Regulation & Policy

The European Medicines Agency is reviewing the safety of three medicines used for the treatment of weight loss and type 2 diabetes in light of reports from the Icelandic Medicines Agency of suicidal thoughts and attempted self-injury amongst patients taking the drugs. The review will focus on the glucagon-like peptide 1 (GLP-1) receptor agonists liraglutide (Saxenda) and semaglutide (Ozempic and Wegovy), all three of which have been developed by Novo Nordisk A/S. The investigation will involve an analysis of 150 reports of possible self-harm and suicidal thoughts.

The first gene therapy to treat severe haemophilia A was approved by the US Food and Drug Administration on 30 June, adding a novel treatment to the existing group of drugs for the bleeding disorder. The therapy, Roctavian, is an adeno-associated virus (AAV) vector-based therapy intended to be used as a one-time treatment. It is administered as a single dose by intravenous infusion.

The US Food and Drug Administration approved the first allogeneic cell therapy for type 1 diabetes on 28 June, making it possible for people with severe hypoglycaemia to receive an additional treatment for achieving targeted blood glucose levels. Lantidra is an allogeneic pancreatic islet cell therapy made from deceased donor pancreatic cells.

The US Food and Drug Administration has awarded a rare paediatric disease designation to a subsidiary of Nippon Shinyaku Co Ltd for an experimental treatment for Duchenne muscular dystrophy (DMD). DMD is a genetic disease that causes progressive weakness and the loss of skeletal, cardiac and respiratory muscles. It occurs primarily in males.

Leqembi (lecanemab), a monoclonal antibody that binds to beta-amyloid protofibrils, was approved by the US Food and Drug Administration for Alzheimer’s disease on 6 July, the first drug to show clinical benefit in patients with a mild stage of the disease. The full approval comes six months after Leqembi was given an accelerated authorisation on 6 January which was based on a surrogate endpoint – reducing amyloid plaques in the brain.

The US Food and Drug Administration approved the first gene therapy for the treatment of paediatric Duchenne muscular dystrophy on 22 June, saying that the drug, Elevidys, addresses an urgent unmet medical need. About one in every 3,300 boys in the US are affected by DMD  which is caused by a defective gene that results in the absence of dystrophin, a protein that helps keep the body’s muscle cells intact. As the disease progresses, life-threatening heart and respiratory problems may occur.

Two marketed drugs for adults with Type 2 diabetes have now been approved by the US Food and Drug Administration for children 10 years and older in order to help them improve control over abnormally high levels of glucose in their blood. The drugs, Jardiance (empagliflozin) and Synjardy (empagliflozin and metformin hydrochloride), are both approved oral therapies intended to supplement diet and exercise. Both medicines contain empagliflozin, a sodium-glucose co-transporter which works by increasing the excretion of glucose in the urine.

Mendus AB of Sweden announced on 19 June that it has received a certificate from the European Medicines Agency confirming that the manufacturing preparations and preclinical data for its lead dendritic cell vaccine candidate meet the current regulatory standard. The EMA’s advanced therapy medicinal product certificates may be issued to cell and gene therapy developers at the start of product development to verify quality controls. They are not required for a future regulatory submission.

An advisory committee that reviews drug applications for the US Food and Drug Administration has concluded that the Alzheimer’s disease treatment Leqembi (lecanemab) has shown clinical benefit for patients and should be approved. The Peripheral and Central Nervous System Drugs Advisory Committee voted unanimously to recommend full approval on 9 June. A decision by the FDA is expected on 6 July.

A lawsuit has been filed by the US Chamber of Commerce against the American government seeking to revoke part of the Inflation Reduction Act that gives the government powers to negotiate the prices of some drugs directly with industry. The suit was filed on 9 June in the US District Court in Dayton, Ohio saying the legislation violates constitutional protections for free enterprise. Merck & Co Inc registered a similar complaint in the US District Court in Washington DC on 6 June.