Pfizer has secured approval from the US Food and Drug Administration (FDA) for its Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) to treat patients with transthyretin amyloid cardiomyopathy.
The approval has been granted for Vyndaqel and Vyndamax to treat cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization.
Transthyretin amyloid cardiomyopathy is a rare and fatal disease indicated by the buildup of abnormal deposits of misfolded protein called amyloid in the heart. It is defined by restrictive cardiomyopathy and progressive heart failure.
Vyndaqel and Vyndamax are oral transthyretin stabilizers that selectively bind to transthyretin, enabling to stabilize the tetramer of the transthyretin transport protein and slowing the formation of amyloid that causes ATTR-CM.
According to the company, the recommended dosage is either Vyndaqel 80mg orally once-daily, taken as four 20mg capsules, or Vyndamax 61mg orally once-daily, taken as a single capsule.
The approval was based on results from the pivotal phase 3 transthyretin amyloidosis cardiomyopathy clinical trial (ATTR-ACT).
The global double-blind, randomized and placebo-controlled clinical study was designed to assess a pharmacological therapy to treat transthyretin amyloid cardiomyopathy.
In the trial, Vyndaqel significantly decreased the hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo over a 30-month period, said the company.
In 2012, Vyndaqel secured orphan drug designation for ATTR-CM in the EU and the US in 2012, while in Japan in 2018.
In June 2017 and May 2018, respectively, the Vyndaqel secured fast track and breakthrough therapy designations from FDA for ATTR-CM.
Pfizer global product development rare disease chief development officer and senior vice president Dr Brenda Cooperstone said: “The approvals of VYNDAQEL and VYNDAMAX are a testament to the significant research and development investment in our innovative cardiovascular outcomes trial, ATTR-ACT. We are proud to bring these medicines to ATTR-CM patients who are in dire need of treatment.
“VYNDAQEL and VYNDAMAX reduce cardiovascular mortality and the frequency of cardiovascular-related hospital stays in patients with wild-type or hereditary forms of this rare disease, giving them a chance for more time with their loved ones.”