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FDA Grants Cerecor’s Three Substrate Replacement Therapies Orphan Drug Designation firstwordpharma
January 17, 2019
Each indication is an ultra-rare CDG estimated to have fewer than 1,000 patients in the United States.
SLN124 granted Orphan Drug Designation by EMA for the treatment of β-Thalassemia firstwordpharma
January 16, 2019
Following an application by Silence last year, the COMP concluded that SLN124 will be of significant benefit to those affected by the chronic and potentially life-threatening condition, β-Thalassemia.
IntraBio Receives Niemann-Pick Disease European Orphan Drug Designation for IB4000 firstwordpharma
January 10, 2019
IntraBio Inc., a late-stage biopharmaceutical company developing novel therapies for rare ("orphan") and common neurodegenerative diseases, announced that the European Commission has granted Orphan Medicinal Drug Designation to IB4000 for the treatment of
Moleculin Announces FDA Filing for Orphan Drug Designation for Glioblastoma Drug americanpharmaceuticalreview
December 10, 2018
Moleculin Biotech, Inc., a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license ....
Catalyst's long-delayed Firdapse snags FDA nod fiercepharma
December 05, 2018
It took a breakthrough designation, orphan drug status, an embarrassing up-front rejection letter and some extra trials, but Catalyst Pharmaceuticals has finally crossed the finish line with Firdapse.
Vybion Receives Orphan Drug Designation for INT41 in Huntington's Disease firstwordpharma
December 04, 2018
Vybion Inc., a leading Gene Therapy Company developing Intrabodies to treat serious and rare diseases, today announced that the United States Food and Drug Administration (FDA) has granted orphan drug designation for INT41, the Company's Intrabody deliver
Scholar Rock Announces Positive Opinion by the European Medicines Agency on Orphan Drug Designation for SRK-015 for the Treatment of Spinal Muscular Atrophy firstwordpharma
December 04, 2018
SRK-015 was previously granted orphan drug designation by the U.S. Food and Drug Administration (FDA) in March 2018.
Pliant Therapeutics Receives FDA Orphan Drug Designation for Lead Program PLN-74809 in Primary Sclerosing Cholangitis pharmafocusasia
November 26, 2018
Pliant Therapeutics, Inc., a biotechnology company focused on discovering, developing and commercializing treatments for fibrotic diseases, today announced that ...
Santhera’s cystic fibrosis drug POL6014 in line for orphan status pharmatimes
October 19, 2018
Santhera says its experimental cystic fibrosis drug POL6014 is likely to be designated an orphan drug in the next 30 days, putting the firm in line to receive...
FDA grants orphan drug status to ASLAN’s treatment for AML biospectrumasia
August 21, 2018
ASLAN003 is an orally active, potent inhibitor of human dihydroorotate dehydrogenase (DHODH) that has the potential to be first-in-class in AML.

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