The European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended that the orphan medicine, Spinraza (nusinersen) be granted a marketing authorisation for treating patients with the rare genetic disease, 5q spinal muscular atrophy (SMA).
There is currently no approved therapy in the European Union for the treatment of SMA, with patients instead receiving supportive treatment. The US Food and Drug Administration approved Spinraza for the treatment of SMA in December 2016.
The recommendation from the CHMP is based on the results of one completed clinical trial and a number of ongoing trials in patients with SMA with different stages of disease severity.
In the main trial, the risk of death or permanent ventilation was 47 per cent lower in patients treated with Spinraza. Twenty-two per cent of patients achieved full head control, 8 per cent achieved independent sitting, and 1 per cent achieved standing with support, whereas no subjects in the control group achieved any of these milestones.
Spinraza is available as a 2.4mg/ml solution for injection once every four months. The most common side effects found in participants in the clinical trials were upper and lower respiratory tract infection and constipation.