Spinal Muscular Atrophy

Rare Disease·MeSH: Muscular Atrophy, Spinal

Prevalence

1.5 / 100K

Incidence

1.0 / 100K/yr

21 drugs in development·12 companies·9 clinical trials

Drug Pipeline by Phase

Approved(4)

Phase 3(2)

Drug	Company
salanersen	BIOGEN INC. (BIIB)
Salanersen (BIIB115)	BIOGEN INC. (BIIB)

Phase 2(7)

Drug	Company
apitegromab	Scholar Rock Holding Corp (SRRK)
Apitegromab	PTC THERAPEUTICS, INC. (PTCT)
argx-119 iv	ARGENX SE (ARGX)
Nusinersen	PTC THERAPEUTICS, INC. (PTCT)
Nusinersen	Scholar Rock Holding Corp (SRRK)
Risdiplam	Scholar Rock Holding Corp (SRRK)
taldefgrobep alfa	Biohaven Ltd. (BHVN)

Phase 1(2)

Drug	Company
avxs-101	AveXis, Inc. (AVXS)
Nusinersen	BIOGEN INC. (BIIB)

Preclinical(3)

Drug	Company
ALPHA-0702	Alpha Cognition Inc. (ACOG)
ALPHA-0802	Alpha Cognition Inc. (ACOG)
stl-182	SPOTLIGHT INNOVATION INC. (STLT)

Phase 1/Phase 2(1)

Drug	Company
gb221	IMMAGE BIOTHERAPEUTICS CORP. (IMMG)

Phase 4(2)

Drug	Company
onasemnogene abeparvovec	NOVARTIS AG (NVSEF)
onasemnogene abeparvovec	Novartis AG (NVS)

BioSniper tracks 21 drugs in development for Spinal Muscular Atrophy across 12 companies.

There are 9 clinical trials targeting Spinal Muscular Atrophy tracked by BioSniper.

The estimated prevalence of Spinal Muscular Atrophy is approximately 1.5 per 100,000 population.

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