Experimental Drug for Cushing’s Syndrome Gets Orphan Drug Designation

According to a story from 4-traders.com, the pharmaceutical company SteroTherapeutics recently announced that its drug candidate ST-002 was awarded Orphan Drug Designation by the U.S. Food and Drug Administration (FDA). ST-002 is in development for the treatment of complications such as hyperglycemia, nonalcoholic steatosis, nonalcoholic fatty liver disease in Cushing’s syndrome.
Cushing’s syndrome consists of an array of signs and symptoms that occur as a result of prolong cortisol exposure. There can be multiple potential causes. It commonly develops as a result of taking medications such as glucocorticoids to treat other illnesses, such as inflammatory illness. However, it is also possible for changes within the body to affect its ability to secrete cortisol and cause Cushing’s syndrome. People with Cushing’s are at a greater risk of suffering from cardiovascular disease. Symptoms of the syndrome are distinctive, and include high blood pressure, weight gain,, reddish skin and stretch marks, a rounded, fat face, acne, weakening of muscles, increased body hair, and a lump of fat between the shoulders and around the neck. Women are more likely to experience Cushing’s syndrome. Sometimes, the brain is affected, which can cause mental health problems. To learn more about this syndrome, click here.

Generally, treatment for Cushing’s syndrome often involves simply tapering off of the medication that is responsible for the symptoms, but when another cause is implicated, the situation can become more complicated. This is where ST-002 comes in. The company is hoping that is treatment will become the new standard for patients whose symptoms cannot be easily resolved. The drug hopes to offer protection against liver damage and address symptoms such as weight gain.

Orphan Drug Designation is an FDA standard that helps incentivize drug companies to develop treatments for rare diseases and conditions. In order to achieve the designation, a drug must address a previously unmet medical need or demonstrate a considerable improvement in treatment outcomes. In addition, the designation is only given to treatments in development to treat rare diseases, which is defined as affecting 200,000 people or less in the U.S. The designation also waives certain fees that the company would be expected to pay and also gives the company a seven year period of market exclusivity if the drug is approved for public use.

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