You are here: Industry Insights > orphan

News List

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.
U.S. FDA Grants Orphan Drug Designation to BCMA CAR-T Cell Therapy Co-Developed by IASO Bio and Innovent prnasia
February 14, 2022
IASO Biotherapeutics ("IASO Bio"), a clinical-stage biopharmaceutical company engaged in discovering, developing, and manufacturing innovative cell therapies and antibody products, and Innovent Biologics, Inc.
IASO, Innovent CART-cell Therapy Gets Orphan Designation contractpharma
February 14, 2022
anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T-cell therapy for the treatment of relapsed and/or refractory multiple myeloma.
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.
CTB001, Bioheng auto CAR - T Product, received Orphan Drug Designation (ODD) from the U.S. FDA prnasia
January 29, 2022
Recently, Nanjing Bioheng Biotech Co., Ltd. ( hereinafter referred to as Bioheng) announced that its anti-claudin 18.2 autologous CAR-T cell therapy product CTB001 received Orphan Drug Designation (ODD) from the Food...
Neurophth Therapeutics' Treatment of Leber's Hereditary Optic Neuropathy Gene Therapy NR082 was Granted Orphan Drug Designation by EMA prnasia
January 28, 2022
Neurophth Therapeutics, Inc., (hereinafter referred to as "Neurophth"), today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) granted the orphan drug designation (ODD)...
Senhwa's Silmitasertib Receives US FDA Orphan Drug Designation for the Treatment of Biliary Tract Cancer prnasia
January 21, 2022
Senhwa Biosciences, Inc. (TPEx: 6492), a drug development company focusing on first-in-class therapeutics for oncology, rare diseases, and novel coronaviruses, announced that the US Food and Drug Administration (FDA) ...
Zydus receives Orphan Drug Designation from US FDA for malaria treatment expresspharma
December 17, 2021
The Orphan Drug Designation provides up to seven years of marketing exclusivity in the US.
FDA Grants Longeveron Orphan Drug Designation for Lomecel-B in Infants with Hypoplastic Left Heart Syndrome AmericanPharmaceuticalReview
December 07, 2021
Longeveron Inc., a clinical-stage biotechnology company developing cellular therapies for chronic aging-related and certain life-threatening conditions, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD)