New Orphan Drug Designation for Potential Spinocerebellar Ataxia Treatment

Spinocerebellar Ataxia (SCA) refers to a group of neurodegenerative disorders. Ataxias are a component of the nervous system which control movement. SCA occurs when the ataxias in the brain or spinal cord are damaged. The type of SCA is determined by which gene has mutated to cause the condition. There are over 40 currently known types of the disease and it affects somewhere between one and nine of every 100,000 children born.

There is currently no cure for SCA, but researchers are working to develop new therapies for the condition.

IntraBio has just received Orphan Drug Designation for IB1000s, a potential new treatment for SCA. The company also believes that this therapy may be effective for a wide array of other neurodegenerative diseases, as well as lysosomal storage disorders.


IB1000s is a compound series. Researchers believe that it has the potential to reduce symptoms for SCA.

The European Commission has already granted the treatment Orphan Medicinal Drug Designation. The hope is that this series could improve the quality of life of SCA patients by reducing their most debilitating symptoms.

The National Ataxia Foundation (NAF) is supportive of IntraBio’s research. They explain that there is a dire need to improve treatment options for this population of patients.

This new designation will help to accelerate the development of this new potential therapy, shortening the timeline that it could actually be available for patients.

Looking forward

IntraBio is already working on applications for multinational clinical trials which will evaluate IB1000s in patients with Niemann-Pick disease Type C, inherited Cerebellar Ataxias, and GM2 Gangliosidosis.

In the future though, they hope to also investigate IB1000s as a treatment option for 17 other conditions including Amyotrophic Lateral Sclerosis, Parkinsonisms, Multiple Sclerosis, Lewy Body Dementia, and Restless Leg Syndrome.

The potential of this therapy has been confirmed by various compassionate-use studies. All together, IntraBio has examined the effects of the treatment in over 175 patients.

This is an exciting new development in SCA research. Stay tuned to hear more about IntraBio’s progress! In the meantime, you can read more about IBI000s and IntraBio’s research for Spinocerebellar Ataxia here.

Share this post

Share on facebook
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email