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The European Medicines Agency (EMA), which evaluates medicinal products for use in EU, has recommended nusinersen (Spinraza; Biogen) as a treatment for patients with spinal muscular atrophy (SMA).
There is currently no approved therapy in the EU for SMA, a rare and often fatal genetic disease that affects the motor neurons causing muscle weakness and gradual loss of movement. Nusinersen is an “antisense oligonucleotide” medicine.
The EMA’s recommendation is based on the outcome of one complete randomised, double-blind clinical trial examining patients with infantile-onset SMA, and a number of ongoing trials with patients with different levels of disease severity including infantile-onset, childhood-onset and those in the pre-symptomatic phase.
The complete clinical trial was conducted with 121 patients who randomly received either an injection of nusinersen into the spinal cord fluid or a mock procedure skin prick. Of those who received the drug, 51% met the criteria as responders, compared to 0% in the control group.
In the drug arm, 22% achieved full head control, 8% were able to sit independently and 1% were able to stand unsupported. None of the individuals in the control group were able to reach these motor milestones.
The most common side effects observed with the drug were upper and lower respiratory infection and constipation. It is not yet known whether the effects of the drug will be maintained in the long term or whether it may be able to provide a cure for some patients in future.
