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Eikonoklastes Therapeutics Receives FDA Orphan Drug Designation for 1st-in-class Treatment of Amyotrophic Lateral Sclerosis (ALS)

Thursday, November 10, 2022

Eikonoklastes Therapeutics, a preclinical biotech company developing a 1st-in-class, AAV9-based gene therapy for treatment of sporadic and familial amyotrophic lateral sclerosis (ALS), announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for the ET-101 program.

ALS, also known as Lou Gehrig’s Disease, is a rare, progressive, debilitating, and universally fatal neurodegenerative disease affecting the motor neurons connecting the brain and spinal cord. Degeneration of these neurons leads to paralysis as the brain loses its ability to control muscle movement. Most people with ALS succumb to respiratory failure, usually within three to five years from when symptoms first appear. An estimated 10 percent of ALS is familial and caused by genetic mutations that are inherited. The 90%+ of sporadic ALS may be due to a combination of environment and genetic risk factors. There is currently no cure for either familial or sporadic ALS.

“The FDA’s Orphan Drug Designation for ET-101 reflects the compelling data underlying the ET-101 program and its potential as a 1st-in-class treatment option for both familial and sporadic ALS,” said Bruce Halpryn, chairman and CEO of Eikonoklastes Therapeutics. “Our goal is to significantly increase survival and improve quality of life for patients with this terrible disease.”

“The ODD program is critical for incentivizing the development of new treatments for rare diseases, especially those like ALS with severe morbidity and mortality,” said Samuel Lee, Eikonoklastes’ President and Chief Business Officer. “Achieving Orphan Drug Designation is an important regulatory milestone that further validates our efforts to efficiently develop ET-101. We look forward to rapidly advancing this novel technology towards our First-in-Human clinical trial.

ET-101 is licensed by Eikonoklastes and was pioneered within the lab of Brian Head, Ph.D. at the University of California-San Diego.

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