Regulation & Policy

Autolus Therapeutics Plc, a UK-based cell therapy company, reached its first regulatory milestone on 8 November when the US Food and Drug Administration approved its chimeric antigen receptor (CAR) T cell therapy for cancer. The therapy, Aucatzyl (obecabtagene autoleucel), targets the CD19 antigen on B cells. The approval is to treat B cell acute lymphoblastic leukaemia (ALL), an aggressive type of blood cancer. It was based on evidence from a single-arm trial in adults whose disease had relapsed after two or more prior lines of therapy.

A new treatment for acute respiratory distress syndrome (ARDS) received a positive opinion from the European Medicines Agency on 15 November paving the way for its use in patients with a coronavirus infection. The treatment, Gohibic (vilobelimab), is a monoclonal antibody directed against a component of the complement system which is part of the body’s immune system. By targeting the complement component C5a, the antibody is believed to block an inflammatory response induced by severe SARS-CoV-2 infection. The treatment has been developed by InflaRx NV of Germany. 

A new gene therapy has been given an accelerated approval in the US for an inherited disorder affecting the way neurons transmit information to other cells in the body. The therapy, Kebilidi (eladocagene exuparvovec), has been shown to be effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency, a rare disease. It is caused by mutations in the DDC gene leading to shortages of the AADC enzyme which plays an important role in the nervous system.

After a second review, the main scientific committee of the European Medicines Agency has recommended approval of Leqembi (lecanemab) for the treatment of mild cognitive impairment due to early Alzheimer's disease in patients who have only one, or no copy, of the ApoE4 gene. The EMA opinion, announced on 14 November, will be forwarded to the European Commission for a decision. 

Mario Draghi, former president of the European Central Bank, has issued another appeal to policymakers to build an integrated capital market for the EU. In a commentary in the Financial Times on 2-3 November, Mr Draghi argues that a unified market would mobilise private capital to back projects in defence, the building of a digital infrastructure, and the mitigation of climate change. Currently the EU is home to more than 30 national and regional stock exchanges, each with its own regulations. A similar decentralised structure exists for bond markets.

A new oral medicine for schizophrenia was approved by the US Food and Drug Administration on 27 September introducing a mechanism of action for treating the disease – a leading cause of disability worldwide. The drug, Cobenfy (xanomeline and trospium chloride), is the first antipsychotic drug approved to treat schizophrenia that targets cholinergic receptors as opposed to dopamine receptors, which is currently the standard of care. The drug was developed by Bristol-Myers Squibb Co.

Itovebi (inavolisib), a kinase inhibitor for use in combination with two other drugs to treat breast cancer, has been approved by the US Food and Drug Administration. This follows a priority review and breakthrough therapy designation for the combination with the marketed breast cancer drugs palbociclib (Ibrance) and fulvestrant (Faslodex). Itovebi targets the PI3K enzyme, a product of the PIK3CA gene. According to the US National Cancer Institute, mutations in the PIK3CA gene have been identified in multiple cancers including breast, lung, ovary, stomach and brain.

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.