The National Institute for Health and Care Excellence (NICE) has announced its approval for seladelpar, a new medication for adults living with primary biliary cholangitis (PBC). This decision marks a significant development for patients in England who have not responded sufficiently to, or cannot tolerate, existing first-line treatments like ursodeoxycholic acid (UDCA).
PBC is a chronic liver disease that causes progressive destruction of the small bile ducts within the liver, potentially leading to cirrhosis and liver failure. It predominantly affects women, often between the ages of 40 and 60, with symptoms that can include debilitating fatigue, severe itching (pruritus), and dry eyes and mouth. According to the British Liver Trust, PBC affects approximately 1 in 1,000 women over 40 in the UK.
Seladelpar operates by activating the peroxisome proliferator-activated receptor delta (PPARdelta), a protein involved in regulating bile acid production and inflammation in the liver. Clinical trials have shown its effectiveness in reducing levels of alkaline phosphatase (ALP), a key biomarker of liver damage in PBC, and improving other liver function tests. This mechanism offers a different approach compared to UDCA, which primarily helps bile flow more easily.
The NICE recommendation specifies that seladelpar should be offered to adults with PBC who have an inadequate response to UDCA (defined as an ALP level of 1.67 times the upper limit of normal or greater, and/or total bilirubin greater than the upper limit of normal, after at least 12 months of UDCA treatment at an optimised dose) or who are unable to tolerate UDCA. This targeted use ensures the drug reaches those most in need of alternative treatment options.
This approval is expected to benefit an estimated 2,000 to 3,000 patients in England who currently lack effective treatment alternatives. The introduction of seladelpar could lead to improved liver health outcomes, reduced symptoms, and a better quality of life for this patient group, potentially delaying the progression of the disease and the need for liver transplantation.
While this is positive news, patients are reminded that any changes to their treatment plan should be discussed with their healthcare professional. For more information on PBC and available treatments, individuals should consult their GP or call NHS 111.
Source: Medscape