This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. See section 4.8 for how to report adverse reactions.
Spinraza 12 mg solution for injection
Each 5 ml vial contains nusinersen sodium equivalent to 12 mg nusinersen.
Each ml contains 2.4 mg of nusinersen.
For the full list of excipients, see section 6.1.
Solution for injection.
Clear and colourless solution with pH of approximately 7.2.
Spinraza is indicated for the treatment of 5q Spinal Muscular Atrophy.
Treatment with Spinraza should only be initiated by a physician with experience in the management of spinal muscular atrophy (SMA).
The decision to treat should be based on an individualised expert evaluation of the expected benefits of treatment for that individual, balanced against the potential risk of treatment with Spinraza. Patients with profound hypotonia and respiratory failure at birth, where Spinraza has not been studied, may not experience a clinically meaningful benefit due to severe SMN protein deficiency.
Spinraza is for intrathecal use by lumbar puncture.
The recommended dosage is 12 mg (5 ml) per administration.
Spinraza treatment should be initiated as early as possible after diagnosis with 4 loading doses on Days 0, 14, 28 and 63. A maintenance dose should be administered once every 4 months thereafter.
Duration of treatment
Information on long term efficacy of this medicinal product is not available. The need for continuation of therapy should be reviewed regularly and considered on an individual basis depending on the patient's clinical presentation and response to the therapy.
Missed or delayed doses
If a loading dose is delayed or missed Spinraza should be administered as soon as possible, with at least 14 days between doses, and continue dosing at the prescribed frequency. If a maintenance dose is delayed or missed, Spinraza should be administered as soon as possible and dosing continued every 4 months.
Spinraza has not been studied in patients with renal impairment. The safety and efficacy in patients with renal impairment has not been established and they should be closely observed.
Spinraza has not been studied in patients with hepatic impairment. Spinraza is not metabolised via the cytochrome P450 enzyme system in the liver, therefore dose adjustment is unlikely to be required in patients with hepatic impairment (see sections 4.5 and 5.2).
Method of administration
Treatment should be administered by health care professionals experienced in performing lumbar punctures.
Spinraza is administered as an intrathecal bolus injection over 1 to 3 minutes, using a spinal anaesthesia needle. The injection must not be administered in areas of the skin where there are signs of infection or inflammation. It is recommended that the volume of cerebral spinal fluid (CSF), equivalent to the volume of Spinraza to be injected, is removed prior to administration of Spinraza.
Sedation may be required to administer Spinraza, as indicated by the clinical condition of the patient.
Ultrasound (or other imaging techniques) may be considered to guide intrathecal administration of Spinraza, particularly in younger patients and in patients with scoliosis. Aseptic technique should be used when preparing and administering Spinraza; see instructions for use in section 6.6.
Hypersensitivity to the active substance or to any of the excipients listed in section 6.1.
Lumbar puncture procedure
There is a risk of adverse reactions occurring as part of the lumbar puncture procedure (e.g. headache, back pain, vomiting; see section 4.8). Potential difficulties with this route of administration may be seen in very young patients and those with scoliosis. The use of ultrasound or other imaging techniques to assist with intrathecal administration of Spinraza, can be considered at the physician's discretion.
Thrombocytopenia and coagulation abnormalities
Thrombocytopenia and coagulation abnormalities, including acute severe thrombocytopenia, have been observed after administration of other subcutaneously or intravenously administered antisense oligonucleotides. If clinically indicated, platelet and coagulation laboratory testing is recommended prior to administration of Spinraza.
Renal toxicity has been observed after administration of other subcutaneously and intravenously administered antisense oligonucleotides. If clinically indicated, urine protein testing (preferably using a first morning urine specimen) is recommended. For persistent elevated urinary protein, further evaluation should be considered.
There have been reports of communicating hydrocephalus not related to meningitis or bleeding in patients treated with nusinersen in the post-marketing setting. Some patients were implanted with a ventriculo-peritoneal shunt. In patients with decreased consciousness, an evaluation for hydrocephalus should be considered. The benefits-and risks of nusinersen treatment in patients with a ventriculo-peritoneal shunt are unknown at present and the maintenance of treatment needs to be carefully considered.