Experimental Treatment for Spinocerebellar Ataxia Earns Orphan Drug Designation from the FDA

According to a story from Business Wire, the drug developer Cadent Therapeutics recently announced that its investigational drug CAD-1883 has been awarded Orphan Drug Designation from the US Food and Drug Administration (FDA). This designation is for the treatment of spinocerebellar ataxia, a rare, progressive genetic disorder. Cadent is focused on the development of therapies for disorders that affect movement and cognition.

About Spinocerebellar Ataxia (SCA)

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

CAB-1883 is designed as an innovative regulator of SK channels and is the first drug of this type. The primary goal of its development is the treatment of movement disorders such as essential tremor and spinocerebellar ataxia. About 11,000 people in the US are estimated to be affected by spinocerebellar ataxia.


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