MHRA approves first medicine for rare immune disease APDS (2024)

APDS is an inherited disorder where the patient is unable to fight infections because the immune system (the body’s natural defences) does not work properly.

The main symptoms usually occur in the first two years of life and include repeated lung infections and a failure to grow and develop normally.

APDS is a long-term debilitating and life-threatening condition due to repeated lung infections that can lead to bronchiectasis (enlargement and inflammation of part of the airways). Patients with APDS are more prone to develop blood cell cancers, like lymphoma.

The recommended dose is 1 tablet twice daily, approximately 12hours apart in adults and adolescents from 12 years weighing 45kg or more.

This is also the first time the MHRA has approved a new medicine following an approval by the US Food and Drug Administration FDA. This is because the medicine was approved via a fast-track approval process for medicines, known as the International Recognition Procedure (IRP).

This allows the MHRA to consider the expertise and decision-making of trusted regulatory partners for the benefit of UK patients. In this case, the regulatory partner was the FDA. As a sovereign regulator, the MHRA retains ultimate authority to accept or reject applications submitted under the IRP, ensuring that all medicines meet safety, quality and effectiveness standards to be licensed in the UK.

The MHRA considered the assessment made by the US regulator as part of its own review, facilitating a rapid approval process.

Julian Beach, MHRA Interim Executive Director of Healthcare Quality and Access, said:

We are focused on providing UK patients access to safe and effective medicines in the shortest possible time.

Approval of leniolisib through the IRP means we have facilitated access for people suffering from this serious disease to an effective medicine to fight infections and reduce symptoms of the disease.

This approval means the appropriate regulatory standards of safety, quality and effectivenessof this new medicine have been met.

As with all medicines, we will keep its safety under close review.

The MHRA’s approval of the medicine is supported by evidence from a placebo-controlled study, carried out over 12-weeks. This study was blind and randomised. Thirty-one adult and paediatric patients participated.

During the study, 21 patients received 70 mg of leniolisib and 10 received placebo twice daily. The co‑primary efficacy endpoints were improvement in the reduction in the size of lymph nodes and the normalisation of immunophenotype.

By day 85 of the study, patients taking leniolisib saw a reduction in lymph node size and a correction of the underlying immune defect.

As with any medicine, the MHRA will keep the safety and effectiveness of leniolisib under close review.

A full list of all side effects reported with this medicine are available in the patient information leaflet or from the product information published on the MHRA website.

If a patient experiences any side effects, they should talk to their doctor, pharmacist, or nurse. This includes any possible side effects not listed in the product information leaflets.

Anyone who suspects they are having a side effect from this medicine is encouraged to talk to their doctor, pharmacist or nurse and report it directly to the MHRA’s Yellow Card scheme.

ENDS

Notes to editors 

  • The marketing authorisation was granted for leniolisib (Joenja) on 26 September 2024 to Pharming Technologies B.V via the International Recognition Procedure.
  • The IRP is open to applicants that have already received an authorisation for the same product from one of MHRA’s specified trusted regulators. These are the regulatory authorities from Australia, Canada, the European Union, Japan, Switzerland, Singapore and the United States.
  • Applications under the International Recognition Procedure (IRP) can be received via the MHRA website. Time horizons for authorisations are set at 60 to 110 days – considerably shorter than the current 150-day time horizon for applications. To further streamline the IRP application process, the MHRA launched an ‘Eligibility Checker’ tool on 20 November 2023.
  • More information can be found in the Summary of Product Characteristics and Patient Information leaflets which will be published on the MHRA Products website within 7 days of approval.
  • The MHRA is an executive agency of the Department of Health and Social Care.
  • The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe.  All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks.
  • For media enquiries, please contact the newscentre@mhra.gov.uk, or call on 020 3080 7651.
MHRA approves first medicine for rare immune disease APDS (2024)
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