• SKIP TO CONTENT
  • SKIP NAVIGATION
  • Patient Resources
    • COVID-19 Patient Resource Center
    • Clinical Trials
    • Search Clinical Trials
    • Patient Notification System
    • What is Clinical Research?
    • Volunteering for a Clinical Trial
    • Understanding Informed Consent
    • Useful Resources
    • FDA Approved Drugs
  • Professional Resources
    • Research Center Profiles
    • Clinical Trial Listings
    • Market Research
    • FDA Approved Drugs
    • Training Guides
    • Books
    • eLearning
    • Events
    • Newsletters
    • White Papers
    • SOPs
    • eCFR and Guidances
  • White Papers
  • Trial Listings
  • Advertise
  • COVID-19
  • iConnect
  • Sign In
  • Create Account
  • Sign Out
  • My Account
Home » Directories » FDA Approved Drugs » Spinraza (nusinersen)

AND
  • A
  • B
  • C
  • D
  • E
  • F
  • G
  • H
  • I
  • J
  • K
  • L
  • M
  • N
  • O
  • P
  • Q
  • R
  • S
  • T
  • U
  • V
  • W
  • X
  • Y
  • Z

Spinraza (nusinersen)

  • Profile

Profile

Contact Information

Contact: Biogen
Website: http://www.spinraza.com/

Currently Enrolling Trials

    Show More

    General Information

    Spinraza (nusinersen) is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide.

    Spinraza is specifically indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

    Mechanism of Action

    Spinraza (nusinersen) is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide. It was designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, Spinraza was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein.

    Side Effects

    Adverse effects associated with the use of Spinraza may include, but are not limited to, the following:

    • lower respiratory infection
    • upper respiratory infection
    • constipation

    Dosing/Administration

    Spinraza is supplied as a solution to be administered intrathecally. The recommended dosage is 12 mg (5 mL) per administration. Initiate Spinraza treatment with four loading doses. The first three loading doses should be administered at 14-day intervals. The fourth loading dose should be administered 30 days after the third dose. A maintenance dose should be administered once every four months thereafter.

    Missed Dose: If a loading dose is delayed or missed, administer Spinraza as soon as possible, with at least 14 days between doses and continue dosing as prescribed. If a maintenance dose is delayed or missed, administer Spinraza as soon as possible and continue dosing every four months. 

    Clinical Trial Results

    The FDA approval of Spinraza was based on ENDEAR, a randomized, double-blind, sham-controlled phase 3 study in 121 patients with infantile-onset (most likely to develop type 1) SMA. At a planned interim analysis of ENDEAR, a greater percentage of infants treated with Spinraza achieved a motor milestone response compared to those who did not receive treatment (40 percent versus 0 percent; p<0.0001) as measured by the Hammersmith Infant Neurological Examination (HINE). Additionally, a smaller percentage of patients on Spinraza died (23 percent) compared to untreated patients (43 percent). Data from the other efficacy endpoints analyzed were consistently in favor of infants who received treatment.

    The results of the controlled trial in infantile-onset SMA patients were supported by open-label uncontrolled trials conducted in symptomatic SMA patients who ranged in age from 30 days to 15 years at the time of first dose, and in presymptomatic patients, who ranged in age from 8 days to 42 days at the time of first dose. The patients in these studies had or were likely to develop Type 1, 2 or 3 SMA. Some patients achieved milestones such as ability to sit unassisted, stand or walk when they would otherwise be unexpected to do so, maintained milestones at ages when they would be expected to be lost and survived to ages unexpected considering the number of SMN2 gene copies of patients enrolled in the studies.

     

    Approval Date: 2016-12-01
    Company Name: Biogen
    Back to Listings

    Upcoming Events

    • 16Feb

      Fundamentals of FDA Inspection Management: Reduce Anxiety, Increase Inspection Success

    • 21May

      WCG MAGI Clinical Research Conference – 2023 East

    Featured Products

    • Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

      Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

    • Surviving an FDA GCP Inspection

      Surviving an FDA GCP Inspection: Resources for Investigators, Sponsors, CROs and IRBs

    Featured Stories

    • Revamp-360x240.png

      Califf Calls for Major Evidence Generation Revamp, Experts’ Opinions Differ

    • AskTheExpertsGreen-360x240.png

      Ask the Experts: Managing Investigational Products

    • SurveywBlueBackground-360x240.png

      Survey Outlines Site Challenges, Successes on Diversity

    • PatientCentricity-360x240.png

      Site Spotlight: DM Clinical Shows Patient Centricity Doesn’t Have to Break the Bank

    Standard Operating Procedures for Risk-Based Monitoring of Clinical Trials

    The information you need to adapt your monitoring plan to changing times.

    Learn More Here
    • About Us
    • Contact Us
    • Privacy Policy
    • Do Not Sell or Share My Data

    Footer Logo

    300 N. Washington St., Suite 200, Falls Church, VA 22046, USA

    Phone 617.948.5100 – Toll free 866.219.3440

    Copyright © 2023. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing