News

The US Food and Drug Administration has approved a cell-based gene therapy for multiple myeloma – the first therapy of its type for myeloma, an uncommon type of blood cancer. Abecma (idecabtagene vicleucel) is a chimeric antigen receptor (CAR) T cell therapy targeting the B cell maturation antigen (BCMA), a cell membrane receptor expressed exclusively on late stage B cells and plasma cells. Abecma has been approved for adult patients who have not responded to, or whose disease has returned after, at least four prior lines of therapy.

A new endocrine treatment for patients with congenital adrenal hyperplasia is being recommended for marketing in Europe after achieving positive results in a Phase 3 study of 122 patients with the disease. The European Medicines Agency delivered the positive opinion on 26 March, ahead of a decision on marketing authorisation by the European Commission.

Evotec SE ended 2020 with liquidity of €481.9 million and a portfolio of co-owned research assets, equity investments and clinical-stage pharma projects underpinning its work as a global drug discovery company. Revenue for the year was €500.9 million, up by 12% from 2019. Operating profit was €48.5 million, down by 22% due to delayed milestone payments and higher investments in R&D.

AstraZeneca Plc has issued fresh data from its US Phase 3 trial of a Covid-19 vaccine developed jointly with Oxford University (AZD1222) showing an efficacy rate of 76% against symptomatic disease. The primary efficacy analysis included the accrual of 190 symptomatic cases of Covid-19 from the 32,449 trial participants. This represents an additional 49 cases as compared with an interim analysis of the trial which was published on 22 March and showed an efficacy rate of 79%.

A new radiotherapy for advanced prostate cancer has delivered positive results in a Phase 3 trial when compared with a standard of care for the disease, Novartis announced on 23 March. Lu-PSMA-617 met both primary endpoints of the trial, improving both overall survival and radiographic progression-free survival. The trial enrolled patients whose disease had progressed following prior taxane and androgen receptor-directed therapies.

In a bid to secure an adequate supply of Covid-19 vaccines for the European population, the European Commission has tightened a regulation, introduced at the start of the year, to monitor vaccine exports from the EU to other countries in the world. The move has generated concern from industry groups worried about a possible disruption to vaccine supply chains. However during a press briefing on 24 March, the Commission said the goal of the regulation was to achieve a “security of supply” for Europe while maintaining the integrity of the international trading system.

Roche has stopped dosing patients in a Phase 3 study of an investigational drug for Huntington’s disease, a rare genetic condition that causes nerve cells in the brain to break down. The drug, tominersen, is an antisense therapy designed to reduce the production of all forms of huntingtin protein, including its mutated variant. The trial was the largest study to date in patients with this disease.

An independent data and safety monitoring board (DSMB), which had oversight over AstraZeneca’s latest Covid-19 vaccine trial, has expressed concern that the company may have included outdated information from the trial when it reported an efficacy rate of 79% for the vaccine on 22 March. The DSMB concern was cited in a press release issued by the National Institute of Allergy and Infectious Diseases (NIAID), an agency of the US National Institutes of Health, on 23 March.

The Astra Zeneca/Oxford University vaccine (AZD1222) has shown an efficacy rate of 79% in preventing symptomatic Covid-19 disease and 100% efficacy at preventing severe disease and hospitalisation, AstraZeneca announced on 22 March. The interim data come from a Phase 3 trial conducted in the US which enrolled 32,449 participants. Speaking to reporters, company executives said data from the trial will be filed shortly with the Food and Drug Administration with a view to gaining an emergency authorisation and starting US distribution soon thereafter.

The European Medicines Agency has concluded that the benefits of the AstraZeneca/Oxford University Covid-19 vaccine outweigh the risks despite a possible link to rare blood clots with low blood platelets. The statement was issued on 18 March following a meeting of the agency’s Pharmacovigilance Risk Assessment Committee.