Regulation & Policy

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.

A new drug has been approved by the US Food and Drug Administration for polycythemia vera, a rare disease that causes the bone marrow to make too many red blood cells, which can thicken the blood and cause clots. Besremi (ropeginterferon alfa-2b-njft) is the first approved medicine for this disease that patients can take, irrespective of their treatment history. It is also the first interferon therapy specifically approved for polycythemia vera.

Robert Califf, a professor of medicine at the Duke University School of Medicine, is to be nominated to lead the US Food and Drug Administration – a position he held during the final year of the Obama administration and will return to under the Biden presidency. A cardiologist, Dr Califf has been vice chancellor for clinical and translational research at Duke and was founding director of the Duke Clinical Research Institute, where he led many clinical trials.

The US Food and Drug Administration has amended the emergency approvals for three Covid-19 vaccine booster shots to make them available to more people. At the same time, the agency said a booster shot from any of the approved vaccines may be administered to people whose first vaccination was with a different product.

“A single booster dose of any of the available Covid-19 vaccines may be administered as a heterologous booster dose following completion of primary vaccination with a different available Covid-19 vaccine,” the agency said on 20 October.

Boehringer Ingelheim has received a second US approval for its anti-inflammatory drug Cyltezo (adalimumab-adbm), a biosimilar version of Humira, one of the most widely sold drugs in the developed world. Humira is still protected by patents which means that Cyltezo will not become commercially available in the US until 1 July 2023.

The European Medicines Agency has recommended approval of an antibody-drug conjugate (ADC) for the treatment of metastatic triple-negative breast cancer, an aggressive cancer that does not respond to hormonal therapy medicines or medicines that target HER2 protein receptors. The positive opinion was based on data from a Phase 3 trial which showed that the therapy, Trodelvy (sacituzumab govitecan), produced an overall survival figure of 11.8 months and progression-free survival of 4.8 months, compared with 6.9 months and 1.7 months respectively, for chemotherapy.

The US Food and Drug Administration has approved the first allogeneic tissue product for the treatment of children born without a thymus, a condition known as congenital athymia. The product, Rethymic, is a donor derived product that is processed and cultured and implanted into patients to help reconstitute immunity in the absence of a thymus gland. The thymus is located behind the sternum and between the lungs, and is active until puberty. The gland secretes thymosin, a hormone necessary for T cell development and production.

CureVac NV has withdrawn its application for a marketing authorisation for its first Covid-19 vaccine from the European Medicines Agency, citing plans to focus instead on a second generation product that has shown an improved immune response in an animal study. The second generation vaccine, CV2CoV, is being developed with GlaxoSmithKline Plc. It encodes for the same protein as the company’s earlier vaccine but differs in the design of non-coding regions of the mRNA backbone.

The US Food and Drug Administration has given an emergency use authorisation for a booster dose of the Pfizer/BioNTech Covid-19 vaccine for individuals 65 years and older and those who are at high risk of the disease. The high-risk population includes people who are healthcare workers and teachers, as well as those in homeless shelters and prisons.