Ipsen Stock Declines After Pulling Rare Disease Application

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  • Ipsen IPSEY withdrew its US marketing application for palovarotene following discussions with the FDA indicating additional data analyses needed from two pivotal trials.
  • Palovarotene is an oral, selective RARγ agonist to prevent heterotropic ossification (new bone formation) as a potential treatment for ultra-rare genetic disorder fibrodysplasia ossificans progressiva (FOP).
  • The action date under a Priority Review status was 30 November.
  • The extra work will take too long to fit into the review cycle, forcing Ipsen to pull the submission and ending hopes of bringing palovarotene to market this year.
  • Ipsen plans to resubmit to the FDA upon completion of the additional data analyses.
  • The move surprised some as Ipsen had made strides to resolve issues stemming from a partial clinical hold in 2019 and a failed futility test soon after.
  • In December 2019, regulators slapped Ipsen with a partial hold on patients 14 years & younger dosing in Phase 2 and Phase 3 studies. 
  • The hold resulted from "early growth plate closure" reports, a condition that can severely stunt a child's growth.
  • In January 2020, the Independent Data Monitoring Committee review said the program failed a Phase 3 futility test, resulting in Ipsen pausing dosing in all remaining pediatric patients.
  • Price Action: IPSEY shares are down 8.66% at $24.25 on the last check Friday.
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