Palovarotene Regulatory Submission Accepted

We’re pleased to share breaking news from Ipsen. Palovarotene, the first potential treatment for FOP, will be considered for approval by the US Food and Drug Administration (FDA), European Medicines Agency and Swissmedic MAA in Switzerland!

It is important to share that this does NOT mean that palovarotene has been approved as a prescribed medication for FOP; it means that it is being reviewed and considered by government authorities.

Regulatory submission is the process through which pharmaceutical companies submit the information about their proposed drug treatment to a regulatory agency or agencies for review and a decision on approval. Acceptance of Ipsen’s regulatory submission package means that regulators (in this case the FDA, EMA and Swissmedic) have taken a preliminary look at the data and feel that it is sufficient to conduct a thorough review and make a decision whether palovarotene can be prescribed for the treatment of FOP.

Ipsen has been granted priority review by the FDA, fast-track by Swissmedic and standard review by the EMA. FDA priority review designation means their goal is to take action on an application within 6 months (compared to 10 months under standard review). Swissmedic fast-track review is 140 days. The EMA standard review timeline is 12 months.

The FDA’s PDUFA date is November 30, 2021. PDUFA is the date by which the FDA is expected to deliver its decision on whether or not to approve Ipsen’s New Drug Application (NDA) for palovarotene as a treatment for FOP.

It’s important to remember that each country has its own version of the FDA and drugs must be approved in each country in order for physicians located there to prescribe them. Decisions on whether or not insurance companies or national health services will pay for a drug will only be made following approval.

Any decision as to which patients (e.g. by age group) can be prescribed a drug will be made during the review process.

This is the first time a drug to treat FOP has made it this far in the drug development cycle. We are grateful to each of you that have participated in the Ipsen (formerly Clementia) Natural History Study and the MOVE Trial. Without your support and involvement, this critical research would not have advanced. The same is true for those participating in the FOP Registry and other studies and trials, you are serving in a role that only you can fill. Thanks to each of you for advancing FOP research!

We are also grateful to the teams at Clementia and Ipsen for the investment they have made in researching this potential treatment for those living with FOP and their families.

Read Ipsen’s full press release.

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