Regulation & Policy

The European Medicines Agency has proposed to extend the use of two messenger RNA (mRNA) vaccines for Covid-19 in order to give protection against the SARS-CoV-2 virus to younger people. In separate decisions, the agency is recommending that primary vaccination with Spikevax be approved for use in children from the ages of six to 11 years. Spikevax was developed by Moderna Inc. The agency is also proposing that Comirnaty, developed by BioNTech SE and Pfizer Inc, be authorised as a booster dose for adolescents from 12 years of age.

The US Senate has confirmed President Joe Biden’s nomination of Robert Califf to be head of the Food and Drug Administration – a position he held during the final year of the Obama administration. The vote was 50 to 46, the narrowest margin of any confirmed FDA commissioner.

The US Food and Drug Administration has approved the first bispecific antibody medicine for  the eye – a treatment that targets two pathways thought to contribute to vision loss by destabilising blood vessels. The drug, Vabysmo (faricimab-svoa), has been authorised for the treatment of neovascular or ‘wet’ age-related macular degeneration (AMD) and diabetic macular oedema. Both diseases impair central vision, with the latter affecting people with Type 1 and Type 2 diabetes.

The cell-based gene therapy Breyanzi (lisocabtagene maraleucel) has received a positive opinion from the European Medicines Agency to treat patients with relapsed or refractory large B-cell lymphoma. Breyanzi was approved for the same indication in the US in 2021. It will be available to patients in the EU once the European Commission issues a marketing authorisation.

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.

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.