SPINRAZA Solution for injection Ref.[9367] Active ingredients: Nusinersen

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.

Posology

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.

Special populations

Renal impairment

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.

Hepatic impairment

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.

No cases of overdose associated with adverse reactions were reported in clinical studies.

In the event of an overdose, supportive medical care should be provided including consulting with a healthcare professional and close observation of the clinical status of the patient.

3 years.

Store in a refrigerator (2°C-8°C).

Do not freeze.

Keep the vial in the outer carton in order to protect from light.

If no refrigeration is available, Spinraza may be stored in its original carton, protected from light at or below 30°C for up to 14 days.

Prior to administration, unopened vials of Spinraza can be removed from and returned to the refrigerator if necessary. If removed from the original carton, the total combined time out of refrigeration should not exceed 30 hours, at a temperature that does not exceed 25°C.

5 ml in a Type I glass vial with bromobutyl rubber stopper and an aluminium over-seal and plastic cap.

Pack size of one vial per carton.

For single use only.

Instructions for preparation of the medicinal product before administration:

1. The Spinraza vial should be inspected for particles prior to administration. If particles are observed and/or the liquid in the vial is not clear and colourless, the vial must not be used.
2. Aseptic technique should be used when preparing Spinraza solution for intrathecal administration.
3. The vial should be taken out of the refrigerator and allowed to warm to room temperature (25°C) without using external heat sources, prior to administration.
4. If the vial remains unopened and the solution is not used, it should be returned back to the refrigerator (see section 6.4).
5. Just prior to administration, remove the plastic cap and insert the syringe needle into the vial through the centre of the over-seal to remove the appropriate volume. Spinraza must not be diluted. The use of external filters is not required.
6. Once drawn into the syringe, if the solution is not used within 6 hours, it must be discarded.
7. Any unused product or waste material must be disposed of in accordance with local requirements.