A BridgeBio Pharma drug developed for dwarfism elevated youngsters’s progress charges in a pivotal medical trial, assembly the examine’s important objective and boosting the day by day capsule’s possibilities 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 examine that enrolled 113 youngsters age 3 to 18 who’ve achondroplasia, the most typical type of dwarfism. Preliminary information reported Thursday present that day by day doses of the examine drug led to a mean 2.1 cm enhance in annualized peak velocity from baseline, measured at one 12 months and in comparison with placebo. Absolutely the change in peak was a mean 1.74 cm in comparison with placebo at one 12 months.
Achondroplasia also can have an effect on physique proportionality. BridgeBio reported infigratinib met a key secondary objective attaining statistically important enchancment in proportionality in comparison with placebo in youngsters youthful than 8 years previous.
Infigratinib was properly tolerated with no stories of significant adversarial occasions associated to the examine drug. and no discontinuations or dose reductions. There have been three circumstances of hyperphosphatemia, which is elevated ranges of phosphate within the blood. BridgeBio mentioned these circumstances had been thought of delicate 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 12 months.
Achondroplasia results in slowing of bone progress, significantly 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 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 develop into BioMarin’s top-selling product. Within the first 9 months of 2025, BioMarin reported $654 million in Voxzogo income, a 24% enhance in comparison with the identical interval within the prior 12 months. For the complete 12 months, the corporate projected the product would account for $900 million to $935 million in income.
Voxzogo requires day by day injections, which may be difficult for some sufferers, significantly youngsters. Ascendis Pharma goals to ease the dosing burden with its peptide drug, navepegritide, developed as a once-weekly injection. This drug is beneath FDA evaluation with a Feb. 28 goal date for a regulatory choice.
BridgeBio would have the ability to stand aside from the BioMarin and Ascendis medicine by bringing sufferers an oral possibility. The capsule’s preliminary information additionally counsel it might supply higher efficacy. Whereas these medicine 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 medicine confirmed of their respective pivotal research.
Leerink Companions analyst Joseph Schwartz mentioned in a analysis observe that the Ascendis drug additionally confirmed enchancment in physique proportionality, which key opinion leaders (KOLs) say might be a key level for choosing a remedy. He added that an oral drug generally is a drawback for younger youngsters who could also be unable to swallow tablets, leaving a spot out there 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/dad or mum desire for differing routes of administration,” Schwartz wrote.
Leerink expects regulatory approvals for infigratinib and initiatives the drug will attain $1.5 billion in peak gross sales.
BridgeBio is conducting further medical 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 contributors within the observational run-in interval to pick sufferers for a Section 3 medical trial on this indication.
Photograph by Flickr consumer Sean MacEntee through a Inventive Commons license
