Zolgensma 2 × 1013 vector genomes/mL solution for infusion *
Pharmacy Only: Prescription DHPC

  • Company:

    Novartis Ireland Limited
  • Status:

    Updated
  • Legal Category:

    Product subject to restricted prescription (C)
  • Active Ingredient(s):

    This medicinal product is subject to additional monitoring.

    *Additional information is available within the SPC or upon request to the company

Updated on 12 October 2023

File name

6527c235dd2ce.pdf

Reasons for updating

  • Change to section 4.4 - Special warnings and precautions for use

Legal category:Product subject to restricted prescription (C)

Free text change information supplied by the pharmaceutical company

Section 4.4 updated to include a theoretical risk of tumourigenicity due to integration of AAV vector DNA into the genome.

Updated on 12 October 2023

File name

6527c1d7c932b.pdf

Reasons for updating

  • Change to section 2 - what you need to know - warnings and precautions

Free text change information supplied by the pharmaceutical company

Risk of tumours associated with potential insertion into the DNA

EDM Updated on 31 July 2023

File name

64c7be5646431.pdf

Reasons for updating

  • Add New Doc

Free text change information supplied by the pharmaceutical company

First version of the HCP guide

EDM Updated on 31 July 2023

File name

64c7bd87449e3.pdf

Reasons for updating

  • Replace File

Free text change information supplied by the pharmaceutical company

Approval of new version of this material

Updated on 31 May 2023

File name

647728c2c0f7f.pdf

Reasons for updating

  • Change to section 6.3 - Shelf life

Legal category:Product subject to restricted prescription (C)

Free text change information supplied by the pharmaceutical company

Shelf life changed from 2 years to 18 months

Updated on 09 March 2023

File name

6409e12107dda.pdf

Reasons for updating

  • Change to section 2 - what you need to know - warnings and precautions
  • Change to section 4 - possible side effects

Free text change information supplied by the pharmaceutical company

Information added to 'the 'Liver problems', 'Infection' and 'Abnormal clotting of blood in small blood vessels (thrombotic microangiopathy' sections of the "Warning and Precautions".

Liver failure added as a possible side effect

Updated on 09 March 2023

File name

6409c1e30a1e0.pdf

Reasons for updating

  • Change to section 4.2 - Posology and method of administration
  • Change to section 4.4 - Special warnings and precautions for use
  • Change to Section 4.8 – Undesirable effects - how to report a side effect

Legal category:Product subject to restricted prescription (C)

Free text change information supplied by the pharmaceutical company

Section 4.2:

  • Pre-treatment laboratory testing updated to include albumin, prothrombin time, partial thromboplastin time (PTT) and international normalised ratio (INR) in the liver function tests
  • Include recommendation that patients are clinically stable in their overall health status prior to infusion
  • Liver function should be monitored before treatment and regularly for at least 3 months after infusion. Promptly assess patients with worsening liver function tests and/or signs or symptoms of acute illness.
  • Patients with positive serology for hepatitis B or hepatitis C have not been studied in clinical studies with onasemnogene abeparvovec.


Section 4.4

Following information added to ‘Hepatotoxicity’:

  • Fatal cases of acute liver failure have been reported in patients treated with onasemnogene abeparvovec.
  • Promptly assess patients with worsening liver function tests and/or signs or symptoms of acute illness.
  • If patients do not respond adequately to corticosteroids, consult a pediatric gastroenterologist or hepatologist and consider adjustment of the corticosteroid regimen.
  • AST/ALT/total bilirubin should be assessed weekly for the first month after onasemnogene abeparvovec infusion and during the entire corticosteroid taper period
  • Corticosteroids should not be tapered until liver function tests become unremarkable (normal clinical examination, total bilirubin, and ALT and AST levels below 2 × ULN).


Following information added to ‘Thrombotic microangiopathy’ (TMA)

  • Cases generally occurred within the first two weeks after onasemnogene abeparvovec infusion. TMA is an acute and life-threatening condition, which is characterised by thrombocytopenia and microangiopathic haemolytic anaemia. Fatal outcomes have been reported.
  • In case of thrombocytopenia, further evaluation including diagnostic testing for haemolytic anaemia and renal dysfunction should be undertaken promptly.


Following information added to ‘Systemic immune response’ (‘Immunomodulatory regimen’ renamed)

  • Due to the increased risk of serious systemic immune response, it is recommended that patients are clinically stable in their overall health status (e.g. hydration and nutritional status, absence of infection) prior to onasemnogene abeparvovec infusion
  • Patients with infection were excluded from participation in clinical trials with onasemnogene abeparvovec.
  • Increased vigilance in the prevention, monitoring, and management of infection is recommended before and after onasemnogene abeparvovec infusion.

 

Section 4.8:

  • Fatal cases of liver failure added to table of adverse reactions
  • Post-marketing cases with transient decrease in platelet count to levels <25 x 109/L within two weeks of administration have been reported (see section 4.4).


COM Updated on 20 February 2023

File name

63f3825815244.pdf

Reasons for updating

  • Add New Doc

EDM Updated on 28 November 2022

File name

6384d5b778b64.pdf

Reasons for updating

  • Add New Doc

Free text change information supplied by the pharmaceutical company

First upload to medicines.ie

Updated on 21 November 2022

File name

637b736ece02f.pdf

Reasons for updating

  • Change to section 6.3 - Shelf life

Legal category:Product subject to restricted prescription (C)

Free text change information supplied by the pharmaceutical company

Shelf life increase from 1 to 2 years

Updated on 06 September 2022

File name

63170f5f3c509.pdf

Reasons for updating

  • Change to section 2 - what you need to know - warnings and precautions
  • Change to section 6 - marketing authorisation holder

Free text change information supplied by the pharmaceutical company

Further information regarding low blood-platelet counts (thrombocytopenia): "Most of the reported cases of a low blood-platelet count occurred within the first two weeks after the child was given Zolgensma".

Change in Marketing Authorisation Holder to Novartis Europharm

Updated on 06 September 2022

File name

631709fe4243e.pdf

Reasons for updating

  • Change to section 4.4 - Special warnings and precautions for use
  • Change to section 4.8 - Undesirable effects
  • Change to section 7 - Marketing authorisation holder

Legal category:Product subject to restricted prescription (C)

Free text change information supplied by the pharmaceutical company

SmPC Section 4.4 and 4.8 updated with additonal information regarding thrombocytopenia

SmPC Section 7 updated with Novartis Europharm as new Marketing Authorisation Holder

Updated on 26 May 2022

File name

628f6a3aacaab.pdf

Reasons for updating

  • Change to further information section

Free text change information supplied by the pharmaceutical company

Sentence regarding conditional authorization is removed

Updated on 23 May 2022

File name

628ba0ca29dcb.pdf

Reasons for updating

  • Change to section 4.8 - Undesirable effects
  • Change to section 5.1 - Pharmacodynamic properties
  • Change to section 5.3 - Preclinical safety data

Legal category:Product subject to restricted prescription (C)

Free text change information supplied by the pharmaceutical company

Updates to 4.8, 5.1 and 5.3 as part of renewal procedure

Updated on 23 May 2022

File name

628b9f93f1864.pdf

Reasons for updating

  • Change to section 1 - what the product is used for
  • Change to section 2 - what you need to know - warnings and precautions
  • Change to section 3 - how to take/use
  • Change to section 4 - possible side effects
  • Change to section 5 - how to store or dispose

Free text change information supplied by the pharmaceutical company

Changes due to approval of renewal

Updated on 01 December 2021

File name

Zolgensma REG PIL PF21-122_IPHA_1638348631.pdf

Reasons for updating

  • New PIL for new product

Free text change information supplied by the pharmaceutical company

PIL for new product

Updated on 01 December 2021

File name

Zolgensma REG SPC PF21-292 November 2021 IPHA_1638348519.pdf

Reasons for updating

  • New SPC for new product

Legal category:Product subject to restricted prescription (C)

Free text change information supplied by the pharmaceutical company

SPC for new product