Delandistrogene moxeparvovec

Delandistrogene moxeparvovec (also known as Valoctocogene Roxaparvovec or BMN 270) is a gene therapy product developed by BioMarin Pharmaceutical for the treatment of hemophilia A. It is designed to deliver a functional copy of the human factor VIII (FVIII) gene into the patient's liver cells, thereby enabling the body to produce a continuous supply of factor VIII for blood clotting.

Mechanism of Action

Delandistrogene moxeparvovec uses a viral vector based on the adeno-associated virus (AAV) to deliver the FVIII gene into the patient's liver cells. The AAV vector is engineered to be non-pathogenic and to express the FVIII gene under the control of a liver-specific promoter. Once inside the liver cells, the FVIII gene is integrated into the host cell's DNA, enabling the cell to produce and secrete FVIII protein into the bloodstream.

Clinical Trials

Several clinical trials have been conducted to evaluate the safety and efficacy of delandistrogene moxeparvovec. In a phase 1/2 trial, patients with severe hemophilia A who received a single infusion of the gene therapy showed sustained increases in FVIII levels and a significant reduction in bleeding episodes. A phase 3 trial is currently underway.

Regulatory Status

In 2019, BioMarin submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for delandistrogene moxeparvovec. However, the FDA requested additional data from the phase 3 trial before making a decision on approval. The product has also been granted Orphan Drug status by the FDA and the European Medicines Agency (EMA) for the treatment of hemophilia A.

See Also

• Gene therapy
• Hemophilia A
• BioMarin Pharmaceutical
• Adeno-associated virus
• Clinical trial
• U.S. Food and Drug Administration
• European Medicines Agency