Aerial BioPharma, a biopharmaceutical company, has won FDA orphan drug designation for its narcolepsy drug in development. The issuance of designation from the FDA follows the completion of a Phase IIa study in which N05 demonstrated ...
Pluristem Therapeutics, a developer of placenta-based cell therapies, is seeking FDA orphan drug status for its PLacental eXpanded (PLX) cells for the treatment of aplastic anemia. Pluristem earlier received an orphan drug status from the ...
Clinical-stage biopharmaceutical company OXiGENE's acute myelogenous leukemia (AML) drug candidate, OXi4503, has received FDA orphan drug status. The second-generation anticancer agent with a dual-mechanism vascular disrupting feature ...
The US FDA has granted fast track status to Soligenix's SGX203 for the treatment of pediatric Crohn's disease. SGX203 contains an active corticosteroid, beclomethasone 17,21-dipropionate (BDP), that targets local inflamed tissue. ...
Athersys has received FDA orphan drug designation for its proprietary cell therapy, MultiStem, to treat Hurler's syndrome, also known as mucopolysaccharidosis type I or MPS-I. The orphan drug designation provides substantial potential ...
REGENX BioSciences has received FDA orphan drug designation for treatment of familial hypercholesterolemia (HoFH) using NAV rAAV8 vectors. The REGENX program consists of a NAV rAAV8 vector expressing a normal copy of the human ...
Tags: orphan drug designation, familial hypercholesterolemia, clinical study