FDA Lifts All Clinical Holds on Seladelpar

The U.S. Food and Drug Administration has lifted a clinical hold CymaBay Therapeutics’ seladelpar for three liver disease indications including two rare diseases.

Photo: Sujal Shah, CEO of CymaBay

Seladelpar is in development to treat Primary Biliary Cholangitis (PBC) and Primary Sclerosing Cholangitis (PSC), as well as Nonalcoholic Steatohepatitis (NASH).

PBC is a rare condition that causes the bile ducts in the liver to become inflamed, damaged, and destroyed. This causes bile, a fluid that helps in digestion, to build up in the liver. This build-up damages the liver over time, eventually causing it to lose its ability to function. PSC is a rare, chronic, and progressive liver disorder that frequently occurs in the setting of inflammatory bowel disease. It is characterized by inflammation, and eventually scarring of the liver and its bile ducts.

On November 25, 2019, CymaBay halted all clinical trials of seladelpar after atypical histologic findings with no clinical or laboratory correlates were identified at the planned end-of treatment biopsy review of a 52-week phase 2 NASH study. The FDA placed all active INDs for seladelpar on clinical hold.

Seladelpar is a potent, selective, orally active PPARδ agonist that is in development for the treatment of the liver diseases with the lead indication being PBC. For PBC, The FDA and the European Medicine Agency granted seladelpar an orphan designation. Seladelpar also received Breakthrough Therapy designation from the FDA and PRIority MEdicine status from the EMA for PBC.

“This is a pivotal event for seladelpar, which had garnered a high degree of patient interest based on its promising potential for anti-cholestatic, anti-inflammatory, and reduced symptom burden in patients with PBC,” said Sujal Shah, CEO of CymaBay. “It is our unwavering goal to one day make it available to patients with PBC, and potentially for other chronic, inflammatory liver diseases.”

Author: Rare Daily Staff