Products
Nusinersen was approved in the United States in 2016 and in many countries and the EU in 2017 as a solution for intrathecal injection by lumbar puncture and as the first drug for SMA treatment (Spinraza).
Structure and properties
Nusinersen is a modified antisense oligonucleotide.
Effects
Nusinersen (ATC M09AX07) increases the formation of the full-length SMN protein. It binds to -pre-mRNA and promotes the inclusion of exon 7 in SMN2 mRNA. This allows functional SMN protein to be formed. The half-life in CSF is 135 to 177 days.
Indications
For the treatment of 5q-associated spinal muscular atrophy (SMA).
Dosage
According to the SmPC. The drug is administered intrathecally as a lumbar puncture because it does not cross the blood–brain barrier.
Contraindications
- Hypersensitivity
For complete precautions, see the drug label.
Interactions
Drug-drug interactions via CYP450 enzymes or transporters are considered unlikely.
Adverse effects
The most common potential adverse effects include lower and upper respiratory tract infections and constipation.