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.
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.
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.
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.
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.
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.