Paroxysmal Nocturnal Hemoglobinuria

Rare Disease·MeSH: Hemoglobinuria, Paroxysmal

Prevalence

5.0 / 100K

24 drugs in development·10 companies·13 clinical trials

Drug Pipeline by Phase

Approved(4)

Drug	Company
Aspaveli	Apellis Pharmaceuticals, Inc. (APLS)
empaveli	KORU Medical Systems Inc. Common Stock (DE) (KRMD)
EMPAVELI	Apellis Pharmaceuticals, Inc. (APLS)
Ultomiris	Xencor Inc (XNCR)

Phase 3(9)

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

Phase 2(5)

Drug	Company
ach-4471	ACHILLION PHARMACEUTICALS INC (ACHN)
oms906	OMEROS CORP (OMER)
OMS906	Xencor Inc (XNCR)
zaltenibart	OMEROS CORP (OMER)
Zaltenibart (OMS906)	Xencor Inc (XNCR)

Phase 1(2)

Drug	Company
Pozelimab	REGENERON PHARMACEUTICALS, INC. (REGN)
xh-s003	RA PHARMACEUTICALS, INC. (RARX)

Preclinical(1)

Drug	Company
oral factor d inhibitors	ACHILLION PHARMACEUTICALS INC (ACHN)

Phase 1/Phase 2(1)

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

Phase 4(2)

Drug	Company
eculizumab	REGENERON PHARMACEUTICALS, INC. (REGN)
eculizumab	ALEXION PHARMACEUTICALS, INC. (ALXN)

BioSniper tracks 24 drugs in development for Paroxysmal Nocturnal Hemoglobinuria across 10 companies.

There are 13 clinical trials targeting Paroxysmal Nocturnal Hemoglobinuria tracked by BioSniper.

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

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