Regulation & Policy

BC Platforms AG of Switzerland is to collaborate with a US data company to significantly increase its access to patient information for real world evidence studies. The agreement with GeneVault Lifesciences of Cambridge, US, was announced on 18 December and is expected to give the Swiss company new data sources across Asia, the Middle East, Africa and Latin America. These regions represent some of the world’s most genetically diverse yet historically under-studied populations.

GSK Plc has received US approval for a new biologic to treat severe asthma, a disease driven high levels of white blood cells in the airwaves which can lead to inflammation and a shortness of breath. The Food and Drug Administration decision, announced on 16 December, will introduce a new drug to the market requiring dosing only twice per year. Exdensur (depemokimab) is a long-acting interleukin-5 (IL-5) antagonist. It prevents IL-5 from activating its receptor on eosinophils, thereby reducing an overproduction of the white blood cells.

Twelve former commissioners of the US Food and Drug Administration have rebuked proposed changes to vaccine policy at the agency saying that the measures will impede the development of new products to prevent infectious disease. Led by Robert Califf, the commissioners outlined their concerns in an article in the 3 December 2025 edition of The New England Journal of Medicine. Dr Califf was FDA commissioner until January 2025. Altogether, the group represents more than 35 years of FDA oversight and policymaking.

The European Commission launched a comprehensive package of measures in early December designed to realise the full potential of the single market for financial services. The package is a key component of the EU Savings and Investments Union (SIU) strategy, which aims to create a more integrated, efficient and competitive financial system and to support businesses across Europe in accessing funding.

The US Food and Drug Administration has approved a new gene therapy for spinal muscular atrophy (SMA), a neurological disorder that can lead to progressive muscle weakness and in the most severe cases, death. The therapy, Itvisma (onasemnogene abeparvovec), has the same active ingredient as the gene therapy Zolgensma, but formulated at a different concentration. Zolgensma was approved by the FDA in 2019 to treat paediatric patients with SMA. The newest therapy extends the treatment group to older individuals with a confirmed mutation in the survival motor neuron 1 (SMN1) gene.

A novel antibody that has shown it can delay the onset of Type 1 diabetes received a positive opinion from the European Medicines Agency on 14 November. Teizeild (teplizumab) has been developed for the most advanced stage of disease onset when most pancreatic beta cells have been destroyed, insulin production is down, and blood sugar levels are rising. The same product, known as Tzield, was approved by the US Food and Drug Administration in 2022.

A candidate radio-diagnostic has received ‘fast track’ designation from the US Food and Drug Administration for its protential use in combination with a therapeutic to identify and treat clear cell renal cell carcinoma, the most common type of kidney cancer. ITM-94 is a radiolabeled gallium 68 imaging agent which is in a Phase 1/2 trial in patients with locally advanced or metastatic solid tumours. It is being developed with ITM-91, an investigational radioparmaceutical. The combination is known as a theranostic.

A gene therapy for Wiskott-Aldrich syndrome, whose development was supported by the Italian non-profit organisation, Fondazione Telethon ETS, received a positive opinion from the European Medicines Agency on 14 November. Wiskott-Aldrich syndrome is a rare genetic disorder that keeps a child’s immune system from functionally normally. It mainly affects young boys. The treatment, Waskyra (etuvetidigene autotemcel) is the first gene therapy to be evaluated for this disease.

The US Food and Drug Administration has updated a warning about the safety of Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy (DMD), and restricted its use. This follows reports earlier this year of fatal acute liver failure in certain non-ambulatory, male, paediatric patients taking the drug. Following the incidents, the developer, Sarepta Therapeutics Inc, voluntarily paused distribution of Elevidys to this patient group. This has now been formalised in a notice issued on 14 November.

The European Medicines Agency has issued a positive opinion for a new indication of an antibody therapeutic that targets B cells in order to treat immunoglobulin G4 related diseases. These are autoimmune disorders caused when the body’s own defence system attacks normal tissues. This can lead to fibrosis and inflammation in one or multiple organs of the body. The therapy, Uplizna (inebilizumab), has already been approved for the treatment of neuromyelitis optica spectrum disorder, an inflammatory disorder of the central nervous system.