ABBISKO-B (02256.HK): Lavengratinib wins EMA orphan status for ACH
- Lavengratinib (ABSK061), developed by subsidiary Abbisko Therapeutics, has been granted Orphan Designation by the EMA for achondroplasia; the Company said it cannot guarantee the product will ultimately be marketed.
- The drug had already received Orphan Drug Designation and Rare Pediatric Disease Designation from the US FDA for ACH, with its IND cleared; it is the first FGFR2/3 selective inhibitor globally to enter clinical trials.
- A Phase II, multicentre, open-label, dose-escalation study (ABSK061-202) is ongoing in children aged 3 to 12, with preliminary data showing positive growth signals and favourable safety and tolerability.
NewTimeSpace News: On 21 October 2026, Abbisko Cayman Limited (stock code: 02256) announced that its subsidiary Abbisko Therapeutics Co., Ltd. said the proprietary selective orally available small-molecule FGFR2/3 inhibitor lavengratinib (ABSK061) has been granted Orphan Designation (OD) by the European Medicines Agency (EMA) for the treatment of achondroplasia (ACH). The Company said it cannot guarantee that ABSK061 will ultimately be successfully marketed.
ACH is a rare, potentially life-threatening and debilitating growth disorder with limited treatment options currently available worldwide. Lavengratinib for ACH previously received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the US FDA, and its IND application has been cleared by the FDA. It is the first FGFR2/3 selective inhibitor in the world to enter clinical trials.
ACH is an autosomal dominant disorder primarily caused by mutations in the FGFR3 gene. Using Abbisko Therapeutics' oral mini-tablet technology, each tablet is less than 3 mm in diameter and can be conveniently taken with food or drink. The company is advancing a Phase II, multicentre, open-label, dose-escalation study (ABSK061-202) in children aged 3 to 12 with ACH; recent preliminary data showed positive growth-promotion signals with favourable overall safety and tolerability.
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