Oral achondroplasia therapy receives FDA breakthrough therapy designation
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Key takeaways:
- Infigratinib increased annualized height velocity for children with achondroplasia in a phase 2 trial.
- BridgeBio Pharma is currently enrolling children with achondroplasia for a phase 3 trial.
An oral medication to treat children with achondroplasia has received breakthrough therapy designation from the FDA, BridgeBio Pharma announced.
The FDA has granted breakthrough therapy designation to infigratinib (BridgeBio Pharma) as a therapeutic option to treat achondroplasia. The designation was granted based on data from the PROPEL 2 phase 2 study. In the trial, children receiving infigratinib had a significant increase in annualized height velocity of 2.51 cm per year at 12 months that was sustained at 18 months at 2.5 cm per year (P = .0015). Body proportionality also improved from baseline to 18 months with infigratinib (P = .001).
The breakthrough therapy designation is the latest FDA designation granted to infigratinib. The medication previously receiving an orphan drug designation, fast track designation and rare pediatric disease designation from the FDA for the treatment of achondroplasia. According to BridgeBio Pharma, the medication would be the first-in-class oral therapy to treat children with achondroplasia if it is approved by the FDA.
“The receipt of breakthrough therapy designation, the first ever for a treatment being developed for children with achondroplasia, marks another important milestone for our skeletal dysplasia program,” Adora Ndu, PharmD, JD, chief regulatory affairs officer at BridgeBio Pharma, said in the press release. “This recognition by the FDA further confirms the strength of our phase 2 data, and the substantial improvement on clinically significant endpoints over available therapies. We are excited by what this means for the community, and we look forward to working closely with FDA to expedite the development of infigratinib and access for families to an oral option.”
BridgeBio Pharma announced it is currently enrolling participants for a phase 3 study to assess infigratinib in achondroplasia. The company announced it plans to explore the use of the drug on a range of medical and functional impacts of achondroplasia, hypochondroplasia and other skeletal dysplasias.
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