Ipsen Withdraws Application for Ultra-Rare Bone Disease Treatment
Ipsen has withdrawn its new drug application (NDA) for palovarotene, an experimental treatment for an ultra-rare genetic disorder that causes tendons and ligaments to be replaced by bone.
The FDA granted the NDA a Priority Review in late May, but the Paris-based drugmaker said recent discussions with the agency revealed the need for additional analyses of a phase 3 trial that is investigating palovarotene for treating the disorder known as fibrodysplasia ossificans progressive.
The FDA’s target action date on the NDA was Nov. 30.
“Unfortunately, as there is no regulatory mechanism to ‘pause’ the current ongoing review process, we have taken the decision to withdraw the NDA for palovarotene,” said Howard Mayer, the company’s head of research and development. Mayer added that the company plans to resubmit its application for the drug “as soon as possible.”