The orphan drug designation by the EC is issued for drugs which are intended to treat diseases that affect fewer than five in 10,000 people in the European Union
GC1130A has previously achieved notable milestones by securing both RPDD and ODD from the U.S. FDA in January 2023
LG Chem is making progress in the development of the world's first oral treatment for rare forms of obesity.
In Phase I studies, ZYIL1 was found to be safe and well-tolerated in human volunteers
Deal includes DTx-1252, a potential therapy for the neuromuscular disorder Charcot-Marie-Tooth disease type 1A (CMT1A), and two additional preclinical programs for other neuroscience indications
As Caeregen’s CDMO partner for CTR-107, Wacker Biotech will produce drug substance at its site in Halle, Germany, and complete clinical trial drug product production at its site in Amsterdam, the Netherlands
The primary endpoint is to measure the change in liver stiffness measurement performed by transient elastography from Baseline to Week 52
Both the companies intend to facilitate faster clinical trial enrollment and provide patients with access to novel medications
Extends patent protection for Microbion's proprietary inhaled pravibismane for the treatment of pulmonary infections to 2039
Receives Subject Expert Committee recommendation to bring new therapies of Pompe, ASMD to India
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