A BridgeBio Pharma drug developed for dwarfism elevated youngsters’s progress charges in a pivotal medical trial, assembly the research’s fundamental objective and boosting the every day capsule’s probabilities to supply a substitute for power injections used to deal with the situation.
The BridgeBio drug, infigratinib, was examined in a placebo-controlled Section 3 research that enrolled 113 youngsters age 3 to 18 who’ve achondroplasia, the most typical type of dwarfism. Preliminary information reported Thursday present that every day doses of the research drug led to a median 2.1 cm improve in annualized top velocity from baseline, measured at one yr and in comparison with placebo. Absolutely the change in top was a median 1.74 cm in comparison with placebo at one yr.
Achondroplasia may have an effect on physique proportionality. BridgeBio reported infigratinib met a key secondary objective reaching statistically important enchancment in proportionality in comparison with placebo in youngsters youthful than 8 years outdated.
Infigratinib was nicely tolerated with no experiences of significant antagonistic occasions associated to the research drug. and no discontinuations or dose reductions. There have been three instances of hyperphosphatemia, which is elevated ranges of phosphate within the blood. BridgeBio mentioned these instances had 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 regulatory submissions for the drug within the U.S. and Europe within the second half of this yr.
Achondroplasia results in slowing of bone progress, notably 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 downregulates bone progress. Infigratinib is an oral small molecule designed to inhibit FGFR3.
BioMarin Pharmaceutical at the moment markets the one FDA-approved remedy for achondroplasia. This drug, Voxzogo, is an analog of a peptide that counterbalances FGFR3. Since its approval in 2021, the drug has grown to develop into BioMarin’s top-selling product. Within the first 9 months of 2025, BioMarin reported $654 million in Voxzogo income, a 24% improve in comparison with the identical interval within the prior yr. For the complete yr, the corporate projected the product would account for $900 million to $935 million in income.
Voxzogo requires every day injections, which will be difficult for some sufferers, notably youngsters. Ascendis Pharma goals to ease the dosing burden with its peptide drug, navepegritide, developed as a once-weekly injection. This drug is underneath FDA assessment with a Feb. 28 goal date for a regulatory resolution.
BridgeBio would be capable to stand other than the BioMarin and Ascendis medication by bringing sufferers an oral possibility. The capsule’s preliminary information additionally counsel it may provide higher efficacy. Whereas these medication weren’t examined face to face, the annualized top velocity achieved by BridgeBio’s drug in Section 3 testing tops the marks that the BioMarin and Ascendis medication confirmed of their respective pivotal research.
Leerink Companions analyst Joseph Schwartz mentioned in a analysis notice that the Ascendis drug additionally confirmed enchancment in physique proportionality, which key opinion leaders (KOLs) say could possibly be a key level for choosing a remedy. He added that an oral drug could be a drawback for younger youngsters who could also be unable to swallow capsules, leaving a spot available in the market for injectables.
“Whereas the general image of those infigratinib information are compelling we nonetheless suppose it stays to be seen how the dynamic between oral and injectable therapies performs out in achondroplasia as we’ve got heard extremely various suggestions from our MEDACorp KOL discussions about affected person/doctor/mum or dad choice for differing routes of administration,” Schwartz wrote.
Leerink expects regulatory approvals for infigratinib and tasks the drug will attain $1.5 billion in peak gross sales.
BridgeBio is conducting extra medical trials for infigratinib. An ongoing achondroplasia research is evaluating the drug in infants and toddlers youthful than age 3. The corporate can be creating this drug for a much less extreme type of dwarfism referred to as hypochondroplasia. BridgeBio is enrolling contributors within the observational run-in interval to pick out sufferers for a Section 3 medical trial on this indication.
Photograph by Flickr person Sean MacEntee by way of a Artistic Commons license
