Drug

Nusinersen

Regulatory activity · MHRA

Emerging researchLate-stage development

Also known as Spinraza.

RxNorm1863556 ChEMBLCHEMBL3301572 ATCM09AX07UNII5Z9SP3X666

Research papers

Active clinical trials

Regulatory events

Latest activity

beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen

Clinical trial2026-07-31Primary completion · ClinicalTrials.gov

A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam

Clinical trial2026-05-22Results expected Q3 2027 · ClinicalTrials.gov

A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)

Clinical trial2026-05-01Results expected Q4 2028 · ClinicalTrials.gov

An Open Label, Single Cohort Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)

Clinical trial2026-03-18Results expected Q2 2027 · ClinicalTrials.gov

An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Clinical trial2025-10-20Results posted · ClinicalTrials.gov

A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec

Clinical trial2025-10-09Completed · ClinicalTrials.gov

Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

Clinical trial2025-06-05Results posted · ClinicalTrials.gov

An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443

Clinical trial2024-10-22Results posted · ClinicalTrials.gov

Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms): a Multicenter Single-case Experimental Design in Multiple Baselines Across Subjects, Randomized, Single-blinded Evaluation

Clinical trial2023-09-08Completed · ClinicalTrials.gov

Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study.

Research2019-09-12Neuromuscular disorders : NMD

Drug Safety Update: Nusinersen (Spinraza▼): reports of communicating hydrocephalus; discuss symptoms with patients and carers and investigate urgently

Regulatory2018-09-25MHRA

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.

Research2017-11-01The New England journal of medicine

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name

Nusinersen

Aliases & brands

Spinraza

RxNorm CUI

1863556

ChEMBL ID

CHEMBL3301572

ATC codes

M09AX07

UNII

5Z9SP3X666

Regulatory jurisdictions

mhra

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Authorities

MHRA

Total events

mhraMedicines and Healthcare products Regulatory Agency· 1 event

2018-09-25Safety communicationSafety

Drug Safety Update: Nusinersen (Spinraza▼): reports of communicating hydrocephalus; discuss symptoms with patients and carers and investigate urgently

Evidence ↗

Contains public sector information from the Medicines and Healthcare products Regulatory Agency (MHRA) licensed under the Open Government Licence (OGL v3).