People at risk of infection from the HIV-1 virus have a new option for preventing the disease following US Food and Drug Administration approval of Apretude (cabotegravir extended release injectable suspension), developed by ViiV Healthcare. Apretude is a long-acting anti-viral medication which inhibits HIV replication by preventing the viral DNA from integrating into the genetic material of human immune cells.
Regulation & Policy
Belgium-based argenx SE has won US Food and Drug Administration approval for its first product, Vyvgart (efgartigimod), for the treatment of generalised myasthenia gravis, a neuromuscular disease that causes weakness in the skeletal muscles. Vyvgart is an antibody fragment that binds to the neonatal Fc receptor, preventing the receptor from recycling immunoglobulin G (IgG) back into blood. This leads to a reduction in IgG levels, including reductions in abnormal anti-acetylcholine receptor (AChR) antibodies which are present in myasthenia gravis.
A new treatment for sickle cell disease, Oxbryta (voxelotor), received a positive opinion from the European Medicines Agency on 17 December for patients 12 years of age and older. This is the same drug and patient group that received an accelerated approval from the US Food and Drug Administration in 2019. Concurrent with the EMA opinion, the FDA expanded the label of Oxbryta to include children from the age of four up to 11 years.
The European Medicines Agency has issued a negative opinion for the Alzheimer’s disease treatment Aduhelm (aducanumab) saying that studies supporting the drug’s application were conflicting and did not show overall effectiveness in treating adults with early stage disease. Under the EU’s regulatory procedures, the sponsor, Biogen, Inc, has 15 days to appeal against the opinion. Aduhelm is an immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against amyloid-beta levels in the brain.
The US Food and Drug Administration has approved the first drug for the prevention of acute graft versus host disease (GvHD), a condition that occurs when donor bone marrow or stem cells attack healthy tissues in a patient undergoing a treatment for certain cancers, or an organ transplant. The drug, Orencia (abatacept), is already on the market having been approved for rheumatoid arthritis in 2005. The manufacturer is Bristol Myers Squibb Co.
The New England Journal of Medicine has published data from a Phase 3 trial of Dupixent (dupilumab), a widely prescribed anti-inflammatory drug which was recently approved by the US Food and Drug Administration for children with uncontrolled moderate-to-severe asthma. Data from the trial formed the basis of a US approval and have been included in a regulatory filing with the European Medicines Agency.
The Covid-19 vaccine produced by Pfizer Inc and BioNTech SE is able to neutralise the Omicron variant of the SARS-CoV-2 virus after three doses, preliminary laboratory studies have shown. Two doses of the vaccine appear to be less effective, but still offer protection against the most severe forms of the disease, the companies reported on 8 December.
More than 43% of the global population of 7.9 billion people have been fully vaccinated against Covid-19 with all regions of the world except Africa making progress in immunising their people, according to statistics released by the European Commission on 8 December. The World Health Organization has set an immunisation target of 70% to be achieved by September 2022 at the next meeting of the United Nations General Assembly.
Merck & Co Inc and its partner Ridgeback Biotherapeutics LP published new efficacy data for their Covid-19 treatment on 26 November showing that the anti-viral drug reduced the risk of hospitalisation or death by 30%, compared with a placebo, instead of the earlier reported 50%. The updated efficacy figure is based on additional data presented to the US Food and Drug Administration. The FDA’s Antimicrobial Drugs Advisory Committee was scheduled to meet on 30 November to evaluate the latest information.
A preventive treatment for migraine, Vyepti (eptinezumab), has received a positive opinion from the European Medicines Agency – the first step towards getting an authorisation for the EU market. If approved, it will be the first and only intravenous treatment for migraine prevention in the region, the developer H. Lundbeck A/S, announced on 12 November.