The U.S. Department of Justice has slapped BioMarin Pharmaceutical with a subpoena in connection with its sponsored testing programs for the rare disease therapies Vivizim and Naglazyme.
Tag Archive for: BioMarin
Activist investor Elliott Investment Management has bought more than a $1 billion stake in BioMarin Pharmaceutical, according to an exclusive report from Reuters published Tuesday, citing two unnamed sources familiar with the matter.
The label for the achondroplasia drug, which promotes endochondral bone growth, now covers children under five years of age with the rare genetic disease causing the most common form of dwarfism.
The FDA’s schedule is picking up speed, with six target action dates over the next two weeks, including one for a blockbuster cancer treatment and another for a highly anticipated therapy for a rare pediatric disorder.
BioMarin Pharmaceutical Inc. announced that an individual in Germany with severe hemophilia A was treated with Roctavian (valoctocogene roxaparvovec-rvox), marking the first time that the gene therapy has been given commercially in Europe.
After an initial rejection, BioMarin has finally secured the FDA’s approval for Roctavian, the first gene therapy in the U.S. for the most common form of the bleeding disorder.
The FDA will close the month of June with four target action dates, including one for an investigational gene therapy for a rare disease and another for an already top-selling eye disease treatment.
The rare disease biotech announced the FDA has accepted its supplemental New Drug Application to expand Voxzogo (vosoritide) to children younger than five years.
The FDA said late on Monday it needed more time to review a three-year analysis from the company’s ongoing late-stage study, which BioMarin submitted earlier this year, and will make a decision by June 30.
End-to-end vector manufacturing optimizes batch success and delivers more doses at lower cost.
