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Navigating the Landscape of Rare Diseases: Current Trends and Future Perspectives Neeta Ratanghayra
September 29, 2024
Rising Investment and Market Dynamics, Future Perspectives in the Orphan Drug Market and The Scenario in China.
Eleva Receives Orphan Drug Designation in Europe for Proprietary Factor H Therapy in C3 Glomerulopathy (C3G) Biotech Newswire
July 04, 2024
Eleva announced that the European Commission has granted the Orphan Drug Designation for Factor H (CPV-104), the company’s recombinant version of human complement Factor H, to treat C3 Glomerulopathy (C3G).
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.
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.
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
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.
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).
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.
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.
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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