Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...
The FDA granted orphan drug designation to zavabresib, an investigational therapy for the treatment of myelofibrosis, a rare ...
Designation highlights significant unmet medical needAUSTIN, Texas, Jan. 20, 2026 (GLOBE NEWSWIRE) -- Rein Therapeutics ...
We knew we had something interesting with T3p, a single small RNA found in breast cancer but absent from normal tissue.
Pharmaceutical Technology on MSN
Opna Bio’s OPN-2853 secures FDA’s orphan drug status for myelofibrosis
The designation applies to investigational therapies that target rare diseases affecting less than 200,000 people in the US.
The FDA designates ARB1002 as an orphan drug, highlighting its potential as a novel treatment for pancreatic cancer amid limited options. The FDA has granted orphan drug designation (ODD) to ARB1002, ...
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