FDA Revokes Truseltiq's Accelerated Approval for Cholangiocarcinoma

The FDA has officially revoked the accelerated approval for BridgeBio Pharma's drug Truseltiq (infigratinib), which was initially granted in 2021 for patients with advanced cholangiocarcinoma featuring an FGFR2 fusion or rearrangement. The approval was initially conditional upon further post-marketing trials to confirm the clinical benefits observed in the preliminary study. BridgeBio's affiliate QED Therapeutics, the sponsor of the drug, encountered significant challenges in recruiting participants for these confirmatory studies. The trials aimed to assess Truseltiq's effectiveness in first-line cholangiocarcinoma treatment. Consequently, QED deemed it commercially unfeasible to continue the drug's distribution for its approved second-line indication.

BridgeBio had been in partnership with Helsinn, which held the US rights to Truseltiq for oncology and other indications, excluding skeletal dysplasias. This collaboration was valued at approximately $2.45 billion, including both upfront and milestone payments. However, the FDA's decision to rescind the approval did not come as a surprise, given that Helsinn had already opted to stop Truseltiq's distribution and seek its market withdrawal in late 2022 due to business considerations.

LianBio, which owns the rights to Truseltiq in China and other Asian markets, announced plans to terminate the Phase III PROOF-301 trial, which was investigating the drug as a first-line treatment for cholangiocarcinoma.

Despite the setback with Truseltiq, BridgeBio is continuing to explore the potential of the FGFR1-3 tyrosine kinase inhibitor for treating achondroplasia. The company recently granted exclusive rights to Kyowa Kirin in Japan for $100 million upfront, along with additional milestone payments.

Additionally, BridgeBio awaits an FDA decision on its TTR stabilizer, acoramidis, for transthyretin amyloid cardiomyopathy. This decision is expected by November 29. In parallel developments, Bayer has secured exclusive rights to commercialize acoramidis in Europe, contingent upon regulatory approval.

Furthering its oncology ambitions, BridgeBio has spun out a subsidiary, launching it earlier this month with a $200 million investment to support early-stage cancer programs centered on KRAS mutations. This strategic move underscores BridgeBio’s commitment to advancing innovative treatments despite the recent challenges faced with Truseltiq.