Zolgensma 2 × 1013 vector genomes/mL solution for infusion * Pharmacy Only: Prescription DHPC
Company:
Novartis Ireland LimitedStatus:
UpdatedLegal 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
- SPC
- Patient Leaflets
- Ed Material - HCP
- Ed Material - Patient
- Additional Comms
- Licence Info
- Doc History
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
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
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).
Updated on 20 February 2023
File name
63f3825815244.pdf
Reasons for updating
- Add New Doc
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