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The Medicines and Healthcare products Regulatory Agency (MHRA) has on Friday approved leniolisib phosphate (Joenja) to treat a rare immune disease known as activated phosphoinositide 3-kinase delta syndrome or APDS in adults and adolescents aged 12-years-old and older who weigh 45kg or more. The medicine was approved via a fast-track approval process for medicines, known as the International Recognition Procedure (IRP), which allows the MHRA to consider the expertise and decision-making of trusted regulatory partners for the benefit of UK patients. The decision follows an approval for the drug by the US Food and Drug Administration (FDA). This is the first time the MHRA has approved a new medicine following FDA approval. The MHRA said it considered the assessment made by the US regulator as part of its own review, facilitating a rapid approval process. APDS is an inherited disorder where the patient is unable to fight infections because the immune system 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.

British health agencies have secured funding to develop a standardised approach to test the performance of vaccines being used or in development against monkeypox, days after the World Health Organization labelled the growing outbreak a global health emergency. The Coalition for Epidemic Preparedness Innovations (CEPI) said it would give up to $375,000 to the Medicines and Healthcare products Regulatory Agency (MHRA) and Health Security Agency (UKHSA) to develop standard tools to assess the strength and duration of immune responses generated by current vaccines, and for tests used to detect monkeypox antibody levels. Apart from administration fees, these tools will be made freely available to the global scientific community, paving the way for a common standardised assessment between countries documenting vaccine performance against monkeypox, CEPI said. Until this year, the viral disease has rarely spread outside Africa where it is endemic. But reports of a handful of cases in Britain in early May signalled that the outbreak had moved into Europe. So far, there have been more than 16,000 confirmed cases of monkeypox in more than 75 countries.