Friday, July 10, 2026

Drug profile · INN

nusinersen sodium

nusinersen sodium (SPINRAZA INITIAL APPROVAL) is a smn2 pre-mrna positive modulator. Route of administration: intrathecal. Current US regulatory status: approved.

FDA: approved PMDA: approved
US status approved
Patents linked 0
Pipeline programs 4
News articles 0
Drug details — nusinersen sodium
US status
approved
Class
Route
INTRATHECAL
Patents
0
Programs
4
Data quality
0.84

Quick answer

nusinersen sodium (SPINRAZA INITIAL APPROVAL) is a smn2 pre-mrna positive modulator. Route of administration: intrathecal. Current US regulatory status: approved.

Key facts

INN nusinersen sodium
Brand SPINRAZA INITIAL APPROVAL
Mechanism SMN2 pre-mRNA positive modulator
Target SMN2 pre-mRNA
Route INTRATHECAL
Formulation SOLUTION

Mechanism of action

SMN2 pre-mRNA positive modulator

Primary target: SMN2 pre-mRNA

Approval history

Authority Status Date
FDA approved
PMDA approved

Pipeline programs

  1. SPINRAZA 28 mg, SPINRAZA 50 mg Phase 3 · Biogen · Spinal Muscular Atrophy
  2. SPINRAZA 50 mg, SPINRAZA 28 mg Phase 3 · Biogen · Spinal Muscular Atrophy
  3. Spinraza 12 mg solution for injection Phase 1 · Biogen · Spinal Muscular Atrophy
  4. Spinraza 12 mg solution for injection Approved · Biogen · Spinal Muscular Atrophy

Related & competitor drugs

  1. risdiplam Musculo-skeletal system (M09)

Frequently asked questions

What is nusinersen sodium?

nusinersen sodium (SPINRAZA INITIAL APPROVAL) is a smn2 pre-mrna positive modulator. Route of administration: intrathecal. Current US regulatory status: approved.

What is the brand name for nusinersen sodium?

SPINRAZA INITIAL APPROVAL is a marketed brand name for nusinersen sodium. Check FDA Drugs@FDA for the current US label holder and prescribing information.

What is the US approval status of nusinersen sodium?

nusinersen sodium has approved status in the United States according to our Drugs@FDA-derived records. Verify on the official FDA label before clinical or commercial decisions.