What is Spinraza and Nusinersen used for?
Spinraza is a medicine used to treat spinal muscular atrophy (SMA) 5q, a genetic disease that causes weakness and muscle atrophy, even of the lung muscles. The disease is associated with a defect in chromosome 5q and symptoms generally begin shortly after birth.
Because the number of patients with SMA is low, the disease is considered 'rare' and Spinraza was designated an 'orphan medicine' (a medicine used in rare diseases) on 2 April 2012.
Spinraza contains the active ingredient nusinersen.
How is Spinraza used - Nusinersen?
Spinraza can only be obtained with a prescription and treatment must be started by a physician experienced in the management of SMA.
The medicine is available as a solution for injection in 12 mg vials. It is administered by intrathecal injection (in the lumbar region, directly into the spine) by a doctor or nurse who is experienced in performing this procedure. It may be necessary to sedate the patient (administer a medicine to calm him) before administering Spinraza.
The recommended dose is 12 mg (one vial) to be given as soon as possible after the patient has been diagnosed with SMA. The first dose should be followed by another 3 doses, after 2, 4 and 9 weeks and then by a dose every 4 weeks. Treatment should continue until the patient benefits. For more information, see the package leaflet.
How does Spinraza - Nusinersen work?
Patients with SMA lack a protein called "motor neuron survival factor" (SMN), which is essential for the survival and normal functioning of motor neurons (nerve cells in the spinal cord that control muscle movements). The SMN protein is produced by two genes, SMN1 and SMN2. Patients with SMA do not have the SMN1 gene but have the SMN2 gene which mostly produces a short SMN protein that does not work as well as a full length protein.
Spinraza is an antisense synthetic oligonucleotide (a type of genetic material) that allows the SMN2 gene to produce the full-length protein that can function normally. This replaces the missing protein, thus alleviating the symptoms of the disease.
What benefit has Spinraza - Nusinersen shown during the studies?
A main study involving 121 newborns (average age 7 months) with SMA showed that Spinraza is effective in improving movement when compared with placebo (a dummy injection).
After a year of treatment, in 51% of newborns who were given Spinraza (37 out of 73), progress was made in developing head control, rolling, sitting, crawling, standing up and walking while not No similar progress was observed in the newborns who received placebo. Furthermore, most newborns treated with Spinraza survived longer and required assisted breathing later than those who received placebo.
Another study is underway to evaluate the efficacy of Spinraza in children with less severe SMA and diagnosed at a later stage (average age 3 years). The interim analysis showed results consistent with those of newborns in whom the disease had an earlier onset.
What are the risks associated with Spinraza - Nusinersen?
The most common side effects with Spinraza (which may affect more than 1 in 10 people) are headache and back pain; however, in newborns these side effects could not be assessed as they were unable to communicate them. These side effects are thought to be caused by injections into the spine made to administer the medicine.
For the full list of all side effects and restrictions with Spinraza, see the package leaflet.
Why has Spinraza - Nusinersen been approved?
In its assessment, the Committee for Medicinal Products for Human Use (CHMP) recognized the serious nature of the disease and the urgent need for effective treatments.
Spinraza has been shown to result in clinically significant improvements in young children with disease in different degrees of severity. Although the medicine has not been tested in patients with the most severe and mildest forms of SMA, it is expected to provide similar benefits to these patients.
The side effects have been considered as manageable, as most of them are related to the route of administration of the medicine.
Therefore, the CHMP decided that Spinraza's benefits are greater than its risks and recommended that it be approved for use in the EU.
What measures are being taken to ensure the safe and effective use of Spinraza - Nusinersen?
The company that markets Spinraza will complete ongoing studies on the long-term safety and efficacy of the medicine in patients who present with the symptoms of SMA and in patients who still have no symptoms.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Spinraza have also been included in the summary of product characteristics and the package leaflet.
More information on Spinraza - Nusinersen
For the full EPAR version of Spinraza, consult the Agency's website: ema.europa.eu/Find medicine / Human medicines / European public assessment reports. For more information about treatment with Spinraza, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
The summary of the opinion of the Committee for Orphan Medicinal Products related to Spinraza is available on the Agency's website: ema.europa.eu/Find medicine / Human medicines / Rare disease designation.
