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Results For "Orphan-Drug-Designation"

89 News Found

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


Praxis Precision hits milestone in fight against rare paediatric seizures
Clinical Trials | December 08, 2025

Praxis Precision hits milestone in fight against rare paediatric seizures

The EMBOLD study evaluated relutrigine for patients with SCN2A and SCN8A developmental and epileptic encephalopathies


Bayer advances fight against rare kidney disease with new experimental drug
R&D | December 07, 2025

Bayer advances fight against rare kidney disease with new experimental drug

Alport Syndrome is caused by genetic mutations affecting type IV collagen in the kidneys, ears, and eyes