Ipsen Stock Declines After Pulling Rare Disease Application

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.