The European Medicines Agency has issued a positive opinion for Fabhalta (iptacopan), a monotherapy for a rare genetic blood disorder which has shown superior activity to the standard of care. The disorder is paroxysmal nocturnal haemoglobinuria (PNH) which causes the premature breakdown of red blood cells in the body leading to fatigue, shortness of breath and often requiring blood cell transfusions.
A group of medicines approved to treat type 2 diabetes and obesity, which include semaglutide, have passed a safety review by the European Medicines Agency. The review was triggered by reports linking use of the drugs, which are glucagon-like peptide-1 receptor agonists (GLP-1), to suicidal and self-harm thoughts and behaviour. On 12 April, the agency’s pharmacovigilance risk assessment committee (PRAC) said an investigation could not establish a causal association between the medicines and the health risks.
Carvykti (ciltacabtagene autoleucel), a chimeric antigen receptor (CAR) T cell therapy, has been given a more prominent position for the treatment of multiple myeloma following a Phase 3 trial showing it reduced the risk of disease progression or death by 59% compared with standard therapies. On 5 April, a new indication as a second-line therapy was approved by the US Food and Drug Administration. This followed a recommendation for an upgrade by the agency’s oncologic drugs advisory committee.
Germany-based TME Pharma NV has received a Fast Track designation from the US Food and Drug Administration for its investigational treatment for glioblastoma giving it access to more regulatory support during the clinical development of the therapy. The drug, NOX-A12, is an RNA oligonucleotide targeting a chemokine protein in the tumour microenvironment with the goal of breaking tumour protection against the immune system.
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.
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.
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.
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.
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.
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.