Patients with polycythaemia vera, a rare blood disorder that causes the body to make too many red blood cells, will be eligible for treatment with a new peptide therapeutic approved by the US Food and Drug Administration on 28 August. Mimrylo (rusfertide) is a hepcidin mimetic that works by mimicking the action of hepcidin, a hormone that regulates the body’s use of iron. Too little iron can lead to iron-deficiency anaemia while too much can be toxic.
The US Food and Drug Administration approved a new treatment for dermatomyositis on 27 August – the first oral drug for a disease that can cause progressive damage to the muscles, skin and lungs and an impaired quality of life. Existing treatments include combinations of chronic steroids and antirheumatic drugs which have not been effective in controlling the disorder. The new treatment, Lisraya (brepocitinib), works as a Janus kinase inhibitor, blocking pathways that play a key role in the body’s immune responses.
A new small molecule drug has won approval from the US Food and Drug Administration to treat patients with metastatic pancreatic ductal adenocarcinoma – an aggressive cancer with a poor prognosis for survival. The drug, Rasonque (daraxonrasib), has been authorised for adults who have received at least one prior systemic therapy for their cancer, or who are not candidates for multiagent systemic therapy.
The US Food and Drug Administration granted an accelerated approval on 19 August for a new gene therapy for patients with an inherited disorder caused by the shortage of an enzyme needed to maintain stable blood sugar levels in the body. The therapy, Genglycos (pariglasgene brecaparvovec), is the first treatment to be authorised for the disorder – glycogen storage disease type la. Also called von Gierke disease, the disorder results in the build-up of glycogen, a complex sugar, in the body’s cells.
On 4 August, the European Commission completed the final legal steps for the launch of the Scaleup Europe Fund, a public-private partnership that will invest in European companies with potential to compete globally in advanced technologies. The fund is being launched with a target capital of €5 billion. The fund’s first investment, a day after launch, was in the Finnish space technology company ICEYE.
In one of its first policy measures, the new UK government led by prime minister Andy Burnham announced plans on 21 July to merge the department which oversees science and technology with that for business and trade. The new department will be called the Department for Business, Innovation, Science and Trade. It will be led by Jonathan Reynolds, a former business secretary.
The US Food and Drug Administration has approved a new oral treatment for adults with hypercholesterolaemia – a condition characterised by abnormally high levels of cholesterol which increase the risk of a heart attack or stroke. The treatment, Lipfendra (enlicitide), is a macrocyclic peptide that inhibits the enzyme PCSK9, which plays a role in cholesterol metabolism.
The US Food and Drug Administration has approved a new indication for Casgevy, a cell-based gene therapy for two blood disorders, making it available to individuals as young as two years. The disorders are sickle cell disease, a rare inherited blood disorder, and transfusion-dependent beta thalassaemia. Casgevy (exagamglogene autotemcel) was first approved by the FDA in 2023 for children with sickle cell disease from the age of 12 years.
A new study on the economic impact of pharmaceutical innovation in Europe shows sustained benefits across society, according to EFPIA, the European industry federation. Data from the study, released on 22 June, shows that between 2014 and 2024 Europe’s investment of €11.67 billion in new medicines returned more than five times the amount in social, economic and hospital cost savings – equating to €66 billion in total. This figure includes more than €9 billion in direct hospital savings. Key findings include:
The US Food and Drug Administration issued new draft guidance on 29 May about how companies can reduce the use of animals in drug testing – this time for cancer medicines. Entitled, Oncology Pharmaceuticals: Streamlined Nonclinical Safety Studies for Biologics and Conjugated Products, the guidance recommends new criteria for general toxicology studies, including when animal testing may be unnecessary because there is no binding or pharmacologic activity.