FDA approves new treatments for heart disease caused by a serious rare disease, transthyretin mediated amyloidosis

FDAOn May 3, the U.S. Food and Drug Administration approved Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules for the treatment of the heart disease (cardiomyopathy) caused by transthyretin mediated amyloidosis (ATTR-CM) in adults. These are the first FDA-approved treatments for ATTR-CM. 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."

ATTR is caused by the buildup of abnormal deposits of specific proteins known as amyloid in the body's organs and tissues, interfering with their normal functioning. 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.

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 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.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Most Popular Now

Pfizer and Lilly announce top-line results from lo…

Pfizer Inc. (NYSE:PFE) and Eli Lilly and Company (NYSE:LLY) announced top-line results from a Phase 3 study evaluating tanezumab 2.5 mg and 5 mg. The objective of the stu...

Bristol-Myers Squibb reports first quarter financi…

Bristol-Myers Squibb Company (NYSE:BMY) today reported results for the first quarter of 2019 which were highlighted by strong demand for Opdivo (nivolumab) and Eliquis (a...

Walnuts may help lower blood pressure for those at…

When combined with a diet low in saturated fats, eating walnuts may help lower blood pressure in people at risk for cardiovascular disease, according to a new Penn State ...

Amgen ignites a social fitness movement to support…

Amgen (NASDAQ:AMGN) launched the Breakaway Challenge initiative, a national social fitness program to motivate individuals to take action in their health and to support t...

Comprehensive tumor profiling promises new therape…

The WINTHER trial, NCT01856296, led by investigators from Vall d'Hebron Institute of Oncology - VHIO (Spain), Chaim Sheba Medical Center (Israel) (Raanan Berger), Gustave...

AstraZeneca starts artificial intelligence collabo…

AstraZeneca and BenevolentAI today began a long-term collaboration to use artificial intelligence (AI) and machine learning for the discovery and development of new treat...

Possible link between autism and antidepressants u…

An international team led by Duke-NUS Medical School has found a potential link between autistic-like behaviour in adult mice and exposure to a common antidepressant in t...

Chemotherapy or not?

Case Western Reserve University researchers and partners, including a collaborator at Cleveland Clinic, are pushing the boundaries of how "smart" diagnostic-imaging machi...

Researchers define Alzheimer's-like brain disorder

A brain disorder that mimics symptoms of Alzheimer's disease has been defined with recommended diagnostic criteria and guidelines for advancing future research on the con...

Drugs to prevent stroke and dementia show promise …

Treatments that prevent recurrence of types of stroke and dementia caused by damage to small blood vessels in the brain have moved a step closer, following a small study...

Amgen and Syapse enter precision medicine collabor…

Amgen (NASDAQ:AMGN), a world leader in biotechnology, and Syapse, a company powering precision medicine insights through its global provider network, announced a precisio...

Personalizing precision medicine with combination …

Precision oncology often relies on treating patients with a single, molecularly matched therapy that targets one mutation in their tumor. In a report, published online in...