Regulation & Policy

The Food and Drugs Authority (FDA) of Ghana has approved a vaccine developed by the University of Oxford, UK, to prevent malaria – the first regulatory clearance for the product globally. The vaccine, R21/Matrix-M, is a subunit vaccine made up of a protein secreted by the malaria parasite and fused to a fragment of the hepatitis B virus. It is delivered alongside a plant-based adjuvant that enhances a person’s immune response. The adjuvant was developed by Novavax Inc of the US. The vaccine is being manufactured and scaled up by the Serum Institute of India PvT Ltd.

Pharming Group NV has won US regulatory approval for Joenja (leniolisib), a small molecule drug in-licensed from Novartis for the treatment of a rare genetic disorder that impairs the immune system. The disorder is activated phosphoinositide 3-kinase delta syndrome (APDS) which is estimated to affect one to two people per million and can lead to infections in the ears, sinuses and the upper and lower respiratory tracts. Infections usually begin in infancy. Patients with the disease are also susceptible to blood cell cancers like lymphoma, according to the Food and Drug Administration.

The Roche Group has enlisted the support of Eli Lilly and Company to complete development of a new blood test for the early diagnosis of Alzheimer’s disease. The test has received a breakthrough designation from the US Food and Drug Administration and if approved, would be an additional tool for identifying the low likelihood of amyloid pathology in patients with symptoms of Alzheimer’s disease. It could help determine whether these patients should be tested further.

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. 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.