Regulation & Policy

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.

Susan Monarez was dismissed as director of the US Centers for Disease Control and Prevention (CDC) on 28 August in a surprise move that also led to the resignations of other top officials including Debra Houry, the agency’s chief medical officer. Robert F Kennedy Jr, secretary of the Department of Health and Human Services (HHS), announced Dr Monarez’s departure on the social media site X. This was subsequently confirmed by the White House.

The US Food and Drug Administration has suspended the licence of a vaccine developed by Valneva SE to prevent chikungunya virus infection following 32 serious adverse event reports, including 21 hospitalisations and three deaths. The vaccine, Ixchiq (chikungunya vaccine, life), was given an accelerated approval by the FDA in November 2023 and since then has been approved in several other countries, some of which are also reviewing patient data. Valneva is expected to start a post-marketing study of the vaccine on 1 October to further assess the risks.

Efpia, the European federation of pharmaceutical industries and associations, has criticised a new US-EU trade agreement as potentially harmful to patients and the European pharma industry and called for exemptions for innovative medicines. The Efpia statement was issued on 21 August shortly after the European Commission published details of the framework agreement setting a maximum, all-inclusive tariff of 15% for the majority of EU exports to the US, including pharmaceuticals. The exact pharma rate is still to be determined but it would not exceed the 15% ceiling.

The European Commission has expressed confidence that a recent trade agreement between the EU and US will provide stability and predictability for the two parties even as the full details are still to be worked out. In a briefing for journalists on 7 August, a Commission spokesperson said the agreement is the best result possible given the challenging circumstances. This is because it creates a uniform tariff of 15% across multiple product groups, down from an initial 30% demanded by the US.

Sarepta Therapeutics Inc, developer of the gene therapy Elevidys (delandistrogene moxeparvovec) for Duchenne muscular dystrophy, faced a further setback on 18 July after  the US Food and Drug Administration was reportedly poised to stop all shipments of the therapy. This followed the deaths of three patients with the disease. Two of the deaths were reported early in the year and involved young people. The third, disclosed on 18 July, involved a 51-year-old man, according to the Bloomberg news service.

The US Food and Drug Administration has relaxed some rules for chimeric antigen receptor (CAR) T cell therapies in order to minimise the tasks that healthcare providers are required to perform. From 26 June, rules known as Risk Evaluation and Mitigation Strategies (REMS) will be eliminated for six autologous CAR T cell therapies which are on the market for cancer indications. The therapies are directed against the CD19 and B-cell maturation antigens.

A new stem cell therapy has been recommended for conditional approval by the European Medicines Agency for patients with a blood cancer who need a transplant following chemotherapy and/or radiotherapy but have no suitable donors. The allogeneic product, Zemcelpro, is derived from umbilical cord blood and contains two components. These are expanded CD34+ cells and unexpanded CD34- cells, each derived from the same cord blood unit.