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Results For "Orphan-Drug"

91 News Found

USFDA approves first gene therapy for treatment of aromatic L-amino acid decarboxylase deficiency
Drug Approval | November 16, 2024

USFDA approves first gene therapy for treatment of aromatic L-amino acid decarboxylase deficiency

Aromatic L-amino acid decarboxylase deficiency is a rare genetic disorder that affects the production of some neurotransmitters


Fasenra approved in the US for eosinophilic granulomatosis with polyangiitis
Drug Approval | September 19, 2024

Fasenra approved in the US for eosinophilic granulomatosis with polyangiitis

New indication supported by the MANDARA trial which showed nearly 60% of patients achieved remission and 41% of patients fully stopped taking oral corticosteroids


Zydus Lifesciences announces completion of Phase II(a) clinical trial of Usnoflast
Clinical Trials | September 11, 2024

Zydus Lifesciences announces completion of Phase II(a) clinical trial of Usnoflast

ALS patients experience neuroinflammation and rapid neurodegeneration


Merck announces Phase 3 trial initiation for Bomedemstat
Clinical Trials | August 27, 2024

Merck announces Phase 3 trial initiation for Bomedemstat

The initiation of a second Phase 3 clinical trial for bomedemstat demonstrates company’s commitment to advancing research in myeloproliferative neoplasms (MPNs)


Biostar Pharma gets FDA clearance of the IND application for a Phase 2 study of Utidelone Injection
Drug Approval | July 09, 2024

Biostar Pharma gets FDA clearance of the IND application for a Phase 2 study of Utidelone Injection

Utidelone can penetrate BBB due to its unique physicochemical characteristics and insusceptibility to P-glycoprotein-mediated efflux


Voydeya approved in the EU as add-on treatment to ravulizumab
News | April 23, 2024

Voydeya approved in the EU as add-on treatment to ravulizumab

ALPHA Phase III trial showed first-in-class, oral, Factor D inhibitor as add-on to Ultomiris or Soliris improved haemoglobin levels and reduced anaemia and fatigue


USFDA grants ODD to NM5072 for treatment of Paroxysmal Nocturnal Hemoglobinuria
News | April 17, 2024

USFDA grants ODD to NM5072 for treatment of Paroxysmal Nocturnal Hemoglobinuria

NM5072 is an anti-Properdin antibody that selectively blocks the alternative pathway while maintaining the integrity of the classical pathway required for fending off infections


FDA Granted ODD to Utidelone Injectable from Biostar Pharma for treatment of breast cancer brain metastasis
Drug Approval | April 01, 2024

FDA Granted ODD to Utidelone Injectable from Biostar Pharma for treatment of breast cancer brain metastasis

Utidelone has the ability to cross BBB due to its unique physicochemical characteristic and insusceptibility to P-glycoprotein-mediated efflux


USFDA grants ODD to Cevidoplenib for immune thrombocytopenia
News | March 23, 2024

USFDA grants ODD to Cevidoplenib for immune thrombocytopenia

Oscotec has successfully completed phase 2 study in patients with chronic ITP last year


Merck completes acquisition of Harpoon Therapeutics
News | March 12, 2024

Merck completes acquisition of Harpoon Therapeutics

Acquisition broadens oncology pipeline with a portfolio of novel T-cell engagers including HPN328 (MK-6070), an investigational delta-like ligand 3 (DLL3) targeting T-cell engager