How do I subscribe to ongoing alerts?

Subscribe to /feed/disease/spinal-muscular-atrophy-sma-with-a-genetically-confirmed-diagnosis-of-sma-type-1-or-sma-type-2-or-3-in-patients-with-a-body-weight-of-13-kg-or-less.xml in Feedly / Inoreader. Every regulatory action, PDUFA date, or trial completion in this treatment area lands in your feed automatically.

Last reviewed 2026-06-10 · How we verify

Spinal Muscular Atrophy (SMA) with a genetically confirmed diagnosis of SMA type 1, or SMA type 2 or 3 in patients with a body weight of 13 kg or less — Treatment Landscape & Competitive Intelligence

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Spinal Muscular Atrophy (SMA) with a genetically confirmed diagnosis of SMA type 1, or SMA type 2 or 3 in patients with a body weight of 13 kg or less (disease) competitive landscape: 1 marketed treatments tracked, 0 Phase 3 candidates, 0 Phase 2 candidates. Recent regulatory actions and upcoming PDUFA dates across the

1 marketed 0 Phase 3 0 Phase 2 Live · 30-min refresh

Marketed treatment landscape

Approved drugs treating Spinal Muscular Atrophy (SMA) with a genetically confirmed diagnosis of SMA type 1, or SMA type 2 or 3 in patients with a body weight of 13 kg or less, deduplicated by molecule. See the full disease page for line-of-therapy detail.

Drug	Generic	Sponsor	Class	Target	Line of therapy	First approval
Pf-06954522	pf-06954522	Pfizer	Small molecule inhibitor	[specific target]

Phase 3 pipeline

No Phase 3 pipeline candidates tracked.

Phase 2 pipeline

No Phase 2 pipeline candidates tracked.

Recent regulatory actions (last 90 days)

No regulatory actions in the last 90 days for this treatment set.

Upcoming PDUFA dates (next 180 days)

No PDUFA dates in the next 180 days for this treatment set.

Sponsor landscape

Pfizer · 1 drug in Spinal Muscular Atrophy (SMA) with a genetically confirmed diagnosis of SMA type 1, or SMA type 2 or 3 in patients with a body weight of 13 kg or less

Subscribe to ongoing alerts

Every new regulatory action, PDUFA date, or trial completion in Spinal Muscular Atrophy (SMA) with a genetically confirmed diagnosis of SMA type 1, or SMA type 2 or 3 in patients with a body weight of 13 kg or less:

Cite this brief

Drug Landscape (2026). Spinal Muscular Atrophy (SMA) with a genetically confirmed diagnosis of SMA type 1, or SMA type 2 or 3 in patients with a body weight of 13 kg or less — Treatment Landscape & Competitive Intelligence Brief. https://druglandscape.com/ci/disease/spinal-muscular-atrophy-sma-with-a-genetically-confirmed-diagnosis-of-sma-type-1-or-sma-type-2-or-3-in-patients-with-a-body-weight-of-13-kg-or-less. Accessed 2026-06-10.

Spinal Muscular Atrophy (SMA) with a genetically confirmed diagnosis of SMA type 1, or SMA type 2 or 3 in patients with a body weight of 13 kg or less full disease profile — treatment pathway, diagnostics, guidelines
Browse all CI briefs
Build a custom feed

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing