Regulation & Policy

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.

Britain’s plans for expanding its position as a global centre for biopharmaceutical research and development hit a barrier on 10 September when Merck & Co Inc announced a decision to stop development of a £1 billion research centre in London, and vacate two other laboratories in the city.

Genmab A/S has received a ‘breakthrough therapy’ designation from the US Food and Drug Administration for an antibody-drug conjugate (ADC) which is in development for endometrial cancer and has shown promising activity in heavily pre-treated patients. The designation was announced on 26 August, just weeks after the company presented new data  at the 2025 American Society of Clinical Oncology annual meeting in Chicago, US. The drug, rinatabart sesutecan, showed a 50% objective response rate and two complete responses in heavily pre-treated patients with advanced disease.

Food and Drug Administration Commissioner Marty Makary has announced plans to update the regulation of pharmaceutical advertising in the US in order to prevent deceptive messages being sent to patients by developers. On 9 September, the agency issued warning letters to pharmaceutical companies to remove misleading advertisements. Approximately 100 cease-and desist letters were sent to companies judged to have issued misleading ads.

Google LLC, the US technology conglomerate owned by Alphabet Inc, has been fined €2.95 billion by the European Commission for what it described as abusive practices in its online advertising business. The fine relates to practices dating from 2014 in which Google was found to have favoured its own online display advertising technology services, to the detriment of other providers.