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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
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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 ....
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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.
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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
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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.
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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 ...
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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...
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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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FDA Grants Orphan Drug Designation to Lurbinectedin
americanpharmaceuticalreview
August 06, 2018
PharmaMar announces that lurbinectedin has been granted orphan drug status by the FDA for the treatment of small cell lung cancer。
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FDA grants orphan drug designation to elamipretide for LHON
europeanpharmaceuticalreview
May 02, 2018
The FDA granted Orphan Drug Designation to elamipretide, for the treatment of Leber’s hereditary optic neuropathy…