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Results For "Orphan-Drug-Designation"

97 News Found

Vyome Holdings bolsters financial position to support VT-1953 pivotal study through 2027
Biotech | April 02, 2026

Vyome Holdings bolsters financial position to support VT-1953 pivotal study through 2027

As of December 31, 2025, the company reported total cash, cash equivalents and short-term investments of approximately $4.94 million


Merck strikes $6.7 billion deal to acquire terns, bolstering cancer pipeline
News | March 26, 2026

Merck strikes $6.7 billion deal to acquire terns, bolstering cancer pipeline

TERN-701, a next-generation therapy targeting chronic myeloid leukemia (CML), a form of blood cancer that still presents treatment challenges despite decades of advances


Shilpa Biologicals and mAbTree Biologics secure ODD from USFDA
Drug Approval | March 13, 2026

Shilpa Biologicals and mAbTree Biologics secure ODD from USFDA

Marks significant milestone for breakthrough biologic to treat rare blood cancers


HanchorBio scores first FDA orphan drug tag for gastric cancer therapy
Biotech | February 17, 2026

HanchorBio scores first FDA orphan drug tag for gastric cancer therapy

The designation for HCB101covers all forms of gastric cancer, including advanced gastric adenocarcinoma in both HER2-positive and HER2-negative patients


Zydus receives USFDA ODD for Desidustat for the treatment of sickle cell disease
Drug Approval | February 07, 2026

Zydus receives USFDA ODD for Desidustat for the treatment of sickle cell disease

This Orphan Drug Designation from the USFDA underlines the urgent medical need to develop a therapy for sickle cell disease


Shilpa Medicare & mAbTree Biologics win FDA nod for rare blood cancer therapy
Drug Approval | January 30, 2026

Shilpa Medicare & mAbTree Biologics win FDA nod for rare blood cancer therapy

The pioneering monoclonal antibody is for Essential Thrombocythemia (ET) and Polycythemia Vera (PV), rare chronic blood cancers with limited treatment options


AGC Biologics powers commercial launch of its life-changing gene therapy for rare childhood immune disorder
Biotech | January 20, 2026

AGC Biologics powers commercial launch of its life-changing gene therapy for rare childhood immune disorder

Waskyra is an ex vivo gene therapy that uses patients’ own CD34+ hematopoietic stem and progenitor cells, genetically engineered with a lentiviral vector


Takeda & Protagonist submit FDA application for groundbreaking polycythemia vera therapy
R&D | January 08, 2026

Takeda & Protagonist submit FDA application for groundbreaking polycythemia vera therapy

The investigational subcutaneous hepcidin mimetic peptide is designed to regulate iron and red blood cell production


Savara resubmits FDA application for potential treatment in rare lung disease patients
Biopharma | January 01, 2026

Savara resubmits FDA application for potential treatment in rare lung disease patients

The resubmission of BLA for MOLBREEVI names Fujifilm as the drug substance manufacturer