BioMarin Pharmaceuticals Inc.

Case Details

Class Period: February 28, 2020 - August 18, 2020
Date Filed: September 25, 2020
Case Number: 3:20cv06719
Jurisdiction: California Northern District Court
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Case Summary

BioMarin 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 adeno-associated virus (“AAV”) gene therapy, which is in Phase 3 clinical development for the treatment of patients with severe hemophilia A. Based on BioMarin’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 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. Apparently dismissing these concerns, in December 2019, BioMarin 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. 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, 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. On August 19, 2020, BioMarin announced receipt of a Complete Response Letter (“CRL”) from the FDA to the Company’s BLA for valoctocogene roxaparvovec. BioMarin advised investors that in the CRL, “the FDA introduced a new recommendation for two years of data from the Company’s ongoing 270-301 study (Phase 3) to provide substantial evidence of a durable effect using Annualized Bleeding Rate (ABR) as the primary endpoint” and “recommended that the Company complete the Phase 3 Study and submit two-year follow-up safety and efficacy data on all study participants.” In explaining the new recommendation, 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 the durability of effect.” On this news, BioMarin’s stock price fell $41.82 per share, or 35.28%, to close at $76.72 per share on August 19, 2020.