Regulation & Policy

The European Medicines Agency has given a positive scientific review to a new treatment for achondroplasia, a condition that impairs bone growth and causes dwarfism. It is recommending that a marketing authorisation be issued to BioMarin Pharmaceutical Inc, the developer, for use of Voxzogo (vosoritide) in patients two years and older whose epiphyses, or growth plates of the bones, are not yet closed. This condition must be confirmed by genetic testing. The medicine was evaluated in 121 patients with achondroplasia between the ages of five and 18 years.

Janet Woodcock, acting commissioner of the US Food and Drug Administration, has asked federal officials to conduct an investigation of interactions between agency staff and executives at Biogen Inc in the process that led up to the FDA’s approval of Aduhelm (aducanumab) for Alzheimer’s disease on 7 June. A letter was sent to the acting inspector general of the Department of Health and Human Services on 9 July and distributed globally on the same day on Twitter.

A gene therapy for multiple myeloma that was approved in the US in March has now been recommended for a conditional marketing authorisation by the European Medicines Agency. 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.

Actemra (tocilizumab), a biologic treatment for rheumatoid arthritis, has been given an emergency use authorisation in the US for the treatment of adults and children two years and older who have been hospitalised with Covid-19. The approval was based on data from four clinical trials, one of which was RECOVERY, a trial led by researchers in the UK.

After interim data failed to meet the statistical criteria for success, CureVac NV is pressing ahead to complete development of its first Covid-19 vaccine while making plans to start studies of a second generation compound later this year. On 16 June, CureVac announced that its messenger RNA vaccine, CVnCoV, demonstrated an interim efficacy rate of 47% in a Phase 2b/3 trial. The outcome was weaker than expected.

Three industry associations have issued an appeal to the European Commission to exempt advanced therapy medicinal products (ATMPs) from the EU’s genetically modified organism (GMO) legislation. This is to make the region more competitive as a site for clinical trials. The appeal was issued in a statement from the Alliance for Regenerative Medicine; the European Federation of Pharmaceutical Industries and Associations; and the European Association of Bioindustries. A paper was also published online on 24 May in the journal Human Gene Therapy.

A new treatment for Alzheimer’s disease was approved by the US Food and Drug Administration on 7 June using a surrogate end point for clinical benefit: a reduction of amyloid beta plaque in the brain. Aduhelm (aducanumab) was given an accelerated approval. It is the first new treatment approved for Alzheimer's disease in the US since 2003.

Novo Nordisk A/S has received US Food and Drug Administration approval for its drug Wegovy (semaglutide) for the treatment of obesity – the first weight management drug to be approved by the agency since 2014. More than one-third of US adults are estimated to be obese with the highest prevalence among those in middle age. Obesity is a risk factor for multiple disorders including heart disease, stroke and diabetes.

A new drug to treat lung cancer caused by a specific genetic mutation has been approved by the US Food and Drug Administration. Lumakras (sotorasib) has been authorised for patients with non-small cell lung cancer whose tumours have a KRAS G12C mutation and who have received at least one prior systemic therapy. This is the first approved targeted therapy for tumours with any KRAS mutation. KRAS G12C mutations represent about 13% of all mutations in non-small cell lung cancers.

The European Medicines Agency has given a positive opinion to a new gene therapy for cerebral adrenoleukodystrophy (CALD), a rare inherited neurological disease that affects approximately one in 21,000 newborn males. The scientific endorsement, which is the first step towards a formal marketing authorisation, is based on data from a single-arm trial.