According to an article from Financial Buzz, the pharmaceutical company CymaBay Therapeutics has announced that the first of its patients have begun to enroll in a long term seladelpar clinical trial. This extension study is intended to test the effectiveness of seladelpar in patients with primary biliary cholangitis. The study will provide long term seladelpar treatment for participants and will also assess the treatment’s tolerability over the long term.
Primary biliary cholangitis is a progressive, long term disease of the liver and gall bladder in which the bile ducts become heavily scarred and inflamed. In some individuals no symptoms are present, but others may experience problems such as enlargement of the liver and spleen, fatigue, severe and persistent itching, jaundice, and dark urine. The cause of the disease is not well understood. There are no real treatments for the condition, although the symptoms can be managed. Median survival rate is around twenty years, but a liver transplant can resolve the disease in some people, although others may still have symptoms after a transplant. To learn more about this disease, click here.
Seladelpar has already been designated with orphan drug status by the FDA and the European Medicine Agency, which will accelerate its development and give CymaBay exclusive rights and monetary incentives to continue work on the treatment. The treatment has also gotten PRIority MEdicine (PRIME) status from the European Medicine Agency.
In previous low dosage trials, seladelpar has demonstrated significant effectiveness in treating primary biliary cholangitis. Results from these trials were presented to the public this past October. The patients in those trials will continue into the long term extension trial as well per an agreement that the patients would have continuous long term access to the treatment if it performed decently during the first trial. The data for the long term trial will primarily be supplementary support for the seladelpar registration program and will follow the patients’ progress until the registration for the treatment is complete.
The new treatment is meeting a need that has no real precedent in the medical community. Hopefully seladelpar will continue to perform well in the long term trials.
