Technology | Heart Failure | May 06, 2019

FDA Clears New Treatments for Cardiomyopathy Caused by Transthyretin Mediated Amyloidosis

This is the first treatment for this rare but serious form of heart disease that leads to heart failure

Tafamidis meglumine (Vyndaqel) is one of two new drugs cleared by the FDA for the treatment of the cardiomyopathy caused by transthyretin mediated amyloidosis (ATTR-CM) in adults. These are the first FDA-approved treatments for ATTR-CM. U.S. Food and Drug Administration (FDA) approved tafamidis meglumine (Vyndaqel) and tafamidis (Vyndamax) capsules for the treatment of the cardiomyopathy.

Tafamidis meglumine (Vyndaqel) is one of two new drugs cleared by the FDA for the treatment of the cardiomyopathy caused by transthyretin mediated amyloidosis (ATTR-CM) in adults. These are the first FDA-approved treatments for ATTR-CM. 

May 6, 2019 — The U.S. Food and Drug Administration (FDA) approved tafamidis meglumine (Vyndaqel) and tafamidis (Vyndamax) capsules for the treatment of the cardiomyopathy caused by transthyretin mediated amyloidosis (ATTR-CM) in adults. These are the first FDA-approved treatments for ATTR-CM. 

The drugs are two oral formulations of the first-in-class transthyretin stabilizer tafamidis. Vyndaqel and Vyndamax have the same active moiety, tafamidis, but they are not substitutable on a milligram to milligram basis and their recommended doses differ.

“Transthyretin-mediated amyloidosis is a rare, debilitating and often fatal disease,” said Norman Stockbridge, M.D., Ph.D., director of the Division of Cardiovascular and Renal Drugs in the FDA’s Center for Drug Evaluation and Research. “The treatments we’re approving today are an important advancement in the treatment of the cardiomyopathy caused by transthyretin-mediated amyloidosis.”

Reuters reported Pfizer believes this drug may be a block buster and that the company set a list price of $225,000 a year for the medication. Analysts are forecasting annual sales to exceed $1 billion in 2024, according to Refinitiv data.

 

What is Transthyretin Amyloid Cardiomyopathy?

Transthyretin amyloid cardiomyopathy 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. The amyloid interferes with the normal functioning of organs and can lead to progressive heart failure. 

“ATTR-CM is not only fatal, but also significantly underdiagnosed, with some patients cycling through multiple doctors and a myriad of tests over a period of years while the disease progresses,” said Isabelle Lousada, Founder and CEO, Amyloidosis Research Consortium. “ATTR-CM is a rare disease for which more education and awareness is needed. The approval of these medicines represents an important advance for patients; however, it is equally important that we work as a community to recognize the critical importance of early diagnosis.”

These protein deposits most frequently occur in the heart and the peripheral nervous system. Heart involvement can result in shortness of breath, fatigue, heart failure, loss of consciousness, abnormal heart rhythms and death. Involvement of the peripheral nervous system can result in a loss of sensation, pain, or immobility in the arms, legs, hands and feet. Amyloid deposits can also affect the kidneys, eyes, gastrointestinal tract and central nervous system.

Previously, there were no medicines approved to treat ATTR-CM. The only available treatment options included symptom management, and, in rare cases, heart (or heart and liver) transplant. It is estimated that the prevalence of ATTR-CM is about 100,000 people in the U.S. and only 1-2 percent of those patients are diagnosed today.

 

The Efficacy of the New Vyndaqel and Vyndamax Drugs

The FDA said the efficacy of Vyndaqel and Vyndamax in treating ATTR-CM was shown in a clinical trial of 441 patients randomized to receive Vyndaqel or a placebo. After an average of 30 months, the survival rate was higher in the Vyndaqel group than in the placebo group. Vyndaqel was also shown to reduce the number of hospitalizations for cardiovascular problems.

The number of patients in clinical studies was small, but no drug-associated side effects have been identified. Tafamidis may cause fetal harm when administered to a pregnant woman. Women taking Vyndaqel or Vyndamax should discuss pregnancy planning and prevention with their health care professional.

The recommended dosage is either Vyndaqel 80 mg orally once-daily, taken as four 20 mg capsules, or Vyndamax 61 mg orally once-daily, taken as a single capsule. Vyndamax was developed for patient convenience; Vyndaqel and Vyndamax are not substitutable on a per milligram basis.

 

FDA Granted Fast Track, Break-through Approval for The Drugs

The FDA granted Vyndaqel Fast Track, Priority Review and Breakthrough Therapy designations. Vyndaqel and Vyndamax each received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

Approval of Vyndaqel and Vyndamax were granted to FoldRx, a subsidiary of Pfizer.

For more information: www.pfizer.com/news/press-release

 

Related Content:

Tafamidis Improves Survival in Rare Heart Condition

Pfizer Announces Positive Topline Results from Phase 3 ATTR-ACT Study

25 Most Impactful Nuclear Cardiology Articles

Related Content

Research Shows Drug Can Extend Survival Rates for Heart Failure Patients
News | Heart Failure | October 16, 2019
Researchers have shown for the first time in preclinical studies that the drug Aliskiren can delay the progression of...
The COAPT Economic Analysis sub-study found it is cost effective to treat heart failure patients who have severe secondary mitral regurgitation with MitraClip to reduce their MR symptoms. #TMVR #TCT2019 #TCT19

The COAPT Economic Analysis sub-study found it is cost effective to treat heart failure patients who have severe secondary mitral regurgitation with MitraClip to reduce their MR symptoms.

News | Heart Failure | October 04, 2019
October 4, 2019 – Results of a new economic analysis of the COAPT Trial data found that transcatheter mitral valve re
COAPT Trial three-year data show the MitraClip continued effectiveness in heart failure patients with secondary mitral reguritation. #TCT2019 #TCT19 #mitralclip

COAPT Trial three-year data show the MitraClip continues to show safety and effectiveness in heart failure patients with secondary mitral reguritation.

News | Heart Failure | October 03, 2019
October 3, 2019 – The three-year results from the...
Positive Safety, Efficacy Data Reported on AccuCinch Heart Failure Device
News | Heart Failure | September 27, 2019
Ancora Heart Inc. announced results from an interim analysis of heart failure patients treated in the CorCinch FMR...
Max and Debra Ernst Heart Center Opening at Beaumont Hospital, Royal Oak
News | Heart Failure | September 20, 2019
The new Max and Debra Ernst Heart Center at Beaumont Hospital, Royal Oak, Mich., will open Sept. 30. It is dedicated to...
FDA Grants Fast Track Designation for Farxiga in Heart Failure
News | Heart Failure | September 18, 2019
AstraZeneca announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the...
Corvia Medical and physIQ Partner in Global Phase 3 Heart Failure Device Clinical Trial

The REDUCE LAP HF-II study will combine Corvia Medical's InterAtrial Shunt Device (IASD), pictured here, with physIQ's continuous monitoring platform to evaluate the device's efficacy in patients with heart failure. Image courtesy of Corvia Medical.

News | Heart Failure | September 12, 2019
Corvia Medical has sponsored and is actively enrolling patients in a heart failure (HF) device trial that, in addition...
 Tiny Wearable Cameras May Improve Quality of Life in Heart Failure Patients

Image courtesy of Amazon.com

News | Heart Failure | September 11, 2019
The ever-present devices that seem to track all our moves can be annoying, intrusive or worse, but for heart failure...
PARAGON-HF Misses Endpoint in Preserved Heart Failure
News | Heart Failure | September 11, 2019
The angiotensin neprilysin inhibitor sacubitril/valsartan (Entresto) missed its primary endpoint of reducing total...
Farxiga Significantly Reduces Cardiovascular Death and Worsening of Heart Failure
News | Heart Failure | September 09, 2019
AstraZeneca announced detailed results from the landmark Phase III DAPA-HF trial that showed Farxiga (dapagliflozin) on...
Overlay Init