BioMarin Pharmaceutical Inc. (BMRN) $39M Shareholder Settlement

BioMarin Pharmaceutical Inc. (BMRN) agreed to settle $39 million with investors.

On December 23, 2019, BioMarin Pharmaceutical Inc. submitted a Biologics License Application to the FDA for its investigational AAV gene therapy.

BioMarin assured investors that the FDA approval process was “going quite well,” that the FDA had been “quite collaborative” and in a “mesh” with Defendants, and that the drug would “launch in the second half of 2020".

Meanwhile, the company allegedly knew of the FDA’s concerns that the initial Phase 3 trial data showed potential issues with the long-term effectiveness of the drug due to a decline in Factor VIII levels in patients over time; and that company had no dialogue whatsoever with the FDA from mid-April 2020 to June 2020, at which point Defendants learned that a facility inspection—required for FDA approval—would not occur prior to August 21, 2020.

On August 19, 2020, when BioMarin announced that the Company had received a Complete Response Letter from the FDA, which included the FDA’s concerns about the initial Phase 3 data’s indication of declining durability of effect. BioMarin disclosed that the FDA declined to approve the BLA and would require that BioMarin complete its Phase 3 trial and submit two-year follow-up safety and efficacy data on all participants before any possible approval. Accordingly, FDA approval of BioMarin’s BLA for Valrox cannot happen until at least 2022. In response, BioMarin’s stock price dropped from from $118.16 to $76.72.