PRAC January 2023 - recommendations for Janus kinase (JAK) inhibitors and information about Zolgensma

date: 24/01/2023

Alignment of dose recommendations for Janus kinase (JAK) inhibitors in patients with certain risk factors
EMA’s safety committee (PRAC) has further reviewed measures to minimise the risk of serious side effects associated with Janus kinase (JAK) inhibitors used to treat several chronic inflammatory disorders.

The PRAC has recommended the use of a lower dose of Olumiant (baricitinib) for patients at higher risk of blood clots, cardiovascular conditions and cancer in line with the dosing recommendations for other JAK inhibitors subject to the review: Rinvoq (updacitinib), Cibinqo (abrocitinib) and Jyseleca (filgotinib). 

More information on the review is available here

The following immunomodulatory drugs, belonging to the pharmacological class of protein kinase inhibitors of the Janus Kinase (JAK) family, are authorised and on the market in Belgium:

  • Olumiant (with the active substance baricitinib),
  • Jyseleca (with the active ingredient filgotinib),
  • Xeljanz (with the active substance tofacitinib),
  • Rinvoq (with the active ingredient upadacitinib).

New safety information for healthcare professionals
As part of its advice on safety-related aspects to other EMA committees, the PRAC discussed a direct healthcare professional communication (DHPC) containing important information on Zolgensma (onasemnogene abeparvovec).

Zolgensma: fatal cases of acute liver failure
Fatal cases of acute liver failure were recently reported in patients treated with Zolgensma (onasemnogene abeparvovec), a gene therapy medicine for the treatment of spinal muscular atrophy (SMA), a serious rare condition of the nerves that causes muscle wasting and weakness.

This DHPC informs healthcare professionals of the fatal cases of liver failure and the updated recommendations for monitoring liver function, assessing suspected liver injury after infusion and further advice regarding tapering the corticosteroid treatment.

Healthcare professionals should promptly assess patients with worsening liver function tests and/or signs or symptoms of acute liver illness. If patients do not respond adequately to treatment with corticosteroids, treating physicians should consult a paediatric gastroenterologist or hepatologist and consider adjustment of the corticosteroid regimen.

The DHPC for Zolgensma will be forwarded to EMA’s committee for advanced therapies (CAT) and to EMA’s human medicines committee (CHMP). When adopted, the DHPC will be disseminated to healthcare professionals by the marketing authorisation holders, according to an agreed communication plan, and published on the ‘Direct healthcare professional communications’ page and in national registers in European Union (EU) Member States.

Zolgensma is authorised and marketed in Belgium.

More information on the EMA website.

Last updated on 01/03/2023