Latest news and insights on the pharmaceutical industry | Indian Pharma Post | Page 3

Results For "Orphan-Drug-Designation"

91 News Found

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


Diamyd Medical accelerates Phase 3 Type 1 diabetes trial following FDA guidance
Clinical Trials | December 31, 2025

Diamyd Medical accelerates Phase 3 Type 1 diabetes trial following FDA guidance

The interim efficacy readout, involving around 170 participants with 15-month data, remains on track for the end of March 2026


Chugai’s Tecentriq gets green light in Japan for hard-to-treat Thymic Carcinoma
Drug Approval | December 27, 2025

Chugai’s Tecentriq gets green light in Japan for hard-to-treat Thymic Carcinoma

The drug had previously received orphan drug designation for this rare cancer on March 31, 2025


EU nod to Wayrilz as breakthrough treatment for hard-to-treat ITP
Drug Approval | December 27, 2025

EU nod to Wayrilz as breakthrough treatment for hard-to-treat ITP

Wayrilz represents a new approach to ITP by targeting the disease at its root through multi-immune modulation


EMA grants orphan drug status to Sanofi’s Efdoralprin Alfa for rare lung disease
Drug Approval | December 20, 2025

EMA grants orphan drug status to Sanofi’s Efdoralprin Alfa for rare lung disease

Efdoralprin alfa, a recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, showed superior efficacy to standard plasma-derived therapy in adults with AATD


Ambros Therapeutics launches with $125 million to advance breakthrough therapy for rare pain disorder
News | December 19, 2025

Ambros Therapeutics launches with $125 million to advance breakthrough therapy for rare pain disorder

The funding will support the pivotal Phase 3 clinical trial of neridronate in CRPS-1


GSK’s experimental lung cancer drug gains FDA orphan drug status
Drug Approval | December 12, 2025

GSK’s experimental lung cancer drug gains FDA orphan drug status

GSK holds exclusive global rights (excluding mainland China, Hong Kong, Macau, and Taiwan) from Hansoh Pharma to advance its development and commercialization