Regulation & Policy

Lecanemab, a monoclonal antibody drug to treat Alzheimer’s disease, was given an accelerated approval by the US Food and Drug Administration on 6 January, based on evidence that it reduced amyloid plaque, a marker for the disease. Known commercially as Leqembi, the drug is only the second antibody treatment to be given a preliminary authorisation to treat the disease, which is an irreversible, progressive brain disorder affecting millions of people. The first was Aduhelm (aducanumab), which also targets amyloid plaque.

The US Food and Drug Administration has approved a gene therapy for bladder cancer, the second authorisation of a novel gene treatment within a month. The new drug, Adstiladrin (nadofaragene firadenovec), is indicated for the treatment of patients with bladder cancer which hasn’t yet invaded the muscle and is unresponsive to the Bacillus Calmette-Guérin (BCG) immunotherapy. The decision comes not long after the agency approved a gene therapy for haemophilia B.

The first faecal transplant therapy derived from the gut microbiome of healthy donors was approved by the US Food and Drug Administration on 30 November, paving the way for regulatory reviews of other products in the sector. The therapy, Rebyota, was developed by Ferring Pharmaceuticals Inc and approved for the prevention of recurrent Clostridioides difficile infection, a potentially life-threatening disease.

The first gene therapy for adults with haemophilia B has been approved by the US Food and Drug Administration after showing that a single dose of the treatment was able to significantly reduce abnormal bleeding.

Haemophilia B is a genetic disease arising from missing or insufficient levels of blood clotting factor IX, a protein that is needed to produce blood clots to stop bleeding. Symptoms can include prolonged or heavy bleeding after an injury or surgery, or spontaneous bleeding without a clear cause. Most people affected by the disorder are men.

The use of antibiotic medicines in animals has dropped sharply in Europe following multiple public policy initiatives and national campaigns, according to the European Medicines Agency. In a report issued on 18 November, the agency said that sales of veterinary antibiotics in animals decreased by 47% in the ten years to 2021 – the lowest level ever reported. The report is based on data from 25 European countries.

A safety review of the Janus kinase (JAK) inhibitor group of medicines for chronic inflammatory disorders has concluded that the drugs should only be used in patients with certain risk factors if no suitable alternatives exist. The review was conducted by the pharmacovigilance risk assessment committee of European Medicine Agency and its conclusions were endorsed on 11 November by the agency’s main scientific committee, the CHMP. The recommendations now go to the European Commission for formal approval.

The European Medicines Agency has launched a new project to help academic scientists and those working for non-profit organisations understand the regulatory requirements for developing and eventually commercialising cell, gene and tissue engineered therapies. The project complements the agency’s priority medicines scheme, PRIME, which was launched in 2016 to provide support to developers of medicines that meet a major public health need.

The US Food and Drug Administration has approved a new drug for amyotrophic lateral sclerosis, a rare disease that attacks and kills the nerve cells that control voluntary muscles. In a Phase 2 trial, the drug Relyvrio (sodium phenylbutyrate and /taurursodiol), showed an ability to slow the rate of neuromuscular decline. Moreover in a follow-up study, it extended patient survival compared with a placebo.

A new treatment designed to protect infants from infection from the respiratory syncytial virus (RSV) has received a positive opinion from the European Medicines Agency. The accelerated review was in response to evidence that infections from RSV are on the rise following a relaxation of restrictions that have been in place to prevent Covid-19.

The European Medicines Agency has recommended approval of a bivalent vaccine to protect against two sub-variants of the SARS-CoV-2 virus. The authorisation is for an updated booster shot developed by Pfizer Inc and BioNTech SE to protect people against the omicron subvariants BA.4 and BA.5 in addition to the original strain of SARS-CoV-2. The new vaccine is to be used in people aged 12 years and older who have received at least a primary course of vaccination to prevent Covid-19.