Regulation & Policy

SSI Strategy Holdings LLC, a US-based life science consultancy, is to acquire the NDA Group AB of Sweden in a move that will expand its repertoire of services for pharmaceutical and biotech companies to include regulatory advice as well as support for drug development. The combined group will have a commercial presence across the US and Europe with a capacity to provide services from clinical development to medical affairs and regulation, including the use of real-world evidence to evaluate the effectiveness of new drugs.

GSK Plc’s antibody treatment Jemperli (dostarlimab) for solid tumours and endometrial cancer has been given a third indication by the US Food and Drug Administration, this time as a frontline treatment for recurrent endometrial cancer. It is now authorised in combination with carboplatin and paclitaxel chemotherapy, followed by Jemperli as a single agent, for patients with endometrial cancer that is mismatch repair deficient, as shown by an FDA test. Mismatch repair deficient (dMMR) cancers are those where errors arising during DNA replication are not corrected.

The US Food and Drug Administration has approved a new medicine to treat postpartum depression (PPD) – the first oral treatment for a condition that can affect women after childbirth or in the later stages of pregnancy. Zurzuvae (zuranolone), is a neuroactive steroid that is thought to work by rebalancing brain networks responsible for mood and cognition. It is the second drug to be approved for this condition after Zulresso (brexanolone) which was authorised by the agency in 2019 but can only be administered intravenously.

A new vaccine to protect against a disease caused by the respiratory syncytial virus (RSV) has received a positive accelerated assessment from the European Medicines Agency. If approved, Abrysvo would be used for the passive immunisation of infants from birth through six months of age following administration to the mother during pregnancy. It would also be indicated for the active immunisation of adults 60 years and older. A formal decision is still to be issued by the European Commission.

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.