According to the company, the only available drug for this rare disease until now has been Shire's (LON:SHP) Elaprase (idursulfase).
The company further said it is seeking international partners to commercialise its medicine.
Fosun Pharma agrees XH-S004 overseas licensing deal
Itepekimab meets primary endpoint in one of two Phase 3 COPD trials
Inogen secures FDA clearance for SIMEOX 200 Airway Clearance Device
Infex Therapeutics begins Phase IIa trial for RESP-X in NCFB patients
Destiny Pharma launches research on XF-73 for cystic fibrosis MRSA infections
Polarean Imaging enters into trade-in agreement with University of Kansas Medical Center
Sionna Therapeutics doses first healthy subject in SION-109 phase one clinical trial
Arcturus Therapeutics granted FDA Orphan Drug Designation for ARCT-032 to treat cystic fibrosis
SpliSense completes SPL84 first-in-human, Phase one clinical trial for cystic fibrosis treatment
Positive Phase 2, Proof-of-Concept Results for VX-548 Published in New England Journal of Medicine
Inogen acquires Physio-Assist, expanding respiratory product portfolio and global presence