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. Once the EMA positive opinion is confirmed by the European Commission, the drug will be available to health authorities in Europe.
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.
According to the BBC, the number of cases in Italy reached 400 on 26 February, up by 25% in just 24 hours. To date, there have been 12 fatalities.
The Type 2 diabetes medicine Trulicity (dulaglutide) has been approved in the US to treat adults with diabetes who have cardiovascular disease or are at risk of the disease. The makes Trulicity, first approved in 2014, the only Type 2 diabetes medicine that can also be used to reduce the risk of major adverse cardiovascular events.
The US Food and Drug Administration has stepped up its oversight of certain pharmacogenetic tests that make claims for treatments that are not supported by sound science. In an announcement on 20 February, the agency said that it was introducing a new table on the internet giving a list of gene-drug interactions that are based on scientific evidence. This can be viewed at https://www.fda.gov/medical-devices/precision-medicine/table-pharmacogenetic-associations.
A new intravenous treatment for the prevention of migraine has been approved by the US Food and Drug Administration on the basis of trial data showing that patients had a sustained reduction in their disease episodes for a period of months after receiving the drug. The treatment, Vyepti (eptinezumab), is a monoclonal antibody that binds to the human calcitonin gene-related peptide with the result that migraine attacks are reduced. It was developed by Alder BioPharmaceuticals Inc, a company that was acquired by H. Lundbeck A/S of Denmark in 2019.
The first gene therapy for haemophilia to be submitted for regulatory review, valoctocogene roxaparvovec, has been given ‘priority review’status by the US Food and Drug Administration. Developed by BioMarin Pharmaceutical Inc, the therapy will be assessed for safety and efficacy for the treatment of patients with haemophilia A. Haemophilia A is the more prevalent of the two disease types, haemophilia A and B. Under a priority review, the FDA aims to issue a decision on marketing in six months rather than the usual 10, which means the outcome of the review should be known by 21 August.
New confirmed cases of infections from the coronavirus in China, also known as COVID-19, are “generally in decline,” China’s ambassador to the EU, Zhang Ming, told journalists on Tuesday. The ambassador was speaking at a meeting in Brussels, organised by the think tank EU-Asia. Topics included EU-China trade relations, but the virus epidemic dominated the conversation. Mr Ming was at pains to detail China’s efforts to contain the epidemic and predicted that the contagion would soon be overcome.
The US Food and Drug Administration has issued new guidance for developers of gene therapies highlighting both the complexity of manufacture as well as the difficulty in establishing duration of response in clinical trials.
In six final guidances, the agency addresses these issues as well as the application of orphan drug regulations to gene therapy. Orphan drug eligibility is an issue because many gene therapies are being developed for rare diseases where the patient populations are small.