Experimental Treatment for Spinocerebellar Ataxia Earns Orphan Drug Designation

According to a story from BioSpace, the drug developer Cadent Therapeutics recently announced that its investigational product candidate CAD-1883 has earned Orphan Drug Designation from the US Food and Drug Administration (FDA). This experimental therapy is in development as a treatment for spinocerebellar ataxia (SCA). Cadent is committed to the development of innovative treatments for disorders affecting movement and cognition.

About Spinocerebellar Ataxia

Spinocerebellar ataxia (SCA), also known as spinocerebellar atrophy, is a progressive, degenerative disease that affects the cerebellum, a portion of the brain that is critical for movement control and coordination. These heritable disorders can often be fatal. There are several different types of spinocerebellar ataxia which are linked to a variety of genetic mutations; many types are caused by repeats of the CAG gene. Symptoms of these disorders include ataxia (abnormal gait), seizures, poor hand coordination, speech difficulties, peripheral neuropathy, chorea, and cognitive impairments. Unfortunately there is no cure for spinocerebellar ataxia and treatment is mostly focused on relieving symptoms. Physical and occupational therapy can help patients maintain their mobility. Gene therapy could be a potential future option for treating spinocerebellar ataxia. There is a dire need for more effective treatments for this disorder. To learn more about spinocerebellar ataxia, click here.

About Orphan Drug Designation

Orphan Drug Designation is intended for therapies that are developed to treat diseases that are considered rare, which is defined as any disease that affects less than 200,000 people in the US. To qualify the drug must display advantages in safety or effectiveness over currently available treatments. Alternatively, the treatment must fulfill a currently unmet medical need. This designation confers several benefits for the recipient company, such as tax breaks, the waiving of certain fees, and a seven year period of market exclusivity if the drug is accepted by the FDA. 

Spinocerebellar ataxia is estimated to affect around 11,000 people in the US. This disease currently has no officially approved treatments. Hopefully the development of CAD-1883 will proceed successfully so that this underserved patient population can have access to a disease modifying therapy for the first time.


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