Regulation & Policy

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.

The US Food and Drug Administration has expanded the permitted medical use of a gene therapy for Duchenne muscular dystrophy (DMD), making it available to patients who are both ambulatory and non-ambulatory. DMD is a genetic disorder characterised by a progressive deterioration of muscle due to abnormalities in, or the absence of, dystrophin protein which helps keep muscle cells intact.

Formycon AG of Germany has started clinical development of a candidate biosimilar of Keytruda (pembrolizumab), the checkpoint inhibitor for melanoma and other cancers that has revolutionised medical practice by blocking the interaction between two proteins and activating the immune system to kill cancer cells. To date, Keytruda has been approved for 48 medical indications.

Blincyto (blinatumomab), a bispecific antibody first approved for cancer in 2014, has received a new indication from the US Food and Drug Administration, extending its coverage for the treatment of B cell acute lymphoblastic leukaemia (B-ALL). B-ALL, an aggressive leukaemia, is the most common type of acute lymphoblastic leukaemia.
The new indication is for the treatment of adults and children from the age of one month for CD19 positive Philadelphia chromosome-negative B cell precursor acute lymphoblastic leukaemia.