Regulation & Policy

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oversight over AstraZeneca’s latest Covid-19 vaccine trial, has expressed concern that the company may have included outdated information from the trial when it reported an efficacy rate of 79% for the vaccine on 22 March.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 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.

The European Commission has given a conditional marketing authorisation to the Covid-19 vaccine developed by Janssen Pharmaceutica NV following a positive scientific review by the European Medicines Agency. The decision, announced on 11 March, comes just under two weeks after the US gave the same vaccine an emergency use authorisation.

The European Medicines Agency is recommending approval of Evrysdi (risdiplam) to treat spinal muscular atrophy (SMA) – a rare and often fatal disease affecting neurons in the brain and spinal cord that control muscle movement. SMA is caused by mutations in the SMN1 gene that encodes for the survival motor neuron protein.

Merck & Co Inc has voluntarily withdrawn the metastatic small cell lung cancer indication for Keytruda (pembrolizumab) following the results of a Phase 3 study which didn’t support the terms of its earlier accelerated approval from the US Food and Drug Administration. The FDA awards accelerated approvals to certain drugs, based on surrogate endpoints, but confirmation of clinical benefit needs to be verified in subsequent trials.

A single dose vaccine developed by Janssen Biotech Inc to prevent Covid-19 has been cleared for emergency use by the Food and Drug Administration, the third vaccine for the coronavirus to be allowed onto the US market. The two other authorisations were for vaccines developed by a partnership between BioNTech SE and Pfizer Inc, and by Moderna Inc.

A new treatment for Duchenne muscular dystrophy (DMD) has been approved in the US which uses a technology called exon skipping to bypasss a specific genetic mutation in the production of dystrophin, a protein which strengthens muscle fibers and protects them from injury. By skipping the exon, normal amounts of dystrophin protein are produced. DMD is a genetic disorder characterised by dystrophin loss and a progressive deterioration of muscle.

AstraZeneca Plc has voluntary withdrawn a bladder cancer indication for its checkpoint inhibitor Imfinzi (durvalumab) in the US, which was given an accelerated approval in 2017 but failed to show the required efficacy in a follow-up study. The Food and Drug Administration awards accelerated approvals to certain drugs, based on surrogate endpoints, but confirmation of clinical benefit needs to be verified in subsequent trials.

The US Food and Drug Administration has approved a new small molecule drug for patients with small cell lung cancer who are set to receive chemotherapy but may be at risk of damage to their bone marrow. Cosela (trilaciclib) is to be administered before chemotherapy in order to preserve bone marrow and immune system function.

The World Health Organization has granted an emergency use authorization to the Covid-19 vaccine developed by AstraZeneca Plc and Oxford University making it eligible for distribution in up to 145 low and middle-income countries. Distribution will take place through Covax (Covid-19 Vaccines Global Access), a global vaccine project being led by the Gavi public-private partnership, the Coalition for Epidemic Preparedness Innovations (CEPI) and the WHO.