Regulation & Policy

Treatment for rare disease

Country
United States

The US Food and Drug Administration approved a new therapy for a rare genetic disease on 18 March, indicating a widening of the scope of regulatory authorisations where there is a serious unmet medical need. The product, Lenmeldy (atidarsagene autotemcel), is a gene therapy intended as a one-time treatment for children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD). MLD is a rare genetic disease that affects the brain and nervous system.

Drug to treat all genetic variants of DMD

Country
United States

A drug for the treatment of patients with Duchenne muscular dystrophy (DMA) was approved by the US Food and Drug Administration on 22 March. Duvyzat (givinostat) is the first nonsteroidal drug for patients with all genetic variants of the disease. DMD is a rare neurological disorder which causes progressive muscle weakness due to a lack of dystrophin, a muscle protein. Over time, individuals with the disease experience muscle weakness that can lead to problems with walking, breathing and an early death.

New treatment for NASH

Country
United States

The US Food and Drug Administration has approved Rezdiffra (resmetirom), a small molecule agonist of the thyroid hormone receptor beta (THR-beta), to treat non-alcoholic steatohepatitis (NASH) in adults with moderate to advanced liver scarring. The developer is Madrigal Pharmaceuticals Inc. The approval, announced on 14 March, is the first for a product that directly addresses liver scarring and is to be used alongside diet and exercise.

Wegovy approved for heart disease

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.