A BridgeBio Pharma drug developed for dwarfism elevated kids’s development charges in a pivotal scientific trial, assembly the examine’s primary objective and boosting the each day tablet’s possibilities to supply a substitute for continual injections used to deal with the situation.
The BridgeBio drug, infigratinib, was examined in a placebo-controlled Section 3 examine that enrolled 113 kids age 3 to 18 who’ve achondroplasia, the most typical type of dwarfism. Preliminary information reported Thursday present that each day doses of the examine drug led to a mean 2.1 cm improve in annualized peak velocity from baseline, measured at one yr and in comparison with placebo. Absolutely the change in peak was a mean 1.74 cm in comparison with placebo at one yr.
Achondroplasia can even have an effect on physique proportionality. BridgeBio reported infigratinib met a key secondary objective reaching statistically vital enchancment in proportionality in comparison with placebo in kids youthful than 8 years outdated.
Infigratinib was properly tolerated with no studies of significant opposed occasions associated to the examine drug. and no discontinuations or dose reductions. There have been three instances of hyperphosphatemia, which is elevated ranges of phosphate within the blood. BridgeBio stated these instances have been thought of gentle and transient, and none required dose discount or discontinuation of infigratinib. Primarily based 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 development, notably within the lengthy bones of the legs and arms. It’s attributable to a genetic mutation that results in extreme exercise of fibroblast development issue receptor 3 (FGFR3), a protein that downregulates bone development. Infigratinib is an oral small molecule designed to inhibit FGFR3.
BioMarin Pharmaceutical presently 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 grow to be BioMarin’s top-selling product. Within the first 9 months of 2025, BioMarin reported $654 million in Voxzogo incomea 24% improve in comparison with the identical interval within the prior yr. For the total yr, the corporate projected the product would account for $900 million to $935 million in income.
Voxzogo requires each day injections, which could be difficult for some sufferers, notably kids. Ascendis Pharma goals to ease the dosing burden with its peptide drug, navepegritide, developed as a once-weekly injection. This drug is beneath FDA evaluate with a Feb. 28 goal date for a regulatory choice.
BridgeBio would be capable of stand other than the BioMarin and Ascendis medication by bringing sufferers an oral choice. The tablet’s preliminary information additionally recommend it might supply higher efficacy. Whereas these medication weren’t examined face to face, the annualized peak 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 stated in a analysis be aware 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 kids who could also be unable to swallow drugs, leaving a spot available in the market for injectables.
“Whereas the general image of those infigratinib information are compelling we nonetheless assume it stays to be seen how the dynamic between oral and injectable therapies performs out in achondroplasia as now we have heard extremely various suggestions from our MEDACorp KOL discussions about affected person/doctor/father or mother 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 scientific trials for infigratinib. An ongoing achondroplasia examine is evaluating the drug in infants and toddlers youthful than age 3. The corporate can also be creating this drug for a much less extreme type of dwarfism known as hypochondroplasia. BridgeBio is enrolling individuals within the observational run-in interval to pick sufferers for a Section 3 scientific trial on this indication.
Picture by Flickr person Sean MacEntee by way of a Artistic Commons license
