Regulation & Policy

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.

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.

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.

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.

The world’s first regulatory approval of a gene-edited therapy, Casgevy (exagamglogene autotemcel), was issued by the UK Medicines and Healthcare products Regulatory Authority (MHRA) on 16 November, paving the way for the launch in the UK of a new treatment for  sickle cell disease and transfusion-dependent beta thalassaemia. Both diseases are inherited blood disorders that affect red blood cells which are essential for carrying oxygen to all organs and tissues in the body.

The first vaccine for the prevention of disease caused by the chikungunya virus was approved by the US Food and Drug Administration on 9 November – the result of years of research and development by the French company Valneva SE. The vaccine, Ixchiq, was given an accelerated approval on evidence of its ability to arrest the spread of chikungunya virus disease, an emerging global health threat.

Switzerland-based MetrioPharm AG has received a rare paediatric disease designation from the US Food and Drug Administration for its candidate therapy for Duchenne muscular dystrophy (DMD). The drug, MP1032, is a small molecule immune modulator that targets dysregulated mitochondrial metabolism in macrophages. In a preclinical study it was able to increase muscle strength comparable to the corticosteroid prednisolone, but without the corticosteroid’s serious side effects.

Sarepta Therapeutics Inc disclosed top-line results on 30 October from a Phase 3 confirmatory study of its gene therapy for Duchenne muscular dystrophy (DMD) which showed statistical significance for key secondary endpoints, but failed to meet the primary endpoint on a measure of motor function. The drug, Elevidys (delandistrogene moxeparvovec), was given an accelerated approval on 22 June 2023 based on a surrogate endpoint. The newest study was intended to confirm this clinical benefit.

Bimzelx (bimekizumab), a monoclonal antibody that inhibits two cytokines driving inflammation, has been approved in the US for the treatment of moderate to severe plaque psoriasis. The Food and Drug Administration cleared the drug for use on 18 October, more than a year after issuing a complete response letter to the developer, UCB SA of Belgium. The CRL was related to certain “pre-approval inspection observations,” according to the company.

The European Medicines Agency has given a positive opinion to a new drug for Duchenne muscular dystrophy (DMD), a genetic disorder primarily affecting males, which can lead to progressive muscle degeneration and weakness. The developer is Santhera Pharmaceuticals AG of Switzerland. The drug, Agamree (vamorolone), is a small molecule anti-inflammatory medicine that is being positioned as an alternative to corticosteroids, the current standard of care.