U.S. Markets closed
  • S&P 500

    4,538.43
    -38.67 (-0.84%)
     
  • Dow 30

    34,580.08
    -59.71 (-0.17%)
     
  • Nasdaq

    15,085.47
    -295.85 (-1.92%)
     
  • Russell 2000

    2,159.31
    -47.02 (-2.13%)
     
  • Crude Oil

    66.22
    -0.28 (-0.42%)
     
  • Gold

    1,782.10
    +21.40 (+1.22%)
     
  • Silver

    22.45
    +0.17 (+0.76%)
     
  • EUR/USD

    1.1317
    +0.0012 (+0.1019%)
     
  • 10-Yr Bond

    1.3430
    -0.1050 (-7.25%)
     
  • Vix

    30.67
    +2.72 (+9.73%)
     
  • GBP/USD

    1.3235
    -0.0067 (-0.5029%)
     
  • USD/JPY

    112.8000
    -0.4090 (-0.3613%)
     
  • BTC-USD

    48,401.95
    -7,829.81 (-13.92%)
     
  • CMC Crypto 200

    1,367.14
    -74.62 (-5.18%)
     
  • FTSE 100

    7,122.32
    -6.89 (-0.10%)
     
  • Nikkei 225

    28,029.57
    +276.20 (+1.00%)
     

FDA Slaps Clinical Hold on BioMarin's Gene Therapy For Genetic Metabolism Disorder

  • Oops!
    Something went wrong.
    Please try again later.
·1 min read
In this article:
  • Oops!
    Something went wrong.
    Please try again later.
  • ​​​​​​BioMarin Pharmaceutical Inc (NASDAQ: BMRN) faces another setback in its gene therapy development program after the FDA placed the phenylketonuria (PKU) gene therapy trial on hold.

  • The Phase 1/2 Phearless study is evaluating BMN 307, an AAV5-phenylalanine hydroxylase (PAH) gene therapy for PKU.

  • PKU is a rare genetic disease that manifests at birth and is characterized by an inability to break down phenylalanine, an amino acid commonly found in many foods.

  • The agency ordered the pause on the study after liver tumors were seen in mice given the therapy in preclinical testing.

  • BioMarin’s preclinical study was carried out to explore the durability of BMN 307 activity in mice with two germline mutations that may predispose them to cancer development.

  • One mutation eliminated the PAH gene that’s missing in PKU, while the second rendered the animals immunodeficient, which could also raise the risk of malignancy.

  • Of 63 animals treated, six of seven animals administered BMN 307 at the highest dose group – 2e14 Vg/kg – developed liver tumors.

  • Five of the mice had adenomas, and one had hepatocellular carcinoma.

  • So far, participants have received doses of either 2e13 vg/kg or 6e13 vg/kg of BMN 307, although the protocol does include a third, higher-dose group.

  • Read Next: Drug For Short-Limbed Dwarfism From Biomarin Wins European Approval.

  • The clinical hold for the PKU program comes after BioMarin’s hemophilia A gene therapy was rejected by the FDA last summer.

  • The rejection would delay the approval by around two years, as the FDA asked for two-year data on all study participants.

  • The last patient will complete two years of follow-up in November 2021.

  • Related content: Benzinga's Full FDA Calendar.

  • Price Action: BMRN shares closed at $84.95 on Friday.

See more from Benzinga

© 2021 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.