February 22, 2021 - An amended complaint was filed.

September 25, 2020 - An investor in shares of BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) filed a lawsuit in the U.S. District Court for the Northern District of California over alleged violations of Federal Securities Laws by BioMarin Pharmaceutical Inc. in connection with certain allegedly false and misleading statements made between February 28, 2020 and August 18, 2020.

San Rafael, CA BioMarin Pharmaceutical Inc., a biotechnology company, develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. Based on BioMarin Pharmaceutical Inc's Phase 1/2 study results, the investing and medical community viewed valoctocogene roxaparvovec as a likely candidate for becoming the first gene therapy approved by the U.S. Food and Drug Administration ( FDA ) for hemophilia in the U.S. In May 2019, BioMarin Pharmaceutical Inc announced the interim results from its Phase 3 study of valoctocogene roxaparvovec for adults suffering from severe hemophilia A. The results, although showing the treatment's effectiveness, disappointed the market because they indicated a reduction in durability of effectiveness from the results shown in the Phase 1/2 results. That is, under the Phase 3 results, it was not clear whether valoctocogene roxaparvovec's effectiveness would last as long as indicated in the Phase 1/2 study, and thus, whether valoctocogene roxaparvovec would be a single treatment, or one requiring more than one treatment over the life of a patient. These concerns echoed those that first arose in 2018, after the Company reported a disappointing two-year update from the Phase 1/2 study, which showed factor VIII levels waning over time. In December 2019, BioMarin Pharmaceutical Inc submitted a Biologics License Application ( BLA ) to the FDA for valoctocogene roxaparvovec for adults with hemophilia A based on the interim analysis of the ongoing Phase 3 study of valoctocogene roxaparvovec, as well as three-year data from the Company's Phase 1/2 study of valoctocogene roxaparvovec. BioMarin also announced that the European Medicines Agency ( EMA ) validated the Company's Marketing Authorization Application ( MAA ) for valoctocogene roxaparvovec for adults with severe hemophilia A, with the MAA review to commence in January 2020 under accelerated assessment.

On August 19, 2020, BioMarin Pharmaceutical Inc announced receipt of a Complete Response Letter ( CRL ) from FDA regarding the Company’s BLA for valoctocogene roxaparvovec. Therein, the FDA concluded that the differences between Study 270-201 (Phase 1/2) and the Phase 3 study limited its ability to rely on the Phase 1/2 study to support durability of effect. As a result, the FDA recommended that BioMarin complete the Phase 3 Study and submit two-year follow-up safety and efficacy data on all study participants. Shares of BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) declined from $131.95 per share on July 21, 2020, to as low as $71.35 per share on September 11, 2020.

According to the complaint the plaintiff alleges on behalf of purchasers of BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) common shares between February 28, 2020 and August 18, 2020, that the defendants violated Federal Securities Laws.

More specifically, the plaintiff claims that between February 28, 2020 and August 18, 2020, the defendants made false and/or misleading statements and/or failed to disclose that 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, that as a result, it was foreseeable that the FDA would not approve the BLA for valoctocogene roxaparvovec without additional data, and that as a result, the Company's public statements were materially false and misleading at all relevant times.