTR-CM, a progressive and fatal rare disease, there are currently no available pharmacologic treatments for patients,” said Jean-Christophe Fidalgo, President of the Amyloidosis Alliance. “The Amyloidosis Alliance applauds the CHMP opinion, and we hope the EC will swiftly approve VYNDAQEL for ATTR-CM so patients can receive timely access to this medicine.”
In 2011, a different form of VYNDAQEL, tafamidis meglumine 20 mg capsule, was approved in the EU for transthyretin amyloidosis in adult patients with stage 1 symptomatic polyneuropathy (ATTR-PN) to delay peripheral neurologic impairment. For ATTR-CM, the tafamidis 61 mg capsule corresponds to an 80 mg tafamidis meglumine dose (4x 20mg capsules) and was developed for patient convenience to enable a single capsule for daily administration.
The European line extension application was based on the Phase 3 ATTR-ACT study, the first and only completed global, double-blind, randomised, placebo-controlled clinical trial to investigate a pharmacologic therapy for the treatment of ATTR-CM. In the primary analysis of the study, VYNDAQEL (tafamidis meglumine) demonstrated a significant reduction in the hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospitalisations compared to placebo over a 30-month period in patients with wild-type or hereditary ATTR-CM (p=0.0006). Additionally, individual components of the primary analysis demonstrated a relative reduction in the risk of all-cause mortality and frequency of cardiovascular-related hospitalization of 30% (p=0.026) and 32% (p<0.0001), respectively, with VYNDAQEL versus placebo. The application is also based on findings from an evaluation of the free acid form of tafamidis 61 mg. The ATTR-ACT primary results were presented in a Hot Line session at the ESC Congress 2018 in Munich, Germany, and simultaneously published online in the New England Journal of Medicine (NEJM) in August 2018.
The CHMP’s opinion will now be reviewed by the EC and a final decision is expected in the coming months.