NDAINTRATHECALSOLUTIONPriority Review
Approved
Dec 2016
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
20
Data completeness
4/8 — Partial
Priority Review
Drug data last refreshed 22h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
SPINRAZA (nusinersen) is an intrathecal RNA therapeutic that modulates survival motor neuron-2 (SMN2) pre-mRNA splicing to increase functional SMN protein production. It treats spinal muscular atrophy (SMA), a rare genetic neuromuscular disorder caused by SMN1 gene loss. The drug addresses a previously untreatable fatal condition by restoring critical motor neuron survival protein.
$3.0MPart D
Peakmonopoly in SMA (pre-competitor entry)
9.7 years to LOEPeak-stage rare disease asset with minimal generic competition through 2036; commercial team focuses on maintaining specialist dominance and managed care access rather than growth.
Mechanism of Action
Pharmacologic Class:
Survival Motor Neuron-2-directed RNA Interaction
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (20)
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
Started Apr 2025
45 enrolled
Spinal Muscular Atrophy
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
Started Jan 2025
58 enrolled
Muscular Atrophy, Spinal
A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies
Started Dec 2023
20 enrolled
Muscular Atrophy, Spinal
Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam
Started Jan 2023
27 enrolled
Spinal Muscular Atrophy
Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
Started Nov 2022
10 enrolled
Spinal Muscular Atrophy
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.