Regulation & Policy

Two marketed drugs for adults with Type 2 diabetes have now been approved by the US Food and Drug Administration for children 10 years and older in order to help them improve control over abnormally high levels of glucose in their blood. The drugs, Jardiance (empagliflozin) and Synjardy (empagliflozin and metformin hydrochloride), are both approved oral therapies intended to supplement diet and exercise. Both medicines contain empagliflozin, a sodium-glucose co-transporter which works by increasing the excretion of glucose in the urine.

Mendus AB of Sweden announced on 19 June that it has received a certificate from the European Medicines Agency confirming that the manufacturing preparations and preclinical data for its lead dendritic cell vaccine candidate meet the current regulatory standard. The EMA’s advanced therapy medicinal product certificates may be issued to cell and gene therapy developers at the start of product development to verify quality controls. They are not required for a future regulatory submission.

An advisory committee that reviews drug applications for the US Food and Drug Administration has concluded that the Alzheimer’s disease treatment Leqembi (lecanemab) has shown clinical benefit for patients and should be approved. The Peripheral and Central Nervous System Drugs Advisory Committee voted unanimously to recommend full approval on 9 June. A decision by the FDA is expected on 6 July.

A lawsuit has been filed by the US Chamber of Commerce against the American government seeking to revoke part of the Inflation Reduction Act that gives the government powers to negotiate the prices of some drugs directly with industry. The suit was filed on 9 June in the US District Court in Dayton, Ohio saying the legislation violates constitutional protections for free enterprise. Merck & Co Inc registered a similar complaint in the US District Court in Washington DC on 6 June.

The US Food and Drug Administration approved a second vaccine on 31 May to protect older adults against infection from the respiratory syncytial virus (RSV). The vaccine, Abrysvo, was developed by Pfizer Inc and is expected to be launched before the next RSV season which typically starts in the autumn and peaks in the winter. The decision follows the agency’s authorisation on 3 May of a vaccine for the same disease from GSK Plc.

Robert Califf, the US Food and Drug Administration Commissioner, took a strike against drug prices on 7 June saying that prices “are too high in the US” with the result that many patients, especially with cancer, are not completing their prescribed treatments because they cannot afford to do so.

The European Medicines Agency is recommending that Adakveo (crizanlizumab), a medicine for preventing vaso-occlusive crises in patients with sickle cell disease, be withdrawn from the EU market. This follows data from a post-authorisation study showing little benefit and a higher rate of adverse events. Developed by Novartis, Adakveo was given a conditional approval on 28 October 2020. Full approval was contingent on positive data from the STAND follow-up study.

The World Health Organization has declared an end to the emergency phase of Covid-19, but cautioned that risks still remain for the appearance of new variants of the SARS-CoV-2 virus. In a statement on 5 May, Tedros Adhanom Ghebreyesus, the WHO director general, said  Covid-19 will no longer be categorised as a public health emergency of international concern. But its status as a pandemic will remain until these risks diminish.

Going forward, the pandemic “appears to be in transition to a more endemic situation globally,” the WHO said.

A vaccine developed by GSK Plc to protect older adults against infection from the respiratory syncytial virus (RSV) has been approved in the US and given a positive opinion in the EU – the first such treatment to be approved anywhere in the world. The vaccine, Arexvy, will be launched in the US before the next RSV season which typically starts in the autumn and peaks in the winter. Launch in Europe is expected to follow a formal decision on marketing authorisation, which will be taken by the European Commission.

Health technology assessment (HTA) bodies in the US, Canada and the UK have reached a consensus about how confidential clinical data should be handled in their public assessments of new medicines. Each of the groups operates in a different healthcare environment, but they agree that more transparency is required in the future.