Experimental Treatment for Wilson’s Disease Earns Orphan Drug Designation

According to a story from Globe Newswire, the biopharmaceutical company Ultragenyx Pharmaceutical Inc. has recently announced that its investigational treatment UX701 has earned the Orphan Drug designation from the US Food and Drug Administration (FDA). This designation is for the treatment of Wilson’s disease, a rare disease. The company aims to file an investigational new drug application (IND) with the agency before year’s end. 

About Wilson’s Disease

Wilson’s disease is a genetic disorder in which the body is unable to process and excrete copper normally. Therefore, this metal accumulates in the body and causes a range of symptoms. It can ultimately be lethal without intervention. This disorder is caused by a genetic mutation affecting the ATP7B gene. Symptoms include nosebleeds, vomiting, weakness, tremors, fluid in the abdomen, muscle stiffness, limb swelling, difficulty talking, jaundice, anxiety, hallucinations, itching, and personality changes. It can lead to more serious complications such as liver failure or liver cancer, as well as kidney issues. Wilson’s disease is usually treated with dietary changes such as a low copper diet and avoiding copper cooking materials, as well as certain medications. Most medications can have serious side effects, however. A liver transplant can be curative but it is usually only done with patients who experience liver complications or who otherwise fail to respond to other approaches. To learn more about Wilson’s disease, click here.

About Orphan Drug Designation

Orphan Drug designation is generally reserved for therapies that are being developed to treat disease states that are considered rare. This is defined as any illness or condition that affects less than 200,000 people in the US. The drug must either have the potential to fulfill an unmet medical need or offer possible advantages in safety or efficacy when compared to current treatments. This designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a period of market exclusivity lasting seven years if the drug is approved by the FDA. 

About UX701

With limited treatment options that can carry serious side effects, there is a dire need for more effective treatments for Wilson’s disease. UX701 is a gene therapy that is aimed to stabilize expression of the copper transporter ATP7B following a single administration.

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