Gilead Receives Health Canada Approval for PBC Treatment
- october 21, 2024 - Gilead Sciences announced today that LYVDELZI (mirikizumab-mrkz) has received conditional approval from the U.S.
- Primary Biliary Cholangitis (PBC) is a chronic, autoimmune liver disease characterized by the progressive destruction of the small bile ducts in the liver.
- The conditional approval of LYVDELZI is based on data from the pivotal Phase 3,randomized,placebo-controlled RESPONSE study.
“`html
LYVDELZI Receives Conditional Approval for Primary Biliary Cholangitis
Table of Contents
october 21, 2024 – Gilead Sciences announced today that LYVDELZI (mirikizumab-mrkz) has received conditional approval from the U.S. Food and Drug Administration (FDA) for the treatment of primary biliary cholangitis (PBC). This marks a significant advancement in the treatment of this chronic liver disease, offering a new option for patients who often experience debilitating symptoms and risk disease progression.
Understanding Primary Biliary Cholangitis (PBC)
Primary Biliary Cholangitis (PBC) is a chronic, autoimmune liver disease characterized by the progressive destruction of the small bile ducts in the liver. This leads to bile buildup, liver damage, and eventually, liver failure. Common symptoms include fatigue, itching (pruritus), and jaundice. PBC affects approximately 300,000 people in the United States, with women disproportionately affected, often diagnosed between the ages of 30 and 60 National Institute of Diabetes and Digestive and Kidney Diseases.
LYVDELZI: Clinical Trial Results
The conditional approval of LYVDELZI is based on data from the pivotal Phase 3,randomized,placebo-controlled RESPONSE study. in this study, 62% of participants treated with LYVDELZI achieved the primary endpoint of composite biochemical response at 12 months, compared to 20% in the placebo group. this composite endpoint included improvements in alkaline phosphatase (ALP), bilirubin, and a decrease in ALP from baseline.
Specifically:
- Biochemical Response: defined as ALP less than 1.67 times the upper limit of normal (ULN), a decrease in ALP of at least 15% from baseline, and total bilirubin less than or equal to the ULN.
- ALP Normalization: 25% of patients treated with LYVDELZI achieved normalization of alkaline phosphatase (ALP ≤ ULN) at 12 months, compared to none in the placebo group. ALP normalization is a key indicator of cholestasis improvement and is associated with reduced risk of liver transplantation and death.
- Pruritus Reduction: LYVDELZI demonstrated a statistically significant and clinically relevant reduction in pruritus (itching) at 6 months in patients who were already experiencing clinically significant itching before treatment initiation.
LYVDELZI is the only drug to demonstrate statistically significant and sustained improvements in both pruritus and cholestasis markers related to the risk of disease progression in a phase 3 trial. These findings suggest LYVDELZI addresses critical aspects of PBC management beyond simply slowing disease progression.
safety and Side Effects
The most common adverse reactions reported in the RESPONSE study (occurring in ≥5% of participants treated with LYVDELZI and more frequently than in the placebo group) were:
- Headache
- Abdominal pain
- Nausea
- Abdominal distention (swelling)
- Dizziness