by Hanae Armitage, Stanford University

Credit: Pixabay/CC0 Public Domain

The U.S. Food and Drug Administration has approved a drug developed at Stanford Medicine that offers hope to people diagnosed with a rare cardiovascular disease, transthyretin amyloid cardiomyopathy, or ATTR-CM.

The drug, acoramidis (Attruby), was originally developed by Isabella Graef, MD, former faculty member at the Stanford School of Medicine who is now the CEO of Shenandoah Therapeutics and an affiliate at the Stanford Medicine SPARK program; and Mamoun Alhamadsheh, Ph.D., a former chemist at Stanford University who is now a professor at the University of the Pacific.

Ron Witteles, MD, professor of medicine, and Michaela Liedtke, MD, associate professor of medicine, also played key roles as clinical collaborators during the early research on the drug.

Previously known as the Alhamadsheh-Graef molecule 10 (AG10), which was designed and synthesized by Alhamadsheh, the drug received FDA approval for the treatment of ATTR-CM Nov. 22.

"It is an exceedingly rare accomplishment to develop a drug in academia that was approved by the FDA without any further optimization in industry," Graef said. "The FDA approval of Attruby is a testament to the transformative power of rigorous science and team effort within university laboratories."

The development of acoramidis was supported by Stanford Medicine's SPARK program, which provides a cost-effective model to advance basic discoveries and translate them into therapies.

"There are many barriers in translating early academic discoveries to drugs that benefit patients," said Daria Mochly-Rosen, Ph.D., founder and co-director of SPARK, professor of chemical and systems biology, and the George D. Smith Professor in Translational Medicine.

"It requires knowhow, often not taught in academia, and much tenacity, as it takes a long time. It is so rewarding to learn that SPARK at Stanford had a role in making Attruby happen, and we are grateful for the invaluable mentoring by many industry expert volunteers and all the other team members and SPARKees who contributed."

Offering new hope to patients

People who are diagnosed with ATTR-CM experience a buildup of a misfolded protein called transthyretin in their hearts, causing stiffness that prevents the heart from beating properly. The new drug is known as a transthyretin stabilizer, which stops the protein from misfolding and aggregating in the heart.

While acoramidis isn't the first transthyretin stabilizer drug, clinical trials demonstrated an improved efficacy compared with those already available: Patients experienced better survival rates and quality of life.

"This drug represents a beacon of hope for patients with ATTR-CM," Graef said. "We are deeply grateful that we have been able to contribute to a therapy that truly makes a difference in patients' lives."

Researchers from Eidos Therapeutics and BridgeBio Pharma, Inc. contributed to the clinical development of the drug.

Provided by Stanford University