According to the Complaint, BioMarin Pharmaceutical Inc. is a biotechnology company that develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. The Company's product candidates include, among others, valoctocogene roxaparvovec, an investigational AAV gene therapy, which is in Phase 3 clinical development for the treatment of patients with severe hemophilia A.

The Complaint alleges that throughout the Class Period, Defendants made materially false and misleading statements regarding the Company's business, operational, and compliance policies. Specifically, the Complaint alleges Defendants made false and/or misleading statements and/or failed to disclose that: (i) differences between the Phase 1/2 and Phase 3 study of valoctocogene roxaparvovec limited the reliability of the Phase 1/2 study to support valoctocogene roxaparvovec's durability of effect; (ii) as a result, it was foreseeable that the FDA would not approve the BLA for valoctocogene roxaparvovec without additional data; and (iii) as a result, the Company's public statements were materially false and misleading at all relevant times.