Halloween adcomm postponed as FDA seeks more Ipsen data on drug candidate for ultra-rare disorders – Endpoints News
In another setback for Paris-based Ipsen, the FDA is postponing an adcomm meeting set for Oct. 31 to review new information about the company’s previously rejected potential treatment for patients with fibrodysplasia ossificans progressiva, an ultra-rare genetic disorder.
The FDA said the delay is “to allow time for the FDA to review new information,” while Ipsen said “the delay relates to an FDA request for new information on palovarot clinical trial data and is not related to the safety profile of palovarot. The company is currently working on to fulfill the request.”
But a future date for the FDA’s Endocrinology and Metabolic Drugs Advisory Committee meeting has not yet been set, and it remains unknown whether palovarotene’s FDA decision date, slated for Dec. 29, will be affected by the new adcomm date.
Shares in Ipsen stock $IPN.PA fell around 5% on Tuesday. That adcomm meeting will cap a rocky plus three years until a final decision.
Ipsen acquired the drug following a $1 billion-plus acquisition of Canadian biotech Clementia Pharmaceuticals in February 2019, which had repurposed it after Roche’s move into emphysema trials.
But in December 2019, the FDA gave Ipsen a partial hold on dosing patients 14 and under in a Phase II and Phase III trial after reports of “early growth plate closure,” a condition that can severely stunt a child’s growth. The next month, the drug failed a phase III futility test, and Ipsen decided to pause dosing in all remaining pediatric patients from that trial.
However, Ipsen appeared to improve the trial in a short time, and resumed dosing a few months later after changing the protocols. The company later won priority review from the FDA in May 2021, but Ipsen decided to withdraw the application in August 2021, citing the FDA’s request for additional data from its two pivotal studies.
Jefferies analyst Peter Welford said at the time that the withdrawal of the NDA did not change his estimate of $180 million in peak sales, which also assumed a 2021 approval.
Ipsen submitted its application again early this summer, and again received a priority assessment. The company applied for approval in FOP in adults and children, including girls over eight and boys over 10. The drug was approved in Canada last January.