On August 19, 2020, BioMarin Pharmaceutical, Inc.(NASDAQ: BMRN) stunned the markets when it announced that the U.S. Food and Drug Administration had issued a Complete Response Letter (CRL) to the Company’s Biologics License Application (BLA) for valoctocogene roxaparvovec (VR)gene therapy for severe hemophilia A. In the CRL, the FDA informed BioMarin that the Company’s BLA was not ready for approval. On this news, BioMarin’s stock fell $41.82 per share, or over 35%, in just one day.
In the CRL, the FDA concluded that “the differences between” BioMarin’s Phase 1/2 study and the Phase 3 study for VR “limited [the FDA’s] ability to rely on the Phase 1/2 study to support durability of effect.” The FDA therefore recommended that BioMarin complete its Phase 3 study (not due to conclude until November 2021) and that BioMarin submit two-year follow-up safety and efficacy data on all study participants.
A lawsuit alleging violations of federal securities laws has been filed against BioMarin and certain of its officers and directors. The suit alleges that beginning on February 28, 2020, BioMarin misled investors about its BLA, namely, that the Company misrepresented the differences between its Phase 1/2 and Phase 3 studies for VR, which limited the reliability of the Phase 1/2 study to support FDA approval. According to the lawsuit, the August19, 2020 disclosure of the CRL from the FDA shocked the market because of the Company’s previous statements about the BLA and the reliability of the Phase1/2 study.