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The US FDA has granted avatrombopag (Doptelet) ODD as around 10 percent of US cancer patients per year experience chemotherapy-induced thrombocytopenia (CIT) with no available treatment.
The US Food and Drug Administration has awarded APR-TD011 Orphan Drug Designation, for the treatment of a rare disease.
The FDA and EMA have granted Orphan Drug Designation to zotiraciclib, currently the subject of two separate Phase Ib clinical trials in glioblastoma.
The US FDA has awarded pemigatinib Priority Review, after the drug met its primary and secondary endpoints in a Phase II trial.
Givlaari (givosiran) has been approved by the FDA for the treatment of adult patients with the rare genetic disorder, acute hepatic porphyria.
The FDA grants Orphan Drug Designation to treatments for rare diseases in R&D; here are the 10 most recent for oncologic conditions.
Orphan Drug Designation has been given to bispecific antibody candidate, GBR 1342, for the treatment of multiple myeloma.
Orphan Drug Designation has been granted to the CT053 anti-BCMA CAR-T programme for multiple myeloma treatment.
A treatment for Centronuclear Myopathies has received Orphan Drug Designation from the FDA and will begin Phase I/II trials.
The FDA has given the XWL-008 compound Orphan Drug Designation for the treatment of narcolepsy.
The FDA has given approval to AveXis for the first treatment of paediatric spinal muscular atrophy (SMA).
The FDA granted Orphan Drug Designation to elamipretide, for the treatment of Leber's hereditary optic neuropathy...
The FDA has approved Crysvita, the first drug approved to treat adults and children ages 1 year and older with x-linked hypophosphatemia, a rare, inherited form of rickets...