The U.S. Food and Drug Administration (FDA) has approved a groundbreaking drug, acoramidis (marketed as Attruby), developed at Stanford Medicine to treat transthyretin amyloid cardiomyopathy (ATTR-CM).
This rare cardiovascular condition causes a buildup of a misfolded protein in the heart, leading to stiffness that prevents it from functioning properly. The drug provides new hope for patients facing this life-threatening disease.
Acoramidis was originally created by two former Stanford researchers: Dr. Isabella Graef, a former faculty member and now CEO of Shenandoah Therapeutics, and Dr. Mamoun Alhamadsheh, a chemist who is now a professor at the University of the Pacific.
Graef and Alhamadsheh collaborated during their time at Stanford, where the drug was initially developed and tested. Their early work on the molecule—then known as AG10—eventually led to its FDA approval on November 22.
“This is a rare and remarkable achievement,” said Dr. Graef. “Developing a drug in an academic setting and seeing it approved by the FDA without further changes in the industry is almost unheard of. It’s a testament to the power of rigorous science and collaboration.”
The Stanford Medicine SPARK program played a crucial role in the drug’s development. SPARK supports translating academic research into practical treatments, offering mentorship and resources to overcome the challenges of drug development. “
There are many barriers to turning academic discoveries into patient therapies,” said Dr. Daria Mochly-Rosen, founder of SPARK. “The process requires knowledge often not taught in academia and a great deal of persistence. Seeing acoramidis reach patients is incredibly rewarding.”
ATTR-CM occurs when a protein called transthyretin misfolds and aggregates in the heart, leading to life-threatening stiffness. Acoramidis is a transthyretin stabilizer, which prevents the protein from misfolding and forming harmful clumps.
While other stabilizer drugs exist, clinical trials showed acoramidis offers better outcomes, including improved survival rates and quality of life for patients.
“This drug is truly transformative,” said Dr. Graef. “It offers patients not just longer lives but also better lives, which is what we all hope for in medicine.”
The development of acoramidis involved contributions from Stanford clinical collaborators, including Dr. Ron Witteles and Dr. Michaela Liedtke, as well as researchers from Eidos Therapeutics and BridgeBio Pharma.
The successful approval highlights the importance of teamwork across academic and industry sectors in addressing rare diseases.
Acoramidis marks a significant step forward for patients with ATTR-CM, offering hope where options were previously limited.
Its development and approval underscore the potential of academic research to create life-changing therapies through persistence, collaboration, and innovation.
If you care about heart health, please read studies about how eating eggs can help reduce heart disease risk, and herbal supplements could harm your heart rhythm.
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