Company Earns Orphan Drug Designation for Autoimmune Hepatitis From the FDA

According to a story from, the Swedish drug company Calliditas Therapeutics has recently announced that the US Food and Drug Administration (FDA) has given one of the company’s experimental drugs Orphan Drug designation as a treatment for autoimmune hepatitis, a rare liver disease. The name of the specific product that the company is working on was not mentioned in the story. Calliditas is committed to the development of therapies for currently unmet medical needs and rare diseases.

About Autoimmune Hepatitis

Autoimmune hepatitis is a rare disease of the liver in which the body’s own immune system begins to mistakenly attack cells of the liver, triggering an inflammatory response. What causes this process to begin is unclear, but liver cells present with MHC class II receptors in the disease, which could be the result of a genetic abnormality or prior liver infection. These receptors cause the response from the immune system. Symptoms of autoimmune hepatitis include abdominal pain, fatigue, jaundice, joint pain, liver failure, weight loss, fever, and nausea. The treatment of the disease typically involves the use of immunosuppressants, such as corticosteroids or chemotherapy agents. Most patients can eventually achieve remission from their symptoms, but they almost always relapse. It is most common among people of middle age (40-50) with about 50,000 cases in the US. This disease can negatively affect life expectancy. To learn more about autoimmune hepatitis, click here.

About Orphan Drug Designation

Orphan Drug designation is reserved for therapies that are intended to treat diseases that are considered rare, which is defined as any condition that affects less than 200,000 people in the US. To qualify, and experimental therapy must display potential advantages in effectiveness or safety in comparison to currently available treatments. Alternatively, it must satisfy a medical need that is not being met by current treatments. This designation carries several benefits, such as the waiving of certain fees, tax breaks, and a seven year period of market exclusivity if the drug is approved for public use.

There is a clear need for more effective therapies for autoimmune hepatitis that lack the harmful side effects of steroids and can bring about a more durable period of remission that reduces the possibility of relapse. Hopefully the development of Calliditas’ product will continue to progress.


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