Submit Your Written Testimony by June 14

Today, the U.S. Food & Drug Administration (FDA) announced that the Endocrinologic and Metabolic Drugs Advisory Committee has rescheduled their meeting to review Ipsen's new drug application (NDA) 215559 for palovarotene capsules for June 28, 2023.

A YouTube link for the June 28 meeting, and all meeting materials, will be made public on June 26. The IFOPA will provide this YouTube link and meeting materials once they are publicly posted by FDA. 

At the end of the meeting on June 28, the Advisory Committee will make a recommendation for or against approval to the FDA. It is important to note that the Committee’s recommendation is NOT the final decision. That decision will be announced later this year – by August 16 (also known as the PDUFA date).

The global FOP community has a valuable role to play in the FDA’s review of palovarotene. As part of their review, Advisory Committee members will have an opportunity to read letters submitted from the FOP community.

It is important to note that the IFOPA has not seen Ipsen’s data and is neutral in our support of the FDA’s approval of palovarotene. What we do believe is critically important is that the FDA and this Advisory Committee hear what it’s like to live with and care for someone with FOP. That is a story only you can tell and why the IFOPA is encouraging you to provide written testimony. 

• If you submitted a letter in 2022, you do not need to submit a second letter. Unsure if you've already submitted your comments? Click here to see letters submitted from August-October 2022
• If you would like to submit a letter, the deadline is June 14, 2023

What You Need to Know About Submitting a Letter for Written Comment

• This written comment opportunity is open to all members of the FOP community worldwide, not just U.S. residents, but all letters must be submitted in English.
• Letters kept to one page in length are more likely to be read. Letters should address the following:
  • Your FOP story as a means of conveying concrete examples of the impact of FOP on day-to-day living including:
    • Physical effects of FOP
    • Psychological impacts of FOP
    • Financial impacts of FOP
    • Caregiving responsibilities
  • Hope for how a drug treatment could protect or maintain your quality of life
  • Your outlook as you consider drug treatments and weigh potential risks and benefits
• Along with their written letters, individuals and families are encouraged to submit one page of photos to help the committee understand the physical impacts of FOP
• Letters must be submitted on or before June 14, 2023 
• Comments submitted electronically, including attachments, will be posted to the docket unchanged. Your comments will be made public so you are solely responsible for ensuring that your comment does not include any confidential information that you or a third party may not wish to be posted, such as medical information, your or anyone else’s Social Security number, or confidential business information. If you include your name, contact information, or other information that identifies you in the body of your comments, that information will be posted on https://www.regulations.gov.
• Comments can be submitted electronically at https://www.regulations.gov/commenton/FDA-2022-N-1778-0001. You can write directly on the webpage and/or attach a Word or PDF file with your commentary. For the drop-down question, “What is your comment about?” the question is asking who is submitting the comment – choose “Individual Consumer.”

Please contact Hope Newport, IFOPA’s Family Services Manager, at [email protected] with any questions you may have regarding submitting a written comment, the Advisory Committee meeting or palovarotene’s priority review by the FDA.

Timeline of FDA Events Leading Up to Today's Announcement

Note: This is not a summary of all regulatory review activity related to palovarotene, but only a summary of activity made publicly available related to the review by the U.S. Food & Drug Administration 

July 21, 2014 - FDA grants Orphan Drug Designation to Clementia's (now Ipsen's) palovarotene

October 5, 2017 - FDA grants Breakthrough Therapy Designation to Clementia's (now Ipsen's) palovarotene

October 23, 2018 - Clementia (now Ipsen) announces plan to submit a New Drug Application for palovarotene for the treatment of FOP

February 11, 2019 - FDA grants Rare Pediatric Disease Designation to Clementia's (now Ipsen's) palovarotene

May 28, 2021 - Ipsen announces that their New Drug Application (NDA) for palovarotene capsules has been accepted for Priority Review 

August 13, 2021 - FDA requests additional data analyses and evaluation of data collected from Ipsen’s Phase III MOVE Trial and FOP program

June 29, 2022 - Ipsen announces that the resubmission of their NDA for palovarotene capsules to FDA has been accepted for Priority Review

August 23, 2022 - FDA announces Public Advisory Committee Meeting for Ipsen's palovarotene capsules

October 25, 2022 - FDA postpones Public Advisory Committee Meeting for palovarotene capsules

December 27, 2022 - Ipsen announces receipt of Complete Response Letter from FDA

March 16, 2023 - Ipsen receives new PDUFA date from FDA