Regulation & Policy

A vaccine developed by GSK Plc to protect older adults against infection from the respiratory syncytial virus (RSV) has been approved in the US and given a positive opinion in the EU – the first such treatment to be approved anywhere in the world. The vaccine, Arexvy, will be launched in the US before the next RSV season which typically starts in the autumn and peaks in the winter. Launch in Europe is expected to follow a formal decision on marketing authorisation, which will be taken by the European Commission.

Health technology assessment (HTA) bodies in the US, Canada and the UK have reached a consensus about how confidential clinical data should be handled in their public assessments of new medicines. Each of the groups operates in a different healthcare environment, but they agree that more transparency is required in the future.

US regulatory approval has been given to MaaT Pharma SA of France to start a Phase 3 trial of an experimental microbiome therapy in patients with graft-versus-host-disease, a potential complication from an allogeneic stem cell transplantation. The therapy, MaaT013, is an enema formulation consisting of samples of faeces from multiple healthy donors. The goal of the treatment is to restore gut microbiome diversity in order to help the body fight the disease.

The US Food and Drug Administration has approved the first oral microbiota product to prevent the recurrence of Clostridioides difficile (C. difficile) infection in individuals suffering repeat infection after treatment with an antibiotic. The product is made from human faeces. This is the second microbiota product targeting C. difficile infection to get a US approval in just five months. The first was a faecal transplant therapy developed by Ferring Pharmaceuticals Inc.

The US Food and Drug Administration has approved a new cell therapy to quicken the recovery of neutrophils, a subset of white blood cells in the body, and reduce the risk of infection in patients being treated for blood cancers. Called Omisirge (omidubicel), the product was developed by Gamida Cell Ltd of Israel.

A project initiated by the European Medicines Agency in 2022 to collect data on healthcare, disease prevalence, and the use of medicinal products, is entering its second year with plans to quadruple the number of studies. The project looks at real-world data, or data on a patient’s health from sources other than clinical trials, and real-world evidence, which is the analysis of this data.

The Food and Drugs Authority (FDA) of Ghana has approved a vaccine developed by the University of Oxford, UK, to prevent malaria – the first regulatory clearance for the product globally. The vaccine, R21/Matrix-M, is a subunit vaccine made up of a protein secreted by the malaria parasite and fused to a fragment of the hepatitis B virus. It is delivered alongside a plant-based adjuvant that enhances a person’s immune response. The adjuvant was developed by Novavax Inc of the US. The vaccine is being manufactured and scaled up by the Serum Institute of India PvT Ltd.

Pharming Group NV has won US regulatory approval for Joenja (leniolisib), a small molecule drug in-licensed from Novartis for the treatment of a rare genetic disorder that impairs the immune system. The disorder is activated phosphoinositide 3-kinase delta syndrome (APDS) which is estimated to affect one to two people per million and can lead to infections in the ears, sinuses and the upper and lower respiratory tracts. Infections usually begin in infancy. Patients with the disease are also susceptible to blood cell cancers like lymphoma, according to the Food and Drug Administration.

The Roche Group has enlisted the support of Eli Lilly and Company to complete development of a new blood test for the early diagnosis of Alzheimer’s disease. The test has received a breakthrough designation from the US Food and Drug Administration and if approved, would be an additional tool for identifying the low likelihood of amyloid pathology in patients with symptoms of Alzheimer’s disease. It could help determine whether these patients should be tested further.

AbbVie Inc has received a complete response letter (CRL) from the US Food and Drug Administration requesting additional information on a pump that is used to deliver its candidate treatment for Parkinson’s disease, the company announced on 22 March. A CRL is a formal notice from the FDA to a company outlining deficiencies in a new drug application (NDA). The agency did not request additional safety or efficacy studies. AbbVie said it plans to resubmit its regulatory application as soon as possible.