It can be extremely difficult to incentivize drug development for rare diseases or conditions. To combat this, decades ago, the FDA created the Orphan Drug Act. Now, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare conditions. These are those that affect fewer than 200,000 Americans. As a benefit, drug developers receive incentives such as fee waivers, tax credits, and seven years of market exclusivity upon FDA approval. An article in ALS News Today recently shared that ET-101, also known as SynCav1, was granted Orphan Drug designation for the treatment of amyotrophic lateral sclerosis (ALS). 

Developed by Eikonoklastes Therapeutics (“Eikonoklastes”), ET-101 is an experimental gene therapy. A separate ALS News Today article explains that:

SynCav1 increases the number of small bubbles containing neurotransmitters and nerve growth factors as well as the number of receptor proteins that respond to these molecules. This increase leads to healthier nerve cells and better communication between them.

Eikonoklastes hopes that ET-101 will be effective for both familial and sporadic ALS. It delivers a working copy of the Caveolin-1 gene to help promote nerve growth and communication. In mice models of ALS with SOD1 mutations, ET-101 helped improve motor function, delayed disease onset and progression, and extended overall survival. In the future, Eikonoklastes hopes to evaluate ET-101 within human studies.

Amyotrophic Lateral Sclerosis (ALS): An Overview

Amyotrophic lateral sclerosis is a progressive neurodegenerative disease which occurs in approximately 4 per every 100,000 people within the country. As described above, ET-101 is being designed for both sporadic and familial ALS. Sporadic ALS occurs without a known cause, while familial ALS is associated with specific genetic mutations. Regardless, ALS causes nerve cell degeneration, loss of voluntary movement and control, and eventually death. It is more prevalent in men than women. Symptoms and characteristics can include:

• Frequent tripping and falling
• Slowed or slurred speech
• Arm, leg, and hand weakness
• Difficulty performing small movements, holding good posture, or walking
• Depression and anxiety
• Difficulty swallowing
• Muscle cramps
• Fatigue
• Unintentional weight loss
• Constipation