European Commission Approves VYNDAQEL®, the First Treatment in the EU for Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

—VYNDAQEL is the only EC-approved medicine proven to reduce mortality and frequency of cardiovascular-related hospitalizations in adults with wild-type or hereditary ATTR-CM—

—VYNDAQEL is the first approved medicine in the EU to treat both ATTR-CM and stage 1 symptomatic transthyretin amyloid polyneuropathy (ATTR-PN)—

NEW YORK, NY, USA I February 18, 2020 I Pfizer Inc. (NYSE: PFE) announced today that the European Commission (EC) has approved 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). VYNDAQEL is the first and only treatment approved in the European Union (EU) for patients with ATTR-CM. Prior to this approval, treatment options for patients with ATTR-CM were restricted to symptom management, and, in rare cases, heart (or heart and liver) transplant.

“Until today, there were no approved medicines to treat patients with ATTR-CM in the EU. Today’s approval represents incredible progress for these patients and reflects our steadfast commitment to delivering breakthrough medicines to rare disease patients,” said Paul Levesque, Global President, Pfizer Rare Disease. “Additionally, with today’s milestone, VYNDAQEL is now the first treatment to have two formulations approved in the EU to treat manifestations of transthyretin amyloidosis: one for cardiomyopathy, and one for stage 1 polyneuropathy.”

ATTR-CM is a rare, underdiagnosed and 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. Once diagnosed, the median life expectancy in patients with ATTR-CM, dependent on sub-type, is approximately two to 3.5 years.

“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, MD, 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.”

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 tafamidis meglumine compared to those who received placebo.

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.

VYNDAQEL also had significant and consistent treatment effects compared to placebo on functional capacity and health status first observed at six months and continuing through 30 months. Specifically, VYNDAQEL reduced the decline in performance on the six-minute walk test (p<0.0001) and reduced the decline in health status as measured by the Kansas City Cardiomyopathy Questionnaire – Overall Summary score (p<0.0001).

VYNDAQEL was well tolerated in this 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. The approval is also based on findings from an evaluation of the free acid form of tafamidis 61 mg, which demonstrated that one 61 mg capsule of tafamidis free acid corresponds to an 80 mg tafamidis meglumine dose (4 x 20 mg capsules). The safety of the 61 mg dose was not evaluated in ATTR-ACT. The tafamidis 61 mg capsule was developed for patient convenience to enable a single capsule for daily administration.

In 2011, the tafamidis meglumine 20 mg capsule formulation of VYNDAQEL was approved in the EU for transthyretin amyloidosis in adult patients with stage 1 symptomatic polyneuropathy (ATTR-PN) to delay peripheral neurologic impairment.

About ATTR Amyloidosis

ATTR amyloidosis is rare, progressive disease characterized by the abnormal buildup of amyloid deposits composed of misfolded transthyretin protein in the body’s organs and tissues. 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. ATTR-CM and ATTR-PN are two presentations of the disease.

ATTR-CM affects the heart and leads to restrictive cardiomyopathy and progressive heart failure. There are two sub-types of ATTR-CM: hereditary, which is caused by a mutation in the transthyretin gene and can occur in people as early as their 50s and 60s; or the wild-type form which is associated with aging, and is thought to be more common, usually affecting men after age 60. Often ATTR-CM is diagnosed only after symptoms have become severe.

ATTR-PN results from a genetic mutation of the transthyretin gene causing amyloid fibrils to form in the peripheral and autonomic nerves. ATTR-PN typically occurs during active adult years with onset as early as the 30s in some patients, followed by disease progression that may reach the terminal stage in approximately 10 years on average from disease onset.

About VYNDAQEL (tafamidis 61 mg) and VYNDAQEL (tafamidis meglumine 20 mg)

VYNDAQEL (tafamidis 61 mg) and VYNDAQEL (tafamidis meglumine 20 mg) are oral transthyretin stabilizers that selectively bind to transthyretin, stabilizing the tetramer of the transthyretin transport protein and slowing the formation of amyloid.

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. VYNDAQEL 61 mg and VYNDAQEL 20 mg are not substitutable on a per milligram basis.

Tafamidis was granted Orphan Drug Designation for ATTR-CM in both the EU and US in 2012 and in Japan in 2018. Tafamidis was approved for the treatment of ATTR-CM in Japan under SAKIGAKE designation in March 2019, in the United States in May 2019, in the United Arab Emirates in November 2019, in Brazil in December 2019 and in Canada in January 2020.

VYNDAQEL (tafamidis meglumine) 20 mg was first approved in 2011 in the EU for the treatment of transthyretin amyloid polyneuropathy (ATTR-PN), in adult patients with stage 1 symptomatic polyneuropathy to delay peripheral neurologic impairment. Currently, it is approved for ATTR-PN in over 40 countries, including Japan, countries in Europe, Brazil, Mexico, Argentina, Israel, Russia, and South Korea. VYNDAQEL is not approved for ATTR-PN in the US.

VYNDAQEL (tafamidis), a once-daily 61 mg oral capsule, was granted marketing authorization for patients with ATTR-CM in the EU in February 2020.

Pfizer Rare Disease

Rare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including hematology, neuroscience, and inherited metabolic disorders.

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.

Click here to learn more about our Rare Disease portfolio and how we empower patients, engage communities in our clinical development programs, and support programs that heighten disease awareness.

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At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.pfizer.com. In addition, to learn more, please visit us on www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

SOURCE: Pfizer

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