Hemophilia

Rare Disease·MeSH: Hemophilia A

Prevalence

5.0 / 100K

56 drugs in development·23 companies·6 clinical trials

Drug Pipeline by Phase

Approved(6)

Drug	Company
alprolix	Bioverativ Inc. (BIVV)
eloctate	Bioverativ Inc. (BIVV)
giroctocogene fitelparvovec	SANGAMO THERAPEUTICS, INC (SGMO)
hemgenix	uniQure N.V. (QURE)
ixinity	XOMA Royalty Corporation (XOMA)
tranexamic acid	NEXUS BIOPHARMA INC (NEXS)

Phase 3(11)

Drug	Company
adynovate	Takeda Pharmaceutical Company Limited American Depositary Shares (each representing 1/2 of a share of Common Stock) (TAK)
antithrombin concentrate	Sanofi (SNY)
Calaspargase Pegol	ENANTA PHARMACEUTICALS INC (ENTA)
clotting factor concentrates or bypassing agents	Sanofi (SNY)
Epoetin Alfa	ENANTA PHARMACEUTICALS INC (ENTA)
fidanacogene elaparvovec	Spark Therapeutics, Inc. (SPARK)
fitusiran	Sanofi (SNY)
Giroctocogene fitelparvovec (SB-525)	SANGAMO THERAPEUTICS, INC (SGMO)
iti	SHIRE PLC (SHPGF)
mri contrast-enhancing agents	Sanofi (SNY)
pegylated recombinant factor viii	SHIRE PLC (SHPGF)

Phase 2(1)

Drug	Company
SerpinPC	Centessa Pharmaceuticals plc (CNTA)

Phase 1(4)

Preclinical(11)

Drug	Company
bivv 001	Bioverativ Inc. (BIVV)
bivv 002	Bioverativ Inc. (BIVV)
bivv002	Bioverativ Inc. (BIVV)
dtx101	Dimension Therapeutics, Inc. (DMTX)
dtx201	Dimension Therapeutics, Inc. (DMTX)
dtx501	Dimension Therapeutics, Inc. (DMTX)
dtx601	Dimension Therapeutics, Inc. (DMTX)
hemophilia a program	Metagenomi Therapeutics, Inc. (MGX)
hemophilia a program	Generation Bio Co. (GBIO)
p-fviii-101	Poseida Therapeutics, Inc. (PSTX)
rt-117	Rani Therapeutics Holdings, Inc. (RANI)

Unknown(6)

Drug	Company
adynovi	SHIRE PLC (SHPGF)
ADYNOVI/ADYNOVATE	ENANTA PHARMACEUTICALS INC (ENTA)
Damoctocog alfa pegol	ENANTA PHARMACEUTICALS INC (ENTA)
HEMGENIX	ENANTA PHARMACEUTICALS INC (ENTA)
obizur	TAKEDA PHARMACEUTICAL CO LTD (TKPHF)
obizur	Takeda Pharmaceutical Company Limited American Depositary Shares (each representing 1/2 of a share of Common Stock) (TAK)

Phase 1/Phase 2(10)

Drug	Company
askbio009	SHIRE PLC (SHPGF)
bax 888	SHIRE PLC (SHPGF)
bivv001	Bioverativ Inc. (BIVV)
lnp1265	REGENERON PHARMACEUTICALS, INC. (REGN)
regv131	REGENERON PHARMACEUTICALS, INC. (REGN)
sig-001	Sigilon Therapeutics, Inc. (SGTX)
spk-8011	Spark Therapeutics, Inc. (SPARK)
spk-8016	Spark Therapeutics, Inc. (SPARK)
spk-9001	Spark Therapeutics, Inc. (SPARK)
valoctocogene roxaparvovec	BIOMARIN PHARMACEUTICAL INC (BMRN)

Phase 2/Phase 3(2)

Drug	Company
single dose intravenous injection of bbm-h803	Q BioMed Inc. (QBIO)
single dose intravenous injection of bbm-h901	Q BioMed Inc. (QBIO)

Phase 4(5)

Drug	Company
antithrombin concentrate	Sanofi (SNY)
clotting factor concentrates or bypassing agents	Sanofi (SNY)
emicizumab	Sanofi (SNY)
emicizumab	Sanofi (SNYNF)
fitusiran	Sanofi (SNY)

BioSniper tracks 56 drugs in development for Hemophilia across 23 companies.

There are 6 clinical trials targeting Hemophilia tracked by BioSniper.

The estimated prevalence of Hemophilia is approximately 5.0 per 100,000 population.

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