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NS-580/NM-8074

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Phase 2Active

Key Facts

Indication

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Phase

Phase 2

Status

Active

Company

Nippon Shinyaku

About Nippon Shinyaku

Nippon Shinyaku's mission is to address high-unmet medical needs through pioneering science, particularly in rare diseases. Its key achievement is the development and commercialization of Viltepso (viltolarsen), an exon-skipping therapy for Duchenne muscular dystrophy, establishing its technological credibility. The company's strategy is built on deep R&D in targeted therapeutic areas, a dominant position in the Japanese market, and strategic international expansion, especially in the United States.

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Therapeutic Areas

Neuromuscular Disorders Hematology Metabolic Diseases Oncology

Other Paroxysmal Nocturnal Hemoglobinuria (PNH) Drugs

Drug	Company	Phase	Status
OMS906	Omeros	Phase 2	Active
Empaveli (pegcetacoplan)	bioRASI	Approved	Commercial
KP104	Kira Pharmaceuticals	Phase 2	Active
EMPAVELI Injector	Enable Injections	Approved	Active
Ruxoprubart (NM8074)	NovelMed Therapeutics	Phase 2	Active
Fabhalta® (iptacopan)	Novartis	Approved	Active
Pegcetacoplan (Systemic)	Apellis Pharmaceuticals	Approved	Commercial