News about "Orphan Drug Designation (ODD)"

US FDA Approves First Therapy to Target Muscle Loss in Spinal Muscular Atrophy

The US Food and Drug Administration (FDA) has approved Isembyld, the first therapy for Spinal Muscular Atrophy (SMA) that directly targets muscle loss alongside existing treatments.

Orphan Drug Designation (ODD) | 14/09/2026 | By News Bureau |  107

Ultomiris Misses Phase 3 Endpoint in Post-Transplant TMA Trial

AstraZeneca shares a phase 3 update on Ultomiris for Haematopoietic Stem Cell Transplant (HSCT)-TMA, showing a trend toward clinical benefit in adults and adolescents as regulatory discussions continue.

Orphan Drug Designation (ODD) | 28/07/2026 | By News Bureau |  157

Japan Approves Sanofi's Wayrilz for Immune Thrombocytopenia

Sanofi’s Wayrilz has been approved in Japan for the treatment of immune thrombocytopenia (ITP). The BTK inhibitor works through multi-immune modulation to address the underlying causes of the disease.

Orphan Drug Designation (ODD) | 24/06/2026 | By News Bureau

Atossa Therapeutics Secures FDA Rare Pediatric Disease Tag for (Z)-Endoxifen in McCune-Albright Syndrome

Atossa Therapeutics receives US Food and Drug Administration (FDA) Rare Pediatric Disease designation for (Z)-Endoxifen in McCune-Albright syndrome, qualifying for Priority Review Voucher award upon approval.

Orphan Drug Designation (ODD) | 05/05/2026 | By News Bureau |  150

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 |  416

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 |  226

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 |  200

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 |  388

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 |  270

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 |  350