BridgeBio is optimistic that infigratinib will have a second opportunity to receive approval following the drug achieving its target in a phase 3 trial of the medication in a form of dwarfism in children.
In 2021, the FDA approved infigratinib, later branded Truseltiq, as an accelerated approval for previously treated, locally advanced, or metastatic bile duct cancer, with an FGFR2 fusion/rearrangement. However, three years later, the regulator rescinded such approval at the request of the then-partner, Helsinn, of BridgeBio due to challenges in finding patients to participate in a mandatory confirmatory trial.
However, BridgeBio has not given up on infigratinib. The firm has been testing the FGFR1-3 selective tyrosine kinase inhibitor therapy in a world phase 3 in vivo trial of children with achondroplasia, the leading cause of dwarfism. This condition is brought about by an activating type of FGFR3.
The Propel 3 study, which was a one-year trial, reached its main study objective of showing a statistically significant positive mean of 2.1 cm/year change in annualized height velocity (AHV) between placebo and baseline, a release of Feb. 12. In the case of the secondary endpoint, the absolute height growth over the year, infigratinib had an improvement in the least squares (LS) mean of 5.96 cm/year with placebo at 4.22 cm/year.
Another drilling down that was done by the company was the change between the baseline of upper-to-lower proportionality of the body among a subpopulation of children that fall less than 8 years of age. To these patients, infigratinib was a first-therapy agent to demonstrate statistical significance over placebo in a randomized trial of achondroplasia, as cited by BridgeBio that identified a -LS mean change of 0.05 versus placebo.
No severe adverse events related to infigratinib were reported, and those three events of hyperphosphatemia, which are an electrolyte imbalance, were all mild and asymptomatic.
The analysis of Mizuho analysts called the data a best-case scenario. The body proportionality results singled out by them are the ones that left the possibility of premium pricing open.
The broad age bracket of the enrolled population and its orality make infigratinib a potentially superior agent of this indication (effectively, becoming the easiest, best-in-indication agent between physicians and patients throughout the indication), the analysts said on a Feb. 12 note.
The Infigratinib trial is the most recent, which comes after a phase 2 trial that reported a mean height growth increase of 2.51cm/year at 12 months. Based on a trial reporting 1.40-cm/year growth of AHV at 12 months, the FDA approved BioMarin’s achondroplasia therapy Voxzogo.
In an attempt to have infigratinib approved in achondroplasia, BridgeBio will submit the phase 3 data to regulators in the second half of the year. In conjunction with this approval move, the company is also putting on a separate phase 3 study on hypochondroplasia, a milder type of achondroplasia, in patients.
In the current release, BridgeBio Chief Medical Officer Daniela Rogoff, M.D., said that there is a great unmet demand for effective, viable, and less invasive therapeutic options in children living with achondroplasia.
The Propel 3 data, according to Rogoff, support the possibility of an oral medicine engaging FGFR3 directly in its overactivity in order to fulfill significant clinical needs, and the ability to fit into families who are interested in a non-injectable treatment. These outcomes are the beginning of significant improvements to the people who have been waiting to get a superior way, and we anticipate bettering this program to global submissions.
According to Ravi Savarirayan, the global principal investigator of the Propel 3 trial, infigratinib should be called the first oral therapy to target FGFR3 and treat the cause of achondroplasia.
Over the widest age range that has yet been studied, oral infigratinib has shown the largest and most pronounced increase in annualized growth velocity, and the first statistically significant increase in body proportionality in children with a 3-8-year age range, reported in any therapy approved or undergoing trials in this condition, Savarirayan added.
BridgeBio Pushes for Approval After Dwarfism Drug Boosts Children’s Height
BridgeBio is preparing to seek regulatory approval for its experimental dwarfism treatment after its Phase 3 clinical trial showed significant increases in height and improved growth outcomes in children. The positive results from the PROPEL 3 study mark a pivotal moment for BridgeBio as it works to bring an innovative oral therapy to market.
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