Regulation & Policy

Wegovy approved for heart diseases

Country
United States

The obesity drug, Wegovy (semaglutide), first approved in 2021 for weight control, has now been authorised in the US to reduce the risk of heart attack and stroke in adults with cardiovascular disease and either obesity or excessive weight. It is the first drug to receive Food and Drug Administration approval for both indications as evidence of the linkage between excessive weight and cardiovascular events becomes clearer.

Amtagvi approved by FDA

Country
United States

A cell therapy for melanoma, the first of its kind, was given an accelerated approval by the US Food and Drug Administration on 16 February for patients whose disease has progressed despite earlier treatment with a checkpoint inhibitor or a personalised cancer therapy. The therapy, Amtagvi (lifileucel), is a tumour infiltrating lymphocyte cell therapy (TIL) which consists of T cells that have been derived from a patient’s own tumour. 

African Medicines Agency

Country
Netherlands

More financial support was announced in late January for the African Medicines Agency (AMA), a regulatory project that will see the 55 countries in the African Union have a dedicated healthcare institution to meet the needs of their combined populations of 1.3 billion people. A treaty formally establishing the agency has been ratified by 27 countries to date. All governments will need to get parliamentary approval of the treaty for the agency to take effect in their countries.

Breakthrough designation for radiotherapy

Country
United States

An early clinical-stage radionuclide therapy for neuroendocrine tumours received a ‘breakthrough therapy designation’ from the US Food and Drug Administration on 12 February – the first targeted alpha therapy to do so. The therapy, AlphaMedix, is being developed by RadioMedix Inc of the US and Orano Med of France to treat patients with somatostatin receptor expressing gastroenteropancreatic neuroendocrine tumours. Neuroendocrine tumours mostly occur in the gastrointestinal tract and pancreas, but also can be found in other tissues such as the thymus.

New drug for eosinophilic esophagitis

Country
Japan

The US Food and Drug Administration has approved a new drug for the treatment of eosinophilic esophagitis (EoE), a chronic, immune-mediated inflammatory disease that can cause difficulty swallowing and affect individuals of any age. The drug, Eohilia, is an oral suspension of budesonide which was developed by Takeda Pharmaceutical Co Ltd and shown to be effective in two trials of adult and paediatric patients. The drug was compared with a placebo and administered for a period of 12 weeks. The first study enrolled patients 11 to 56 years of age and the second, 11 to 42 years of age.

FDA comments on CAR T risks

Country
United States

Healthcare professionals are being advised to monitor patients and clinical trial participants who receive treatment for cancer with a chimeric antigen receptor (CAR) T cell therapy for the risk of secondary malignancies. In a commentary in The New England Journal of Medicine on 25 January, Nicole Verdun and Peter Marks write that while secondary cancers from these medicines appear to be relatively rare, they need to be identified. Drs Verdun and Marks are both senior officials at the US Food and Drug Administration. 

CAR T therapy malignancies

Country
United States

A chimeric antigen receptor (CAR) T cell therapy approved by the US Food and Drug Administration in 2022 for multiple myeloma has been given a boxed warning by the agency after patients treated with the drug developed secondary malignancies. The product, Carvykti (ciltacabtagene autoleucel), consists of a patient’s T cells that have been engineered to attach to the B cell maturation antigen (BCMA) on multiple myeloma calls and kill them. The FDA approved the therapy on the basis of deep and durable responses by patients taking the therapy.

Drug for desmoid tumours

Country
United States

The US Food and Drug Administration has approved a new drug for the treatment of desmoid tumours, a rare subtype of soft tissue sarcoma. Desmoid tumours are non-cancerous and typically treated with surgery, but not all patients are eligible for this procedure. And even when surgery is an option there is a risk that the tumours will return, affecting a person’s quality of life, according to the FDA.

Two gene therapy approvals

Country
United States

The US Food and Drug Administrations has issued approvals for two new cell-based gene therapies for the treatment of sickle cell disease, a rare but life-threatening blood disorder that affects millions of people throughout the world. The approvals, announced on 8 December, are for treatments which have demonstrated an ability to restore haemoglobin function in patients using different mechanisms of action.

EU reaches political agreement on AI

Country
Belgium

Political leaders of the European Union reached an agreement on 9 December on the principles that should govern the regulation of artificial intelligence – the first comprehensive legal framework for AI anywhere in the world.