Regulation & Policy

AbbVie Inc has received a complete response letter (CRL) from the US Food and Drug Administration requesting additional information on a pump that is used to deliver its candidate treatment for Parkinson’s disease, the company announced on 22 March. A CRL is a formal notice from the FDA to a company outlining deficiencies in a new drug application (NDA). The agency did not request additional safety or efficacy studies. AbbVie said it plans to resubmit its regulatory application as soon as possible.

Bank regulators in the US and UK took further steps on 12 March to protect depositors at Silicon Valley Bank in California and its UK subsidiary following the collapse of the parent company the previous Friday. The US Federal Deposit Insurance Corporation (FDIC) acted first on 10 March by closing the parent bank after a run on deposits. Concurrently, it created a new banking entity to protect insured depositors. This move protected customers with accounts of up to $250,000 – the US federally insured level – but it did not stop the contagion.

The European Medicines Agency has issued a report summarising actions that it has taken over the past four years to improve the availability of medicines for children. This activity is regulated by a Paediatric Regulation which came into force in the EU on 26 January 2007. The regulation establishes a special committee to coordinate the agency’s work in this area. The committee in turn decides which studies companies must carry out on children, alongside studies for the same disease that are being investigated in the adult population.

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.

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.