A drug from BridgeBio Pharma developed for dwarfism elevated progress charges in kids in a pivotal medical trial, assembly the examine’s major goal and boosting the day by day capsule’s probabilities of offering a substitute for persistent injections used to deal with the situation.

BridgeBio’s drug, infigratinib, was examined in a placebo-controlled Section 3 trial that enrolled 113 kids ages 3 to 18 with achondroplasia, the most typical type of dwarfism. Preliminary knowledge reported Thursday confirmed that day by day doses of the examine drug led to a median improve of two.1 cm in annualized peak velocity from baseline, measured at one yr and in contrast with placebo. Absolutely the change in peak after one yr was on common 1.74 cm in comparison with placebo.

Achondroplasia also can have an effect on the proportionality of the physique. BridgeBio reported that infigratinib achieved an necessary secondary goal, reaching a statistically important enchancment in proportionality in comparison with placebo in kids lower than 8 years previous.

Infigratinib was properly tolerated and there have been no stories of great antagonistic occasions associated to the examine drug. and no discontinuations or dose reductions. There have been three instances of hyperphosphatemia, which is an elevated stage of phosphate within the blood. BridgeBio mentioned these instances have been thought-about gentle and transient and none required dose discount or discontinuation of infigratinib. Based mostly on the trial outcomes, BridgeBio is now planning registrations for the drug within the US and Europe within the second half of this yr.

Achondroplasia results in slowing of bone progress, particularly within the lengthy bones of the legs and arms. It’s brought on by a genetic mutation that results in extreme exercise of fibroblast progress issue receptor 3 (FGFR3), a protein that regulates bone progress. Infigratinib is an oral small molecule designed to inhibit FGFR3.

BioMarin Pharmaceutical at present markets the one FDA-approved remedy for achondroplasia. This drug, Voxzogo, is an analogue of a peptide that compensates for FGFR3. Since its approval in 2021, the drug has turn into BioMarin’s best-selling product. Within the first 9 months of 2025, BioMarin reported $654 million in income from Voxzogo, a 24% improve in comparison with the identical interval final yr. For the total yr, the corporate anticipated the product to generate gross sales of $900 million to $935 million.

Voxzogo requires day by day injections, which will be difficult for some sufferers, particularly kids. Ascendis Pharma goals to ease the dosing burden with its peptide drug, navepegritide, developed as a weekly injection. This drug is underneath overview by the FDA with a goal date of February 28 for a regulatory choice.

BridgeBio might differentiate itself from the BioMarin and Ascendis medication by providing sufferers an oral possibility. The capsule’s preliminary knowledge additionally suggests it might supply higher efficacy. Though these medication weren’t examined head-to-head, the annualized progress fee achieved by BridgeBio’s drug in Section 3 testing exceeds the numbers proven by the BioMarin and Ascendis medication of their respective pivotal trials.

Leerink Companions analyst Joseph Schwartz mentioned in a analysis observe that the Ascendis drug additionally confirmed an enchancment in physique proportionality, which key opinion leaders (KOLs) mentioned may very well be an necessary level when choosing a remedy. He added that an oral drug may very well be a drawback for younger kids who could not be capable to swallow tablets, leaving a spot out there for injectables.

“Whereas the general image of those infigratinib knowledge is compelling, we nonetheless suppose it stays to be seen how the dynamics between oral and injectable therapies play out in achondroplasia, as we now have heard broadly various suggestions from our MEDACorp KOL discussions relating to affected person/doctor/dad or mum desire for various routes of administration,” Schwartz wrote.

Leerink is anticipating regulatory approvals for infigratinib and expects the drug to achieve peak gross sales of $1.5 billion.

BridgeBio is conducting extra medical trials for infigratinib. An ongoing examine in achondroplasia is evaluating the drug in infants and toddlers underneath 3 years of age. The corporate can also be growing this drug for a much less extreme type of dwarfism referred to as hypochondroplasia. BridgeBio is enrolling members for the observational run-in interval to pick out sufferers for a part 3 medical trial for this indication.

Picture by Flickr person Sean MacEntee by way of a Inventive Commons license