Canceled AdCom, UniQure's Hemgenix Approval Show Positive Signs For BioMarin's Hemophilia Candidate

  • Earlier today, the FDA notified BioMarin Pharmaceutical Inc BMRN that the agency no longer plans to hold an advisory committee meeting to discuss the Biologics License Application (BLA) of Roctavian (valoctocogene roxaparvovec AAV gene therapy) for adults with severe hemophilia A. 
  • Previously, the FDA communicated to the company that it did intend to hold an advisory committee meeting but did not specify a date. 
  • Also read: FDA Approves UniQure's Hemgenix, First Hemophilia B Gene Therapy For Adults.
  • With canceled AdCom, the focus now shifts to the three-year data expected in early 2023, particularly the trend in FVIII levels and annualized bleed rates (ABR) from year 2 to year 3.
  • With an Outperform ratingWilliam Blair writes that the data can be considered a major amendment and trigger a three-month extension to the current March 31 PDUFA date. 
  • The analyst believes the three-year data needs to show a mean ABR <1 and median of 0 and continues to believe this is ultimately the key endpoint given the imperfect correlation between FVIII expression to ABR.
  • The analyst says the cancelation of an AdCom is an incremental positive. However, given gene therapies' recent success in their AdComs, a potential AdCom would not be as much of a risk. 
  • Price Action: BMRN shares are up 7.35% at $97.88 on the last check Wednesday.
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