In the United States, Fast Track designation is granted to help expedite drug development and review for serious, rare, or life-threatening conditions. To receive Fast Track designation, the treatment must also have the potential to fill an unmet need for patients. According to a news release from September 15, 2021, SAGE-718, a potential treatment for patients with Huntington’s disease (HD), received this status from the FDA.
SAGE-718
To begin, what exactly is SAGE-718? Developed by biopharmaceutical company Sage Therapeutics, Inc., SAGE-718 is described as:
a novel, first-in-class, oxysterol-based positive allosteric modulator (PAM) of N-methyl-D-aspartate (NMDA) receptors.
In short, the treatment is being developed as a potential therapeutic for a variety of conditions. Beyond HD, researchers are evaluating SAGE-718 as a potential treatment for patients with Alzheimer’s disease and Parkinson’s disease. Altogether, the treatment works to improve cognitive function and prevent cognitive decline in patients with neurodegenerative conditions. By protecting cognitive function, Sage Therapeutics believes that the treatment can improve patient outcomes and help maintain a higher quality of life (QOL).
Moving forward, the company plans to evaluate the drug within a Phase 2 clinical trial.
Huntington’s Disease (HD)
HTT gene mutations cause Huntington’s disease (HD), a progressive neurological condition. Normally, HTT encodes for the production of the huntingtin protein, which may play a role in neuron health and function. When these mutations occur, a toxic huntingtin chain builds up, breaks down, attaches to neurons, and causes a loss of physical and cognitive function. An estimated 3-7 out of every 100,000 people have HD. In most cases, symptoms appear within one’s 30s or 40s, with a total lifespan of 15-20 years following symptom appearance. Symptoms include:
- Changes in mood, behavior, and personality which may seem to come on fairly quickly
- Impulsivity
- Cognitive decline
- Depression
- Forgetfulness
- Difficulty learning or retaining new information
- Chorea (uncontrolled jerking or twitching)
- Difficulty walking or swallowing
- Loss of, or changes in, coordination
