Experimental Spinocerebellar Ataxia Treatment Earns Orphan Drug Designation

According to a story from Bloomberg, the company Cadent Therapeutics recently announced that the company’s experimental product candidate CAD-1883 has earned Orphan Drug Designation from the US Food and Drug Administration (FDA) as a treatment for the rare disease spinocerebellar ataxia. Cadent is dedicated to the development of therapies for disorders affecting 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 an incentive program that is typically reserved for therapies and treatments that are designed to treat rare diseases, which is defined as any disease that affects less than 200,000 people in the US. To qualify for this designation, a therapy must either satisfy a medical need that is not being met by current therapies or demonstrate superiority in safety or efficacy in comparison to current treatments. Orphan Drug Designation confers a number of benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a seven year period of market exclusivity if the drug is approved for use by the FDA.

About CAD-1883

There are currently no approved treatments for spinocerebellar ataxia. CAD-1883 is designed as a modulator of calcium activated potassium ion channels. This mechanism should give the drug the capability to regulate neuronal activity in spinocerebellar ataxia patients and curtail the degree of disability that they experience.


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