Orphan drug market share has subsequently grown steadily ... in these outcomes between the product and the comparator. Issues with QoL data are linked to the second challenge in HTAs ...

Government incentives, advances in technology, and an army of patient advocates have spun a successful market—but abuses of the system and exorbitant prices could cause a backlash. Along with ...

She has an extremely wide experience of international pharmaceutical industry issues, having worked in ... the emerging pharmaceutical markets Orphan drugs launch challenges Cell and Gene ...

Scilex (SCLX) announced that FDA has approved the company’s request for the orphan drug designation for colchicine for the treatment of ...

Orphan drug designation comes with several benefits for the sponsoring company, including: ...

Gloperba® is the first and only liquid oral version of the anti-gout medicine colchicine indicated for the prophylaxis of painful gout flares in ...

Jin Ding launches a new project examining patients' access to orphan drugs (medicines for rare disease) in the UK - from patients’ own perspectives, as part of the Research England Participatory ...

HLX22 received orphan drug designation, offering incentives like tax credits and market exclusivity to accelerate development for gastric cancer treatment. Phase 2 trial results showed HLX22 improved ...

Sanofi SNY announced that the FDA has granted orphan drug designation to its investigational BTK inhibitor, rilzabrutinib, for the treatment of warm autoimmune hemolytic anemia (wAIHA) and ...

has granted orphan drug designation (ODD) to exidavnemab for the treatment of Multiple System Atrophy (MSA), providing incentives for the development of treatments for rare diseases with a high ...

Korro Bio, Inc. announced that its investigational drug KRRO-110 has received orphan drug designation from ... that can lead to serious lung and liver issues. KRRO-110 is being developed from ...