Regulation & Policy

The US Food and Drug Administration has approved a new indication for Dupixent (dupilumab), broadening its role as a leading anti-inflammatory medicine for allergic diseases. The new use is for eosinophilic oesophagitis (EoE), a chronic inflammatory disorder in which eosinophils, a type of white blood cell, are found in the tissue of the oesophagus. This can impair a person’s ability to swallow. Dupixent is a monoclonal antibody that blocks interleukin 4 and interleukin 13 – part of the inflammatory pathway.

The US Food and Drug Administration has approved a new diabetes medicine which targets two hormones involved in blood sugar control. In clinical studies, Mounjaro (tirzepatide) was shown to be more be more effective than three other approved diabetes drugs. It was developed by Eli Lilly and Co and given an expedited review by the FDA on the basis of its potential for significantly improving the treatment of Type 2 diabetes. It is also under regulatory review in Europe and Japan.

The European Medicines Agency has created a new forum to discuss the way cancer drugs are developed and administered in order to improve the healthcare outcomes for patients. The forum was launched in late March in partnership with the European Organisation for Research and Treatment of Cancer (EORTC), a non-profit organisation located in Belgium,

The US Food and Drug Administration has given an accelerated approval to Novartis for a new drug to treat a rare group of genetic diseases known as PIK3CA-related overgrowth spectrum (PROS). The drug, Vijoice (alpelisib), was approved on the basis of real-world evidence from patients who received alpelisib as part of a compassionate use programme.

The PROS group of disorders cause overgrowth in parts of the body due to mutations in the PIK3CA gene. Vijoice is a kinase inhibitor designed to block the PI3K pathway and predominantly the P13K-alpha isoform.

A new paediatric formulation of the HIV medicine Triumeq (abacavir, dolutegravir and lamivudine) has been approved by the US Food and Drug Administration, extending the treatment to children who otherwise would go unprotected. An estimated 1.7 million children globally were living with HIV in 2020 with most deaths from the disease occurring during the first five years of life, according to the Joint United Nations Programme on HIV/AIDS.

The US Food and Drug Administration has given emergency use authorisations for a second booster dose of two messenger RNA (mRNA) vaccines for Covid-19 in order to strengthen protection against the SARS-CoV-2 virus for the elderly and people whose immune systems have been compromised. This follows an examination of clinical data from Israel and elsewhere which showed that a second booster presented no new safety concerns.

A cell-based gene therapy for multiple myeloma has been recommended by the European Medicines Agency for patients with multiple myeloma, an incurable blood cancer that causes plasma cells to grow out of control. Symptoms can include bone fracture or even kidney failure. The drug, Carvykti (ciltacabtagene autoleucel), is a chimeric antigen receptor (CAR) T cell medicine consisting of a patient’s own immune cells which have been engineered to target and kill cancer cells.

The US Food and Drug Administration has approved the first monoclonal antibody targeting the LAG-3 protein on T cells – one of several proteins that prevent the immune system from fighting cancer. The antibody, relatlimab, has been developed in combination with nivolumab to treat metastatic melanoma. The combination is being commercialised by Bristol Myers Squibb Co under the name Opdualag.

Valneva SE is preparing a regulatory submission in the US for its candidate vaccine, VLA1553, to prevent chikungunya virus infection. This follows positive clinical data from a six-month study which confirmed the results of a large Phase 3 trial of 4,115 adults.

The European Medicines Agency is studying the T cell immune response generated by the currently approved Covid-19 vaccines to find out whether anything can be learned for the future. In a briefing with journalists on 3 March, Marco Cavaleri, head of biological health threats and vaccines strategy, said the review covers all platforms represented by the currently approved vaccines. One of the objectives is to find out if the T cell component of immunity can offer protection against new variants of the virus.