South SFV Today

The U.S. Food and Drug Administration has given its approval to a new drug developed at Stanford Medicine for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM), a rare cardiovascular disease. The drug, known as acoramidis or Attruby, was initially created by Isabella Graef, MD, and Mamoun Alhamadsheh, PhD. Graef is currently the CEO of Shenandoah Therapeutics and an affiliate at Stanford Medicine SPARK program, while Alhamadsheh is now a professor at the University of the Pacific.

Acoramidis was formerly referred to as AG10 and received FDA approval on November 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," said Graef. She added that "the FDA approval of Attruby is a testament to the transformative power of rigorous science and team effort within university laboratories."

The development process was supported by Stanford Medicine's SPARK program, which aims to translate basic discoveries into therapies. Daria Mochly-Rosen, PhD, founder and co-director of SPARK, highlighted the challenges faced in translating academic discoveries into patient-benefiting drugs. "There are many barriers in translating early academic discoveries to drugs that benefit patients," she stated.

ATTR-CM involves a buildup of misfolded transthyretin protein in the heart, leading to stiffness that impairs proper heart function. Acoramidis acts as a transthyretin stabilizer to prevent this misfolding and aggregation. Clinical trials showed improved efficacy over existing treatments with better survival rates and quality of life for patients.

"This drug represents a beacon of hope for patients with ATTR-CM," Graef expressed gratitude for contributing to impactful therapy.

Researchers from Eidos Therapeutics and BridgeBio Pharma, Inc., also played roles in the clinical development phase.

For more details on this development process, interested parties can visit the SPARK website.