What is Spinraza - Nusinersen and what is it used for?
Spinraza is a medicine used to treat 5q spinal muscular atrophy (SMA), a genetic disease that causes muscle weakness and atrophy, including of the lung muscles. The disease is associated with a defect in chromosome 5q and symptoms usually 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 substance nusinersen.
How is Spinraza used?
Spinraza can only be obtained with a prescription and treatment should be started by a doctor experienced in the management of SMA.
The medicine is available as a solution for injection in 12 mg vials. It is given as an intrathecal injection (in the lower back, directly into the spine) by a doctor or nurse experienced in performing this procedure. It may be necessary to sedate the patient (administer a medicine to calm him down) before giving Spinraza.
The recommended dose is 12 mg (one vial) to be administered as soon as possible after the patient has been diagnosed with SMA. The first dose should be followed by 3 more doses, after 2, 4 and 9 weeks and then by one dose every 4 months thereafter. Treatment should continue as long as the patient benefits from it. 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 movement). The SMN protein is produced by two genes, SMN1 and SMN2. Patients with SMA lack the SMN1 gene but have the SMN2 gene which mostly produces a short SMN protein that doesn't function as well as a full-length protein.
Spinraza is a synthetic antisense oligonucleotide (a type of genetic material) that allows the SMN2 gene to make the full-length protein that can function normally. This replaces the missing protein, thereby relieving the symptoms of the disease.
What benefit has Spinraza - Nusinersen shown during the studies?
One main study involving 121 infants (mean age 7 months) with SMA showed that Spinraza is effective in improving movement when compared to placebo (dummy injection).
After one year of treatment, 51% of infants who received Spinraza (37 out of 73) had progress in developing head control, rolling, sitting, crawling, standing and walking, while not No similar progress was observed in infants who received placebo. In addition, most infants 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 (mean age 3 years). The interim analysis showed results consistent with those of infants in whom the disease has 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 neonates these side effects could not be assessed as they were unable to communicate them. These side effects are believed to be caused by the injections into the spine given to administer the medicine.
For the full list of side effects and limitations reported 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 bring about clinically meaningful improvements in young children with the disease in varying degrees of severity. Although the medicine has not been tested in patients with the most severe and milder forms of SMA, it is expected to provide similar benefits to these patients.
Side effects were considered to be 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 have symptoms of SMA and in patients who do not yet have symptoms.
The recommendations and precautions to be observed by healthcare professionals and patients for Spinraza to be used safely and effectively have also been reported in the summary of product characteristics and package leaflet.
More information about Spinraza - Nusinersen
For the full version of Spinraza's EPAR, please consult the Agency's website: ema.europa.eu/Find medicine / Human medicines / European public assessment reports. For more information about Spinraza therapy, read the package leaflet (included with the EPAR) or contact your doctor or pharmacist.
The summary of the opinion of the Committee for Orphan Medicinal Products for Spinraza is available on the Agency's website: ema.europa.eu/Find medicine / Human medicines / Rare disease designation.
The information on Spinraza - Nusinersen published on this page may be out of date or incomplete. For a correct use of this information, see the Disclaimer and useful information page.
