On Wednesday, the FDA granted accelerated approval for Gilead Sciences, Inc.'s (NASDAQ:GILD) Livdelzi (seladelpar) for primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have had an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.
The use of Livdelzi is not recommended for people who have or develop decompensated cirrhosis.
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The accelerated approval was based primarily on pivotal placebo-controlled Phase 3 RESPONSE study data.
In the study, 62% of participants taking Livdelzi achieved the primary endpoint of composite biochemical response at month 12, versus 20% of participants taking placebo.
Treatment with Livdelzi led to the normalization of alkaline phosphatase (ALP) values, a cholestatic marker that is a predictor of risk for liver transplant and death, in 25% of trial participants at month 12.
This change was not seen in any trial participants receiving a placebo.
Change from baseline pruritus (itch) score at month 6 was a key secondary endpoint; patients treated with Livdelzi demonstrated a statistically significant reduction in pruritus compared with placebo.
The FDA approved Livdelzi under accelerated approval based on a reduction of ALP.
Improvement in survival or prevention of liver decompensation events has not been demonstrated.
Continued approval of Livdelzi for the approved indication may be contingent on verification and description of clinical benefit in the confirmatory trial(s).
PBC is a rare, chronic, autoimmune disease of the bile ducts that affects approximately 130,000 Americans, primarily women, and can cause liver damage and possible liver failure if untreated.
Gilead added seladelpar via the acquisition of CymaBay Therapeutics Inc. for $32.50 per share in cash or a total equity value of $4.3 billion.
Seladelpar has also been accepted for review by the UK Medicines and Healthcare products Regulatory Agency (MHRA) and the European Medicines Agency (EMA).
Price Action: At last check Thursday, GILD stock was down 0.04% at $74.11 during the premarket session.
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