Shares of BioMarin Pharmaceutical Inc. (BMRN) are falling more than 8 percent or $7.67 in Monday’s morning trade at $82.19.
Sunday, BioMarin announced positive phase 3 gene therapy trial results in adults with severe hemophilia A, noting that the study met all primary and secondary efficacy endpoints in one-year data set.
Data from the GENEr8-1 phase 3 study enrolling 134 participants with a mean follow-up of 71.6 weeks showed that in the pre-specified primary analysis for annualized bleeding rate or ABR, a single dose of valoctocogene roxaparvovec, an investigational gene therapy, significantly reduced ABR by 84 percent. 80 percent of participants were bleed-free starting at week five after treatment.
BioMarin said that the U.S. Food and Drug Administration or FDA recommended that the company complete the Phase 3 study and submit two-year follow-up safety and efficacy data on all study participants. The company plans to meet with the FDA to review the two-year data request.
Additionally, the European Medicines Agency or EMA has requested one-year results from the full Phase 3 study to inform their benefit-risk assessment. BioMarin said it withdrew the MAA and plans to resubmit the MAA with these data to the EMA in the second quarter of 2021 following discussions with the agency.
BioMarin Pharma has traded in a range of $68.25 to $131.95 in the past 52 weeks.
Source: Read Full Article
Most El Salvador Citizens Not Keen on Accepting Bitcoin as Legal Tender
Corporate Bitcoin Holdings Are Thriving At An Alarming Rate
Missed The Bitcoin Dip? Analysts Pinpoint Next Entry Zones To Watch Out For
Nigeria’s SEC on Bitcoin: “The World Cannot Be Moving Forward and We Will Be Static”
Bitcoin ($BTC) Investors Should Be ‘Accumulating’ Before Rise to $150,000: Bloomberg Analyst