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BioMarin drug acquired in buyout misses goal in rare disease study

Tested in a condition known as ENPP1 deficiency, the treatment failed a trial goal that’s important to regulators, spurring doubts about its approval prospects.

Raccontata dabiopharmadive.comendpoints.news

Confronto fonti

2 prospettive sulla stessa storia
AI · summaries
biopharmadive.comStai leggendo1 mesi fa

BioMarin drug acquired in buyout misses goal in rare disease study

Tested in a condition known as ENPP1 deficiency, the treatment failed a trial goal that’s important to regulators, spurring doubts about its approval prospects.

originale
endpoints.news1 mesi fa

BioMarin's rare disease therapy shows no clinical benefit in Phase 3 test

BioMarin Pharmaceutical’s enzyme replacement therapy for a rare genetic disorder called ENPP1 deficiency delivered mixed results in a late-stage study.

Leggi questa versione → originale

Timeline cronologica

  1. martedì 19 maggio 2026·biopharmadive.com

    BioMarin drug acquired in buyout misses goal in rare disease study

    Tested in a condition known as ENPP1 deficiency, the treatment failed a trial goal that’s important to regulators, spurring doubts about its approval prospects.

  2. martedì 19 maggio 2026·endpoints.news

    BioMarin's rare disease therapy shows no clinical benefit in Phase 3 test

    BioMarin Pharmaceutical’s enzyme replacement therapy for a rare genetic disorder called ENPP1 deficiency delivered mixed results in a late-stage study.

  • giovedì 21 maggio 2026·biopharmadive.com

    BioMarin notches win in study that could expand use of top-selling medicine

    The success came two days after a Phase 3 setback for another experimental drug and helps improve the position of Voxzogo amid increased competition.