Regulation & Policy

AstraZeneca Plc has received US approval for a new indication for Fasenra (benralizumab), an antibody therapeutic originally developed for asthma and now authorised to treat eosinophilic granulomatosis with polyangiitis (EGPA), a rare disease caused by inflammation of small to medium-sized blood vessels. Fasenra binds to a subunit of the interleukin-5 receptor which is predominantly expressed on human eosinophils, a type of white blood cell. Although affecting only an estimated 118,000 people globally, the disease can be fatal without treatment. 

The breast cancer treatment Kisqali (ribociclib), first approved in the US in 2017, has received a new indication from the Food and Drug Administration for patients with early cancer who are at a high risk of disease recurrence. The approval, announced by Novartis on 17 September, is based on data from a Phase 3 trial which showed a clinically meaningful reduction in the risk of disease recurrence in patients with hormone receptor-positive/human epidermal growth factor receptor 2-negative, including those with node-negative disease.

Covid-19 vaccines manufactured by Moderna Inc and Pfizer Inc have been updated and approved by the US Food and Drug Administration to include a component targeting a new strain of the SARS-CoV-2 virus. The update is the latest move by health authorities to better protect the population against variants of the virus which could lead to hospitalisation or even death.

GSK Plc has received a new indication from the US Food and Drug Administration for its endometrial cancer medicine Jemperli (dostarlimab) that significantly increases the scope of its coverage. Jemperli is a checkpoint inhibitor that has already been approved for three endometrial cancer indications and for the treatment of patients with mismatch repair-deficient advanced solid tumours. Mismatch repair is a process that corrects errors during the replication of DNA. When this process fails, it can cause cancer.

A new product designed to reduce proteinuria, a feature of a rare disease potentially affecting kidney function, has been given an accelerated approval by the US Food and Drug Administration. The product, Fabhalta (iptacopan), is a small molecule developed by Novartis and the first of several assets from its portfolio of drugs for rare kidney diseases. The FDA approval was announced on 8 August and is based on interim data from a Phase 3 trial investigating Fabhalta in comparison with placebo in patients with primary immunoglobulin A nephropathy (IgAN).

The US Food and Drug Administration has given an accelerated approval to a new therapy for synovial sarcoma, the first engineered cell therapy for a solid tumour to receive a marketing approval. The product, Tecelra (afamitresgene autoleucel), is directed against tumours that express the melanoma-associated antigen A4 (MAGE-A4), a marker for the disease. Synovial sarcoma is a rare type of soft tissue sarcoma which can occur in many parts of the body but most frequently in the extremities. The product was developed by Adaptimmune Therapeutics Plc.

The European Medicines Agency has issued a negative opinion for Leqembi (lecanemab), a treatment for Alzheimer’s disease, saying that the benefits of the drug do not outweigh the risks. The opinion, issued on 25 July, is in contrast to decisions by regulatory authorities in six other countries, including the US, which have all approved the treatment. Leqembi is currently being marketed in the US, Japan and China. Co-developer Eisai Co Ltd said that it will appeal against the decision.

The US Food and Drug Administration has approved Eli Lilly and Co’s drug for early symptomatic Alzheimer’s disease, less than a month after it received the unanimous support from an outside panel of advisers. The Peripheral and Central Nervous System Drugs Advisory Committee voted 11 to zero on 10 June to recommend approval. The drug, Kisunla (donanemab), is a treatment for early symptomatic disease including people with mild cognitive impairment.

The US Food and Drug Administration has approved a new drug for chronic obstructive pulmonary disease, the third leading cause of death worldwide. Ohtuvayre (ensifentrine), developed by Verona Pharma Plc, is an inhaled therapy for the maintenance treatment of COPD, a disease that is mainly caused by tobacco smoking. It was authorised by the FDA on 26 June.

The European Medicines Agency has given a conditional marketing authorisation to a gene therapy for haemophilia B which is a one-time treatment for the rare inherited blood disorder. Announced on 31 May, the decision is for fidanacogene elaparvovec, an adeno-associated virus based therapy that is designed to deliver a functional copy of the Factor 9 gene into the body enabling patients to produce Factor 9 protein themselves. The developer is Pfizer Inc which received US regulatory approval for the same therapy in late April.