The US Food and Drug Administration has relaxed some rules for chimeric antigen receptor (CAR) T cell therapies in order to minimise the tasks that healthcare providers are required to perform. From 26 June, rules known as Risk Evaluation and Mitigation Strategies (REMS) will be eliminated for six autologous CAR T cell therapies which are on the market for cancer indications. The therapies are directed against the CD19 and B-cell maturation antigens.
A new stem cell therapy has been recommended for conditional approval by the European Medicines Agency for patients with a blood cancer who need a transplant following chemotherapy and/or radiotherapy but have no suitable donors. The allogeneic product, Zemcelpro, is derived from umbilical cord blood and contains two components. These are expanded CD34+ cells and unexpanded CD34- cells, each derived from the same cord blood unit.
A small molecule drug that has shown benefits for patients with a type of non-alcoholic liver disease is being recommended for a conditional approval in Europe following a similar decision in 2024 by the US Food and Drug Administration. The drug, resmetirom, works by activating the thyroid hormone receptor-beta which is expressed in the liver, kidneys and pituitary gland and regulates metabolic activity. Reduced levels of thyroid hormones, particularly associated with a reduced activity of the receptor, can lead to a build-up of fat in the liver.
A long-acting treatment for HIV, first approved in the US in 2022, has returned to the market with a new indication – this time as a prophylactic that only needs to the administered twice per year. The drug, lenacapavir, is a synthetic compound that interferes with the life cycle of the HIV virus by binding to the virus’ capsid and inhibiting its replication at multiple stages of its lifecycle. Developed by Gilead Sciences Inc, lenacapavir was first approved as Sunlenca in 2022 for people who had been heavily treated with HIV drugs and become resistant.
The European Medicines Agency is to update the side effect profile for semaglutide following evidence from epidemiological studies that use of the drug is associated with a very rare eye condition known as non-arteritic anterior ischaemic optic neuropathy (NAION). This may cause loss of vision. The decision was announced on 6 June and will affect the labelling of three approved semaglutide products, Ozempic, Rybelsus and Wegovy. All three are produced by Novo Nordisk A/S and are on the market in the EU.
People suspected of having Alzheimer’s disease will in future be able to have another option for receiving a confirmed diagnosis following the approval of a new blood test by the US Food and Drug Administration. The approval, announced on 16 May, is for an in vitro diagnostic device, the first of its kind. It is called the Lumipulse G pTau217/beta-amyloid 1-42 plasma ratio. The developer is Fujirebio Diagnostics Ltd of Japan.
Efpia, the federation that represents pharmaceutical companies in Europe, has deferred comment on President Donald Trump’s executive order on 12 May which is intended to lower the prices of prescription drugs in the US. The order would introduce the Most Favoured Nation policy to drugs. This is a principle of the World Trade Organization under which all nations treat their trading partners equally. In the context of the Trump order, it is being interpreted to mean that US drug prices would be tied to those set in other countries.
A new nonsteroidal treatment for Duchenne muscular dystrophy (DMD) has been recommended in Europe for a conditional marketing authorisation to treat patients from the age of six. The drug, Duvyzat (givinostat), was approved by the US Food and Drug Administration in 2024. It was given a positive opinion by the European Medicines Agency on 25 April. Duvyzat is an oral suspension to be taken at the same time as corticosteroid, the current standard of care. The EMA is recommending the drug for patients who can still walk.
The European Commission has disclosed a list of potential countermeasures against tariffs imposed by the US should trade negotiations between the two parties fail to reach a mutually beneficial outcome. The measures were submitted to companies and other stakeholders for review on 8 May.
Scientific research across all sectors in the EU will receive more financial support under a plan announced on 5 May by Ursula von der Leyen, president of the European Commission. Speaking at an event at the Sorbonne University in Paris, President von der Leyen outlined four new policies to support research. The first is €500 million in new research funding covering the period from 2025 to 2027. Under this programme, it will be possible for researchers to apply for a new ‘super grant’ giving them seven years of financial support.