According to the Complaint, BioMarin Pharmaceutical Inc. develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. The Company is developing, among other product candidates, BMN 307, an AAV5 mediated gene therapy, which is in a phase 1/2 clinical trial to normalize blood phenylalanine concentration levels in patients with phenylketonuria ("PKU"). The Company’s Phearless Phase 1/2 study is evaluating BMN 307 in adults with PKU.

The Complaint alleges that, throughout the Class Period, Defendants made materially false and misleading statements regarding the Company's business, operations, and compliance policies. Specifically, the Complaint alleges Defendants made false and/or misleading statements and/or failed to disclose that: (i) BMN 307 was less safe than BioMarin had led investors to believe; (ii) BMN 307's safety profile made it likely that the FDA would place a clinical hold on the Phearless Phase 1/2 study; (iii) accordingly, the Company had overstated BMN 307's clinical and commercial prospects; and (iv) as a result, the Company's public statements were materially false and misleading at all relevant times.

On January 10, 2022, the Court issued an Order appointing Lead Plaintiff and Counsel. Lead Plaintiff filed an amended Complaint on March 25. Defendants filed a Motion to Dismiss the amended Complaint on May 25. On January 19, 2023, the Court issued an Order granting Defendants' Motion to Dismiss. Plaintiff was given leave to amend the Complaint.

On February 21, 2023, Plaintiff filed a “Notice of Lead Plaintiffs’ Election to Stand on the Consolidated Amended
Complaint for Violations of the Federal Securities Laws.” Accordingly, the Court dismissed the case with prejudice and directed the Clerk of Court to close the file.

Lead Plaintiffs filed a notice appealing the Court's Dismissal Order on March 22, 2023. On February 15, 2024, the Court of Appeals affirmed the District Court's ruling.