Potential New Treatment for Huntington’s Disease Granted Orphan Drug Designation

Huntington’s Disease is a neurodegenerative condition caused by a genetic mutation. It results in progressive decline of motor skills. It also affects cognitive function. Patients experience decline for 15-20 years after diagnosis and the disease is ultimately fatal.

However, researchers are hard at work to find a cure. A new drug has just been granted Orphan Drug Designation by the FDA. This title is given to some treatments as a way to help accelerate the development of new therapies for rare populations.

The scientific community knows there is an unmet need for this condition, and are making slow but steady progress to fill it.

The New Drug

The drug is called INT41. It’s an intrabody delivered using a adenoassociated virus (AAV). Intrabodies are small antibodies, special because, when delivered using certain means, they can function within cells. Some AAV’s are neurotropic, and have been used to treat various neurologic diseases. INT41’s success in animal trials shows not only that it may be an effective treatment for Huntington’s disease, but that it has the potential to benefit other neurodegnerative conditions as well.

The Wait

Unfortunately, there are still some studies underway for INT41 which must be completed before the drug is filed for approval. It’s predicted that the application for INT41 as a treatment for Huntington’s Disease will be submitted in 2020. If accepted, clinical trials for the drug will start soon after.

While research isn’t always a speedy process, the successful development of new treatments is always worth the wait. Keep your fingers crossed for continued positive results regarding INT41 and stay tuned to read updates on its progress.

In the meantime, you can read more about this development and the organization studying it (Vybion) here. INT41’s Orphan Drug Designation is definitely a hopeful step in Huntington’s Disease research.


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