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News about "Orphan Drug Designation (ODD)"
USFDA Grants ODD to Zydus' Desidustat for Beta-Thalassemia Treatment
Desidustat, a Hypoxia-Inducible Factor Prolyl Hydroxylase Inhibitor (HIF-PHI), has received Orphan Drug Designation (ODD) from the USFDA. The designation grants eligibility for up to seven years of marketing exclusivity upon approval and highlights Desidustat’s potential to boost haemoglobin and red blood cell production.
Orphan Drug Designation (ODD) | 06/11/2025 | By Dineshwori
FDA Grants ODD to Leukogene Therapeutics' M2T-CD33 (LTI-214) for Treating Acute Myeloid Leukemia
The US FDA has granted ODD to Leukogene Therapeutics’ M2T-CD33 (LTI-214) for the treatment of Acute Myeloid Leukemia (AML). The designation highlights the pressing need for new treatment options and acknowledges LTI-214’s potential as a novel and targeted therapy for this aggressive form of blood cancer.
Orphan Drug Designation (ODD) | 05/11/2025 | By Dineshwori
Sanofi's Efdoralprin Alfa Meets Key Goals in Phase-II AATD Emphysema Study
Efdoralprin Alfa was previously granted fast track and Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of AATD Emphysema. It is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority.
Orphan Drug Designation (ODD) | 27/10/2025 | By Dineshwori
NMPA Clears Zhimeng Biopharma's Drug for Phase-II Epilepsy Trial
Zhimeng Biopharma’s drug for Phase II epilepsy trial will be evaluated in a randomized, double-blind, placebo-controlled multinational study to test its efficacy, safety, and tolerability as an adjunct therapy in adults with focal epilepsy.
Orphan Drug Designation (ODD) | 13/09/2025 | By Dineshwori
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