The FDA’s Fast Track designation is designed to speed up the journey from development to approval for therapies that tackle serious conditions and meet previously unfulfilled medical needs. To learn more about the advantages a drug must have over existing therapies to receive this designation, take a look at this helpful guide from the FDA. In a recent news release from biotechnology and biopharmaceutical company Transposon Therapeutics (“Transposon”), the company shared that its therapy TPN-101 received Fast Track designation for progressive supranuclear palsy (PSP).
Transposon explains that TPN-101 is:
the most potent known inhibitor of LINE-1 reverse transcriptase and has excellent systemic and brain bioavailability with once daily oral dosing. TPN-101, also known as censavudine, was well-tolerated in a 48-week study.
Learn more about LINE-1 elements.
So far, a Phase 2 trial of TPN-101 has shown that the therapy reduces interleukin-6 (IL-6) and neurofilament light chain (NfL) levels in progressive supranuclear palsy. Both neurofilament light chain and IL-6 are biomarkers for neuro-degeneration and -inflammation. When these levels drop, it shows an improvement in neuronal health. Over 48 weeks, trial participants saw symptoms stabilize.
If TPN-101 eventually made it to market, it would become the first targeted treatment option for PSP.
The Facts on Progressive Supranuclear Palsy (PSP)
Progressive supranuclear palsy is a rare neurodegenerative disorder characterized by impaired movement and mobility. Doctors do not know the exact caus of PSP. However, we do know that the condition results from gradual nerve cell damage in the brain stem. In rare cases, PSP may result from MAPT mutations. This condition usually manifests in people between 45-75 years old and is more common in males than females. PSP may be misdiagnosed as Parkinson’s disease as many symptoms overlap.
Symptoms associated with progressive supranuclear palsy may include:
- Abnormal speech and difficulty talking
- Problems with swallowing (dysphagia) and eating
- Loss of voluntary eye movement
- Worsening balance and walking impairment
- Tendency to fall backward
- Wide-eyed staring facial expression
- Depression, anxiety, irritability, or apathy
- Slowed movement
- Muscle stiffness
- Dizziness
- Sensitivity to bright light
- Memory and cognitive impairment
- Sleep disturbances
As stated above, there are no targeted therapies for PSP. Available treatments include antidepressants, levodopa, mobility aids, bifocals, physical and occupational therapy, and percutaneous gastronomy.
