Blog Post 1: BioMarin Pharmaceutical INC. - A Promising Investment
- tom.settelmayer
- Jan 16, 2022
- 2 min read
Updated: Jan 16, 2022
Headquartered in San Raphael, California, BioMarin is a pharmaceutical company that specializes in generating enzyme replacement therapies for rare diseases. But why would one consider BioMarin Pharmaceutical to be a lucrative investment as we enter 2022?
With Phase III data being released in early 2022 for the new gene therapy Roctavian, BioMarin demonstrates favorable developments in the field. Roctavian is utilized to treat individuals with severe Hemophilia A: a genetic disorder caused by a missing or defective factor VIII (a clotting protein in the blood). Deployed as a one-time gene therapy, Roctavian, formally known as valoctogene roxaparvovec, makes use of a bioengineered viral vector to deliver a healthy version of the F8 gene (the gene that encodes for the factor VIII protein) to cells in the body, consequently reinstating normal production of the factor VIII protein. Assuming that Phase III data for Roctavian provides positive results, it is anticipated that the Committee for Medicinal Products for Human Use will issue an opinion in the first half of 2022, with a European regulatory approval following shortly after. Furthermore, a Prescription Drug Fee Act filing is expected for the second half of 2022. Nonetheless, BioMarin’s success in coming years does not stop at Roctavian. BioMarin’s Voxzogo, a prescription medicine used to treat symptoms of Achondroplasia (the stunted growth of long bones) in pediatric patients, has recently been approved by the FDA. With both the hopeful developments of Roctavian and the approval of Voxzogo, BioMarin Pharmaceutical has its sights set on high profit margins.
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