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News about "Orphan Drug Designation (ODD)"
FDA Grants Orphan Drug Status to BlueRock's OpCT-001
The ODD supports the clinical development of OpCT-001, the first iPSC-derived investigational cell therapy to enter human trials for Retinitis Pigmentosa (RP), a common inherited retinal disease marked by progressive loss of photoreceptor cells.
Orphan Drug Designation (ODD) | 23/01/2026 | By News Bureau | 120
FDA Grants Orphan Drug Designation to LP-284 of Lantern Pharma
The designation marks the third FDA orphan status for LP-284 and the sixth across Lantern Pharma’s AI-driven oncology pipeline, strengthening the programme’s development momentum in soft tissue sarcomas, a rare cancer segment with significant unmet need and a growing global market.
Orphan Drug Designation (ODD) | 21/01/2026 | By News Bureau
US FDA Grants Orphan Drug Designation to CK0804 Treg Therapy of Cellenkos
The US FDA has granted Orphan Drug Designation to Cellenkos’ CK0804, a first-in-class CXCR4hi Treg therapy for myelofibrosis. The investigational therapy is designed to home to the bone marrow and spleen, where it modulates inflammation through in-vivo expansion and IL-10 secretion.
Orphan Drug Designation (ODD) | 07/01/2026 | By News Bureau
Ouro Medicines Receives US FDA ODD for Gamgertamig (OM336) for Immune Thrombocytopenia
Ouro Medicines has secured a second ODD from the FDA for gamgertamig (OM336), this time for immune thrombocytopenia (ITP), following an earlier designation for autoimmune hemolytic anemia (AIHA). It comes as the company completes dosing in the first cohort of its global basket trial in autoimmune cytopenias.
Orphan Drug Designation (ODD) | 09/12/2025 | By Akanki | 162
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 | 125
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 | 179
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 | 216
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 | 127
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