The regulatory round-up: Five important FDA approvals


The US Food and Drug Administration (FDA) has granted marketing authorisations to several new therapeutics over the last month that will have a significant impact on areas of unmet need and the evolution of future treatments.

  1. Elevidys, Sarepta Therapeutics

Elevidys became the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US in June this year. The FDA granted accelerated approval to the adeno-associated virus (AAV) based gene therapy for the treatment of ambulatory paediatric patients aged four through five years with DMD with a confirmed mutation in the DMD gene based on expression of Elevidys micro-dystrophin.

Read more: FDA approves first gene therapy for Duchenne muscular dystrophy

  1. ACI-24.060, AC Immune

At the end of June, AC Immune received Fast Track designation from the FDA for its anti-amyloid beta (Abeta) active immunotherapy (vaccine)-candidate, ACI-24.060, for treatment of Alzheimer’s disease (AD). The Fast Track designation application was supported by positive initial interim safety and immunogenicity data from ABATE’s first, low dose AD cohort.

Read more: FDA fast-tracks immunotherapy for Alzheimer’s disease

  1. Roctavian, BioMarin

Roctavian (valoctocogene roxaparvovec-rvox) was the first gene therapy for adults with severe haemophilia A to be approved in the US. Specifically, it was approved for the treatment of adults with severe haemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test.

Read more: One-time gene therapy for haemophilia A gets FDA green light

  1. Lantidra, CellTrans

In July, the FDA approved Lantidra, the first allogeneic (donor) pancreatic islet cellular therapy made from deceased donor pancreatic cells for the treatment of type 1 diabetes. It was approved for the treatment of adults with type 1 diabetes who are unable to approach target glycated haemoglobin (average blood glucose levels) because of current repeated episodes of severe hypoglycaemia (low blood sugar) despite intensive diabetes management and education.

Read more: FDA approves first cellular therapy for type 1 diabetes

  1. Beyfortus, AstraZeneca

Beyfortus (nirsevimab-alip) was approved for the prevention of respiratory syncytial virus (RSV) lower respiratory tract disease in neonates and infants born during or entering their first RSV season, a group that are at the highest risk for developing severe RSV disease. The drug was also approved in children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season.

Read more: Monoclonal antibody to prevent RSV in babies approved in US

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