Related coverage linked through entity extraction aliases.
May 31, 2026
Positive Phase 3 VOXZOGO results in hypochondroplasia; FDA sNDA submission planned in Q3. Q4 2025 revenue rose 17% to $874.6M, beating estimates; EPS missed. Q1 2026 results due May 4.
Aug 07, 2026
BioMarin reported Q2 2026 revenues of $990 million, up 20% Y/Y, and raised guidance. It filed an sNDA for VOXZOGO in hypochondroplasia and advanced BMN 333, BMN 820, and BMN 351.
Apr 01, 2026
The FDA has cleared Abbisko Therapeutics' IND application for ABSK061, an oral FGFR2/3 inhibitor for achondroplasia, while BridgeBio reports positive Phase 3 results for its oral infigratinib in the same condition. Both companies are advancing oral therapies targeting FGFR3 overactivity in this genetic growth disorder.
Mar 10, 2026
The FDA approved navepegritide, a once-weekly injectable therapy for achondroplasia, while phase 3 data showed infigratinib, an investigational oral medication, achieved the highest growth velocity reported in randomized trials for the condition.
Feb 28, 2026
The FDA has granted accelerated approval for YUVIWEL (navepegritide), the first once-weekly treatment for children with achondroplasia. Commercial availability is expected in early Q2 2026, with a Rare Pediatric Disease Priority Review Voucher granted.
