Spinraza (nusinersen), marketed by Biogen, was the first FDA-approved therapy to treat SMA. It is an SMN-enhancing therapy that works by targeting the SMN2 gene, causing it to make more complete protein.
More About Spinraza
Spinraza is FDA-approved for all ages and types of SMA.
Spinraza is given via an intrathecal (IT) injection, which is an injection into directly into the cerebrospinal fluid through the lower back. Individuals receive four “loading doses” within the first 2 months of treatment. Once those loading doses are completed, they receive a maintenance dose every 4 months for the duration of the individual’s life.
Spinraza is an antisense oligonucleotide. Antisense drugs are small snippets of synthetic genetic material that bind to ribonucleic acid (RNA), so they can be used to fix splicing errors in genes such as SMN2.
As with all treatments, timing is critical. Individuals who began Spinraza as soon after diagnosis as possible had better results than those who waited to begin treatment.
Several sites across the U.S. are dosing individuals with Spinraza. A number of these sites can be found on our map locator tool.
To find insurance policies and payer information for Spinraza, please click here.
Check out Biogen’s patient brochures on Spinraza for pediatrics and adults.
History of Spinraza
From 2003 to 2006, Cure SMA provided the very first research funding needed to begin investigation into this therapeutic approach. We would like to thank and acknowledge Cold Spring Harbor Laboratory (CSHL) and the University of Massachusetts Medical School for generating critical intellectual property for the program that was licensed to Ionis Pharmaceuticals.
We specifically thank Drs. Adrian Krainer, Yimin Hua, and colleagues at CSHL for years of dedication to and hard work on the pre-clinical development of Spinraza for SMA, as well as Drs. Ravindra Singh and Elliot Androphy for their work, funded by Cure SMA, in originally identifying the ISSN1 gene sequence, which is the sequence targeted in Spinraza.
After the pre-clinical work and early clinical testing was complete, Ionis Pharmaceuticals partnered with Biogen to launch a number of clinical trials testing Spinraza.
Recently Shared News on Spinraza
- 06/10/2021 – Biogen Presents New Spinraza Data at the 2021 Virtual SMA Conference
- 06/02/2021 – Biogen SMA Q2 2021 Community Statement
- 04/21/2021 – Biogen Shares New Spinraza Data Exploring Opportunities to Improve Outcomes for SMA Patients
- 03/19/2021 – Nusinersen Research Updates Presented by Biogen
- 03/08/2021 – Biogen SMA Q1 2021 Community Statement
- 12/28/2020 – Biogen Provides SMA Community Update on DEVOTE Study
- 11/20/2020 – Biogen Q4 2020 Community Statement
- 08/27/2020 – Biogen Q3 2020 Community Statement: COVID-19 Updates and Latest Milestones
- 07/21/2020 – Biogen Plans to Initiate Phase 4 Study Evaluating Benefit of Spinraza in Patients Treated with Zolgensma
- 06/10/2020 – Biogen Shares Results from Landmark NURTURE Study of Pre-Symptomatic SMA Patients Treated with Spinraza
- 05/18/2020 – Biogen Shares SPINRAZA Research Update
- 04/02/2020 – Biogen Announces First Patient Treated with Higher Dose of Spinraza in Phase 2/3 DEVOTE Study
- 12/16/2019 – Biogen Issues Q4 2019 Community Statement on Spinraza
- 10/03/2019 – Landmark NURTURE Study of Infants with SMA Treated Pre-Symptomatically with Spinraza Published in Neuromuscular Disorders
- 09/18/2019 – Biogen Advances SMA Clinical Research with New Study Evaluating a Higher Dose of SPINRAZA and Additional Data in a Broad Range of Patients
- 08/05/2019 – Biogen Issues Community Statement on Spinraza
- 06/06/2019 – Biogen Issues Q2 2019 Community Statement on Spinraza
- 05/01/2019 – Biogen to Present Data at AAN Highlighting Spinraza and Emerging Biomarkers for SMA
- 03/05/2019 – Biogen Provides Rare Disease Day 2019 Community Update on Spinraza