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Rare Disease Drug 101 - What are the Benefits to Apply for and Receive Orphan Drug Designation?
Lin Zhang
June 29, 2021
There are many common diseases that affect millions of people across the world. On the other hand, there are so-called rare conditions that affect a small number of people.
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FDA grants orphan drug status to Syros’ myelodysplastic syndrome therapy
Pharmaceutical-Technology
February 07, 2022
Tamibarotene is currently being analysed along with azacitidine in the Phase III SELECT-MDS-1 trial.
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Paratek Pharmaceuticals Announces FDA Orphan Drug Designation for NUZYRA (omadacycline) for the Treatment of Infections Caused by Nontuberculous Mycobacteria
firstwordpharma
August 19, 2021
Paratek Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for NUZYRA® (omadacycline) for the treatment of infections
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Stealth BioTherapeutics Receives Orphan Drug Designation from the European Medicines Agency for Elamipretide for the Treatment of Barth Syndrome
firstwordpharma
June 02, 2021
MITO today announced that the European Medicines Agency (EMA) has granted orphan drug designation (ODD) for elamipretide for the treatment of Barth syndrome, an ultra-rare genetic condition.
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Shylicine Granted Orphan Designation
americanpharmaceuticalreview
October 29, 2020
Vanessa Research (VR) has been granted positive orphan drug opinion by the European Medicines Agency (EMA) for their drug Shylicine™ (patent pending).
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Novartis’ branaplam gets orphan drug designation for Huntington’s disease
pharmaceutical-technology
October 22, 2020
The US Food and Drug Administration (FDA) has granted orphan drug designation to Novartis’ branaplam (LMI070) to treat Huntington’s disease.
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Spero Receives Orphan Drug Designation for Nontuberculous Mycobacterial Treatment
americanpharmaceuticalreview
March 12, 2020
Spero Therapeutics announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for oral SPR720 for the treatment of nontuberculous mycobacterial (NTM) infection.
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Pharma And Health From Ingredients To Pills: Recordati Acquired Novartis’s Isturisa and FDA Approval
Lin Zhang
March 12, 2020
On March 06, 2020, the U.S. Food and Drug Administration has announced the approval of new medication Isturisa (osilodrostat), the first and only cortisol synthesis inhibitor and a new non-surgical option for the treatment of patients with Cushing’s Disea
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F2G Receives Orphan Drug Designation for Olorofim
americanpharmaceuticalreview
March 10, 2020
F2G announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead first-in-class candidate, olorofim (formerly F901318) for treatment of invasive aspergillosis ...
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FDA Grants Orphan Drug Designation for Progressive SupraNuclear Palsy Treatment
americanpharmaceuticalreview
March 09, 2020
Retrotope announced the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development granted orphan drug designation for its chemically-modified polyunsaturated fatty acid drug (RT001) ...