You’re Invited to Submit Written Testimony to the US FDA

Advisory Committee wants to hear about your experience living with or caring for someone with FOP

On June 29, 2022, Ipsen shared the announcement that their resubmission to the US Food & Drug Administration (FDA) has been accepted for priority review. This is significant news for the FOP community as there is potential that palovarotene could become the first drug approved to treat FOP in the United States.

On August 23, the FDA shared an announcement about the next step in their review of palovarotene. On October 31, 20022, the Endocrinologic and Metabolic Drugs Advisory Committee will discuss new drug application (NDA) 215559 for palovarotene capsules submitted by Ipsen Biopharmaceuticals, Inc. The proposed indication is the prevention of heterotopic ossification in adults and children (females aged 8 and above and males 10 years and above) with fibrodysplasia ossificans progressiva (FOP). 

A YouTube link for the October 31 meeting, and all meeting materials, will be made public on October 29. The IFOPA will provide this YouTube link and meeting materials once they are publicly posted by FDA. 

At the end of the meeting on October 31, 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 December 29 (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.

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 US 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 October 17, 2022 and must include Docket No. FDA-2022-N-1778
  • 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 first drop-down question, “What is your comment about?” the question is asking who is submitting the comment – choose “Individual Consumer.”
  • If you prefer to submit your comments via mail, please contact [email protected] for instructions. Keep in mind that your written comments must be received by the FDA no later than October 17, 2022 or they will not be shared with the Advisory Committee. Electronic submission is the preferred method of delivery.

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.

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