Regulation & Policy

Pfizer Inc has received regulatory approval in the US for a personalised, or precision, medicine for lung cancer that demonstrated efficacy in a single-arm trial in both treatment naïve and previously treated patients with BRAF-V600E mutant metastatic non-small cell lung cancer. The medicine, Braftovi (encorafenib) and Mektovi (binimetinib), is a combination therapy that was first approved in 2018, but has since been updated. The newest approval includes two companion diagnostics that can identify BRAF V600E mutations in plasma or tumour tissue.

A diagnostic test for cancer was approved by the US Food and Drug Administration on 30 September which will help physicians detect hundreds of genetic variants associated with a person’s elevated risk of developing certain malignancies. Produced by Invitae Corp, the test also identifies potentially cancer-associated hereditary variants in individuals who have already been diagnosed with cancer.

GSK Plc has received US approval for Ojjaara (momelotinib), a Janus kinase inhibitor for the treatment of myelofibrosis patients with anaemia. The authorisation is based on positive results from two Phase 3 trials both of which were designed to show a reduction in manifestations of the disease. Myelofibrosis is a rare blood cancer that can lead to severely low blood counts, including anaemia and thrombocytopaenia.

In an article published on www.mednous.com on 21 August 2023 entitled SSI Strategy buys NDA Group, the leadership of the new group has been corrected to show that Douglas Locke, Chief Executive Officer of SSI Strategy, has been named CEO of the combined group. A corrected version follows.

A vaccine to prevent lower respiratory tract disease in infants which is caused by the respiratory syncytial virus (RSV) was approved by the Food and Drug Administration on 21 August – the second indication for this product in the US. Abrysvo has also been approved to prevent the same respiratory disease in people 60 years and older. Under the new indication, it will be administered as a single dose injection to pregnant women.

Ipsen SA has received Food and Drug Administration approval to market Sohonos (palovarotene) in the US for the treatment of fibrodysplasia ossificans progressiva (FOP), a rare genetic disease that causes connective tissue such as muscle and tendons to gradually turn into bone. This leads to a progressive loss of mobility and function for those affected. The approval was issued on 16 August and was based on the results of a Phase 3 trial of patients with the disease.

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.