Nusinersen Brand Name– Spinraza
What is Nusinersen
Nusinersen is an intrathecally administered survival motor neuron-2 (SMN2)-directed antisense oligonucleotide.
Nusinersen is the first drug to be approved to treat pediatric and adult patients with spinal muscular atrophy (SMA).
SMA is a rare and often fatal hereditary genetic disease affecting muscle strength and movement. It causes weakness and muscle wasting because of the loss of lower motor neurons controlling movement. The age of onset, symptoms, and rate of progression vary widely between patients.
A clinical trial in 121 patients with infantile-onset SMA (diagnosed before 6 months of age; less than 7 months old when receiving the first nusinersen dose) assessed the percentage of patients with improvement in motor milestones (i.e. head control, rolling, sitting, crawling, standing, or walking).
In a planned interim efficacy analysis including 82 patients, 40% of patients treated with nusinersen achieved improvement in motor milestones, whereas none of the control did.
Additional open-label uncontrolled clinical studies conducted in symptomatic patients (age 30 days to 15 years at the time of the first dose) and in presymptomatic patients (age 8 days to 42 days at the time of the first dose) appear to support the clinical efficacy demonstrated in the controlled clinical trial in infants.
Because thrombocytopenia, coagulation abnormalities, and renal toxicity can occur during nusinersen therapy, platelet count, coagulation laboratory testing (prothrombin time; activated partial thromboplastin time), and quantitative spot urine protein testing are recommended at baseline and prior to each dose.
Nusinersen was approved by the FDA in December 2016.
Indications
- spinal muscular atrophy
For the treatment of spinal muscular atrophy (SMA)
NOTE: The FDA has designated nusinersen as an orphan drug for this indication.
Side Effects
- angioedema
- antibody formation
- back pain
- bleeding
- constipation
- epistaxis
- fever
- flatulence
- glomerulonephritis
- growth inhibition
- headache
- hydrocephalus
- hyponatremia
- infection
- meningitis
- nasal congestion
- nephrotoxicity
- QT prolongation
- rash
- thrombocytopenia
- urticaria
- vomiting
- weight loss
Monitoring Parameters
- partial thromboplastin time (PTT)
- platelet count
- prothrombin time (PT)
- urinalysis
Contraindications
- bleeding
- breast-feeding
- nephrotoxicity
- pregnancy
- requires a specialized care setting
- requires an experienced clinician
- thrombocytopenia
Interactions
There are no drug interactions associated with Nusinersen products.
