Spinraza (nusinersen)

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Spinraza (nusinersen)

Manufacturer Biogen Idec Ltd
Disease Spinal Muscular Atrophy (SMA)
Indication Spinal muscular atrophy (SMA) in pediatric and adult patients
Mode of Action Antisense oligonucleotide (ASO)
Approval Status EMA approved (EU); FDA approved (USA); PMDA approved (JAP); TGA approved (AUS)
Strength 2.4 mg/mL

Who is nusinersen for?

Spinraza (nusinersen) is indicated for the treatment of pediatric and adult patients with spinal muscular atrophy (SMA) [1][4].

Recommended dose

Complete information about Spinraza (nusinersen) dosage and administration can be found here [1][4].
Spinraza is an injection administered into the fluid surrounding the spinal cord (intrathecal injection) [2][5]. The standard dosage is:
  • 12 mg / 5 mL per administration
Initiate treatment with 4 loading doses. The first dose should be followed by 3 more doses after 2, 4, and 9 weeks and then one dose every 4 months thereafter. Treatment should be continued for as long as the patient benefits from it [1][5].
Consult your treating doctor for personalised dosing.

What is nusinersen and how does it work?

Spinraza (nusinersen) is the first medicine approved [2] to treat pediatric and adult patients with spinal muscular atrophy (SMA) [1][4].
SMA is a rare and often fatal hereditary (genetic) disease that causes weakness and muscle wasting due to the loss of motor neurons controlling movement [2].
Spinraza (nusinersen) is an antisense oligonucleotide (ASO) that is designed to treat SMA caused by mutations in the chromosome 5q. This mutation leads to the deficiency of a protein, the survival motor neuron (SMN) protein. Spinraza (nusinersen) alters the synthesis of the deficient protein in order to increase production of full-length SMN protein and thereby promoting the maintenance of motor neurons [3].

What is nusinersen's approval status?

Spinraza (nusinersen) was approved by:
  • FDA (USA) on December 23, 2016 [2]
  • EMA (EU) on May 30, 2017 [5]
for the treatment of children and adults with spinal muscular atrophy (SMA).
The efficacy of nusinersen for SMA was assessed in a clinical trial (CS3B - ENDEAR) involving 121 patients with infantile-onset SMA who were diagnosed before 6 months of age and who were less than 7 months old at the time of their first dose [1][4]. Patients were randomised to receive an injection of Spinraza (nusinersen), into the fluid surrounding the spinal cord, or undergo a mock procedure (sham-control) without drug injection (a skin prick) [1][4].
At the final analysis, the percentage of responders (patients achieving the pre-defined motor milestone responder criteria) among the nusinersen group was 51 % vs 0 %  among the patients in the control group. Fewer patients died or received permanent ventilation among the nusinersen treated patients (39 %) compared with the control group (68 %) [4]. The portion of patients that achieved improvement in total milestone score (which included the ability to kick, head control, rolling, sitting, crawling, standing or walking) was 67 % in the patients treated with nusinersen vs 17% in the sham-control group, while the portion of patients that worsened in the two groups was 1 % and 22 % respectively [4]. When measured with the Children’s Hospital of Philadelphia Infant Test for Neuromuscular Disease (CHOP INTEND) score, the proportion of patients achieving a 4-point improvement was 73 % with nusinersen and 3 % without, while the proportion of those worsening was 7 % with nusinersen and 49 % without [4].
Positive results were achieved also in a study on patients with later onset SMA (after 6 months of age). The study CS4 (CHERISH) involved 126 patients randomised 2:1 to either nusinersen or sham-control. The main measure considered was the Hammersmith Functional Motor Scale Expanded (HFMSE). Among the patients treated with nusinersen, 57.3 % achieved an improvement from baseline of at least 3 points vs. 20.5 % of the patients in the control group [4].

Adverse reactions include back pain, headache and vomiting [4].
[1] Summary of Product Characteristics [FDA]: Spinraza (nusinersen), Biogen, Dec. 2016.
[2] FDA News Release. FDA approves first drug for spinal muscular atrophy, 23/12/2016.
[3] Biogen Press Release. U.S. FDA Approves Biogen’s SPINRAZA™ (nusinersen), The First Treatment for Spinal Muscular Atrophy, 23/12/2016.
[4] Summary of Product Characteristics [EMA]: Spinraza (nusinersen), Biogen, Jun. 2017.
[5] EMA. Human Medicines: Spinraza (nusinersen), (last update: 21/06/2017), cited on 18/10/2017.

Disclaimer: Every effort has been made to ensure that the information provided here is accurate, up-to-date and complete, but no guarantee is made to that effect. Medicine information contained herein may be time sensitive. The absence of a warning for a given medicine or combination thereof in no way should be construed to indicate that the medicine or combination is safe, effective or appropriate for any given patient. Always consult your treating physician before starting a course of treatment.

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