You are here: Industry Insights > Orphan Drug

News List

Dynacure Receives Orphan Drug Designation for DYN101 americanpharmaceuticalreview
August 12, 2019
Dynacure announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2) for ...
FDA grants orphan drug designation to Stoke Therapeutics’ STK-001 pharmaceutical-technology
August 08, 2019
The US Food and Drug Administration (FDA) has granted orphan drug designation to Stoke Therapeutics’ antisense oligonucleotide, STK-001, a new therapy for severe and progressive genetic epilepsy Dravet syndrome.
APB-102 Receives Orphan Drug Designation for Genetic SOD1 ALS Treatment americanpharmaceuticalreview
July 23, 2019
Apic Bio announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to APB-102, a gene therapy soon to be in clinical development for the treatment of genetic SOD1 amyotrophic lateral sclerosis (ALS).
Aridis Receives Orphan Drug Designation from the EMA for AR-501 americanpharmaceuticalreview
July 22, 2019
ridis Pharmaceuticals announced the European Medicines Agency (EMA) has granted Orphan Drug Designation to AR-501, the Company's inhaled formulation of gallium citrate for treatment of lung infection in patients with cystic fibrosis.
FDA orphan drug designation granted for Autolus’ acute lymphoblastic leukemia drug pharmatimes
April 24, 2019
Autolus Theraputics has announced that the United States Food and Drug Administration (FDA) has granted orphan drug designation to its acute lymphoblastic leukemia (ALL) treatment.
US FDA grants saracatinib Orphan Drug Designation for idiopathic pulmonary fibrosis worldpharmanews
March 19, 2019
The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for saracatinib
Verdiperstat Receives Orphan Drug Designation from FDA for Multiple System Atrophy americanpharmaceuticalreview
February 20, 2019
Biohaven announced it received orphan drug designation from the U.S. Food and Drug Administration (FDA) for its product candidate verdiperstat (previously BHV-3241)....
US Senate invites seven pharma majors to testify on price hikes pharmaceutical-technology
February 19, 2019
A US Senate committee has written to seven major pharmaceutical companies, inviting them to testify at a hearing about increases to drug prices in the country....
FDA Grants Orphan Drug Designation for OCU400 for NR2E3 Mutation-Associated Retinal Degenerative Disease Treatment americanpharmaceuticalreview
February 15, 2019
Ocugen announced the U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD) for OCU400, Ocugen's novel gene therapy....
FDA Grants Orphan Drug Designation to Amplyx for APX001 for Cryptococcosis Treatment americanpharmaceuticalreview
February 13, 2019
Amplyx Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) Office of Orphan Product Development has granted orphan drug designation to APX001 ....

Previous page 1 2 3 4 5 6 7 8 Next page