Paroxysmal Nocturnal Hemoglobinuria (PNH)

Rare Disease·MeSH: Hemoglobinuria, Paroxysmal

Prevalence

5.0 / 100K

13 drugs in development·8 companies·5 clinical trials

Drug Pipeline by Phase

Approved(2)

Drug	Company
empaveli	KORU Medical Systems Inc. Common Stock (DE) (KRMD)
Ultomiris	Xencor Inc (XNCR)

Phase 3(6)

Drug	Company
APL-3007, pegcetacoplan	Apellis Pharmaceuticals, Inc. (APLS)
lnp023	Novartis AG (NVS)
nomacopan	Akari Therapeutics Plc (AKTX)
Pegcetacoplan	Apellis Pharmaceuticals, Inc. (APLS)
Pegcetacoplan (systemic)	Apellis Pharmaceuticals, Inc. (APLS)
Systemic pegcetacoplan	Apellis Pharmaceuticals, Inc. (APLS)

Phase 2(4)

Drug	Company
ach-4471	ACHILLION PHARMACEUTICALS INC (ACHN)
OMS906	Xencor Inc (XNCR)
zaltenibart	OMEROS CORP (OMER)
Zaltenibart (OMS906)	Xencor Inc (XNCR)

Phase 1/Phase 2(1)

Drug	Company
aln-cfb	REGENERON PHARMACEUTICALS, INC. (REGN)

BioSniper tracks 13 drugs in development for Paroxysmal Nocturnal Hemoglobinuria (PNH) across 8 companies.

There are 5 clinical trials targeting Paroxysmal Nocturnal Hemoglobinuria (PNH) tracked by BioSniper.

The estimated prevalence of Paroxysmal Nocturnal Hemoglobinuria (PNH) is approximately 5.0 per 100,000 population.

Full trial data, competitive analysis, and AI-powered pipeline tracking.