Regulation & Policy

Trodelvy (sacituzumab), an antibody drug-conjugate first approved for triple-negative breast cancer, has had its label expanded by the US Food and Drug Administration to include the treatment of metastatic hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative breast cancer. The developer, Gilead Sciences Inc, said the approval was based on statistically significant and clinically meaningful progression-free survival and overall survival data from a Phase 3 study.

The US Food and Drug Administration has approved the first oral treatment for anaemia caused by chronic kidney disease in adults. The drug, Jesduvroq (daprodustat), was developed by GSK Plc and works by increasing the level of the hormone erythropoietin which signals the body to make red blood cells. These cells are deficient in anaemia. Jesduvroq has been approved for adults who have been on dialysis for at least four months. It is not authorised for patients who are not on dialysis. Other treatments approved in the US for this condition are injected into the blood or under the skin.

The US Food and Drug Administration has given a full approval to the cancer treatment Jemperli (dostarlimab), having received follow-up data confirming the drug’s effectiveness in treating endometrial cancer. Jemperli was given an accelerated approval in August 2021 for adult patients with mismatch repair-deficient recurrent or advanced solid tumours. Mismatch repair-deficient tumours contain abnormalities that affect the proper repair of DNA when copied in a cell.

The US Food and Drug Administration has issued a complete response letter (CRL) to Eli Lilly and Company for its application for an accelerated approval of the investigative treatment for Alzheimer’s disease donanemab. A CRL is a formal notice to a company in which the agency explains why it cannot accept an application for a drug approval, usually citing deficiencies in the application itself. Donanemab is a monoclonal antibody that targets beta amyloid plaque in the brain for the treatment of patients with early symptomatic Alzheimer’s disease.

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.