Regulation & Policy

Johnson & Johnson Inc has announced the selection of a lead vaccine candidate to test against COVID-19, the disease caused by the coronavirus SARS-CoV-2, and intends to start clinical studies by September at the latest. The first batches of the vaccine could be available for approval under an emergency use regulation by early 2021, the company said on 30 March.

The Zolgensma gene therapy for spinal muscular atrophy, which was approved by the US Food and Drug Administration in May 2019, has now received a positive review by the European Medicines Agency. Pending approval by the European Commission, it is expected to be available to treat babies and young children across the EU soon. Spinal muscular atrophy is a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement.

Pharmaceutical regulators from 17 countries have agreed in principle on the criteria that must be met before developers of candidate vaccines for the coronavirus SARS-CoV-2 start human trials. The officials held a virtual meeting on 18 March, and a summary of their conclusions was released by the European Medicines Agency on 24 March.

The European Medicines Agency has issued a statement urging the research community to give priority to large randomised controlled studies when investigating potential treatments for COVID-19 and to include all EU countries in these trials.

New guidance on the conduct of clinical trials during the COVID-19 pandemic has been published by the European Commission, the European Medicines Agency and heads of national agencies in the EU. https://ec.europa.eu/health/sites/health/files/files/eudralex/vol-10/guidanceclinicaltrials_covid19_en.pdf, Developers including Eli Lilly and Co, have already announced delays in the start of studies and a pause

Seven scientists drawn from six EU member states met for the first time on 18 March to advise the European Commission on how to respond to the multitude of issues facing public health authorities during the coronavirus pandemic. Among the topics discussed were managing hospital capacity, the conduct of new clinical trials, and the protection of vulnerable populations such as refugees.

Novartis has secured approval in the US for Isturisa (osilodrostat), a new treatment for Cushing’s disease, a rare disorder in which the adrenal glands make too much of the cortisol hormone. Announcing the decision on 7 March, the Food and Drug Administration said that Isturisa is the first drug to directly address this cortisol overproduction by blocking the enzyme 11-beta-hydroxylase and preventing cortisol synthesis.

A drug previously approved to treat idiopathic pulmonary fibrosis, Ofev (nintedanib), has received a new indication in the US – to treat patients with chronic fibrosing interstitial lung diseases. Announcing the decision on 9 March, the Food and Drug Administration said it was the first treatment authorised for this group of fibrosing lung diseases which worsen over time. Ofev was developed by Boehringer Ingelheim GmbH.

A new antibiotic capable of treating infections from aerobic Gram-negative bacteria has been given a positive opinion by the European Medicines Agency. Fetcroja (cefiderocol) was shown to be effective in complicated urinary tract infections, nosocomial pneumonia and in critically ill patients with confirmed carbapenem-resistant infections. It was developed by Shionogi & Co Ltd in Japan and approved by the US Food and Drug Administration in November 2019.

Public health and border control officials at the European Commission currently believe that the risk of imminent transmission of the COVID-19 coronavirus across the EU is low to moderate – even as the virus continues to infect increasing numbers of people in Italy.