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Yale Offers New Medication for Fatal Disease

October 07, 2019
by Elisabeth Reitman

New Amyloidosis Therapies

Onpattro®, TegsediTM, and Vyndaqel®/Vyndamax® represent a different treatment approach for hATTR. Onpattro (patisiran), which was developed by Alnylam Pharmaceuticals, uses a promising technology known as RNA interference, or RNAi to regulate the production of genes. Akcea Therapeutics’ drug Tegsedi (inotersen) interferes with the production of transthyretin (TTR) protein to prevent the buildup of amyloid deposits. Pfizer’s oral medications Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) are the first medications approved by the U.S. Food & Drug Administration (FDA) to treat transthyretin amyloid cardiomyopathy (ATTR-CM), an under-diagnosed disease caused by a buildup of transthyretin protein in the heart.

“Yale has rapidly developed a national reputation for the evaluation and treatment of cardiac amyloidosis, offering state-of-the-art diagnostic tools and therapeutic strategies to our patients,” says Edward Miller, MD, PhD, associate professor of medicine and radiology and director of the Clinical Nuclear Cardiology Laboratory at Yale New Haven Hospital.

TTR amloidosis causes polyneuropathy and cardiomyopathy. Onpattro, which was approved by the FDA in August 2018, is the first small interfering RNA-based (siRNA) treatment, and the first FDA-approved drug to treat this condition by inhibiting hepatic synthesis of TTR.

Tegsedi, which was approved by the FDA in October 2018, is made from chemically synthesized RNA molecules. Although both therapies provide valuable benefits for patients, Tegsedi may cause significant reductions in platelet count and could lead to kidney problems. Additional research is needed to understand the long-term benefits of these treatments.

We are very excited to be engaged with the community on how best to offer disease identification tools and emerging therapies to our patients.

Daniel Jacoby, MD


Transthyretin amyloid cardiomyopathy (ATTR-CM) affects an estimated 100,000 people in the U.S. There are two subgroups of ATTR-CM: hereditary, which is caused by a genetic mutation in the transthyretin gene and the wild-type, which is associated with aging. Studies suggest that up to 15% of older adults with heart failure may have wild-type ATTR. Vyndaqel and Vyndamax were both approved in May 2019 by the FDA. Both drugs slow the formation of amyloid that causes the disease.

About the Inherited Cardiomyopathy Program

Daniel Jacoby, MD, associate professor of medicine in cardiology and director of the Cardiomyopathy Program and Comprehensive Heart Failure Program, established the Inherited Cardiomyopathy Program to treat patients with inherited cardiomyopathies and coestablished the Amyloid Clinic with Miller. “We are very excited to be engaged with the community on how best to offer disease identification tools and emerging therapies to our patients,” Jacoby says. “This is an exciting time.”

For more information please contact the Cardiomyopathy & Inherited Cardiovascular Disease Program at 203-785-7191 or visit the Yale Medicine website.

Submitted by Elisabeth Reitman on October 21, 2019