April 2025 eNewsletter

What is FOP? | Clinical Trials | HCP Education

Have a colleague interested in FOP or other rare bone diseases?

Forward them this email and they can sign up to receive it quarterly.

Welcome to the first edition of FOP Link, an eNewsletter created by the International FOP Association (IFOPA) to connect healthcare professionals to the latest news in FOP research and care.

The IFOPA was founded in 1988 to connect and support individuals and families living with FOP. Two years later, we began funding FOP research. Since then, we have funded research at academic institutions around the world, built research and clinical care tools like the FOP Registry, and launched the only scientific meeting dedicated to FOP research—the FOP Drug Development Forum.

Today, the IFOPA is the largest organization serving people with FOP and their families around the globe on topics from disease management to clinical studies & trials, adaptive tools, independent living, and emotional health. Translation services are available for most of our webinars and many of our programs, like our Online Caregivers Support Group, are designed for all members of the family. This eNewsletter will always feature resources you can share with your FOP patients and families.

We are pleased to bring you this eNewsletter to continue generating awareness of FOP and ensure that those caring for FOP patients are connected to educational resources and organizations to support you in caring for your patients.

If you have questions or ideas you'd like to share, please don't hesitate to reach out.

Best,

Michelle Davis

Executive Director

michelle.davis@ifopa.org

(816) 809-2772 (also available on WhatsApp)

Enrollment Update: FOP Clinical Trials

For an ultra-rare disease with a one-in-a-million prevalence, the FOP clinical trial space is very active. The IFOPA offers a suite of resources for patients and healthcare providers, including a clinical trial locator map and frequently asked questions about drug development and clinical trials.

Visit ifopa.org and click "Clinical Studies & Trials" in the Quick Links on the home page to access all of our resources.

There are two clinical trials active and recruiting:

• Regeneron Phase 3 OPTIMA trial – fully enrolled at 63
• STOPFOP Phase 2 trial – fully enrolled at 17
• Ipsen Phase 2 FALKON trial – fully enrolled at 113
• Incyte Phase 2 PROGRESS trial – enrolling soon: Cohort 2 children ages 6 to <12 at sites around the world
• āshibio ANDECAL Phase 2 trial – enrolling children ages 12 and up and adults in the US

Questions? Contact us at together@ifopa.org

Update on Treatments Approved for FOP

At this time, there is only one approved treatment for FOP. Ipsen's palovarotene (now known as Sohonos^TM) has been approved for the treatment of FOP in females aged 8 and over and males aged 10 and over in Canada, the United States, and Australia. Palovarotene has also received provisional approval in the United Arab Emirates. 

FOP is caused by a mutation in the ACVR1 gene that results in excessive bone morphogenetic protein (BMP) signaling, which regulates cartilage and bone development. The FOP mutation increases BMP signaling, resulting in the formation of heterotopic bone. By reducing BMP signaling, palovarotene may reduce the volume of heterotopic bone formed in FOP patients.

For more information, visit sohonos.com or ipsenmedicalinformation.com.

In August 2023, the International Clinical Council on FOP (ICC) issued this policy statement on palovarotene. 

Have You Seen Our Healthcare Provider Education Resources?

The IFOPA is pleased to connect you to FOP awareness and education programs you can participate in online. Some of these programs even offer continuing medical education (CME) credit. These programs have been developed and sponsored by other organizations or companies, with content provided by FOP experts. You will find the developer and sponsor of each program in the program summary.

Be on the lookout for new educational program offerings in 2025. Also, watch for IFOPA staff at various US-based medical conferences, including the American Academy of Pediatrics and the American Academy of Family Physicians' FMX.

Visit ifopa.org/fop-hcp-education to see all of the resources available to you free of charge. 

Did You Know the IFOPA Provides Patient-Friendly Information on FOP Management & Care?

The IFOPA is committed to sharing patient-friendly information on how to manage everyday FOP symptoms, care for the FOP body, and implement good safety practices.

We even have a resource on explaining FOP to children for your pediatric families.

This is great information to share with your patients and their caregivers in the clinic to help empower them to take charge of their health.

Learn more >

IFOPA Also Offers Dental Resources

Recently, in celebration of World Oral Health Day, the IFOPA released a new and improved FOP Oral Health Resource Hub with a variety of tools to help empower people living with FOP to practice the best oral health care. 

These materials were designed with FOP-specific recommendations and expert insights to guide patients in protecting not just their dental health but overall well-being.

Explore the IFOPA's Oral Health Resource Hub >

Updated FOP Treatment Guidelines

The International Clinical Council on FOP (ICC) is an autonomous and independent group of internationally recognized physicians who are clinical experts in FOP. The ICC was established in 2017 to coordinate and consolidate a global voice for best practices in clinical care and clinical research for people who suffer from FOP.

In July 2024, the ICC issued updated FOP Treatment Guidelines, which they originally authored in 2009. The Treatment Guidelines contain detailed medical information and guidelines on the symptomatic management of FOP. To ensure the widest distribution, guidelines are written in English and are accessible to physicians worldwide at iccfop.org/guidelines.

The FOP Treatment Guidelines include contact information for FOP specialists around the world, starting on page 153).

Questions? Contact Editors Fred Kaplan, MD or Bob Pignolo, MD

April 23 is Global FOP Awareness Day

Each year, the IFOPA celebrates Global FOP Awareness Day on April 23—the day it was announced in 2006 that the gene that causes FOP had been discovered.

Since 2021, the IFOPA has come together with FOP organizations around the world to raise awareness of FOP and the lived experience of people with FOP. 

Throughout the month of April, we'll be sharing FOP facts and sharing video stories on our social media (Facebook, Instagram, Twitter/X,

and LinkedIn).

This year, we are offering a new series, "Partnering in Care – FOP Healthcare Provider Perspectives".

Meet Dr. Peter Kannu, Chair of Medical Genetics at the University of Alberta, as he shares his deep experience working with FOP and powerful insights on how healthcare providers can truly make a difference in the lives of those living with this rare and complex condition.

Watch our social media for more videos in this series from Dr. Johannes Roth, Luzerner Kantonsspital, Switzerland, and Dr. Raphaella Stander, University of Cape Town.

Learn more and get involved at ifopa.org/awareness_day_2025

If you'd like to discuss the availability of resources to help you raise awareness of FOP locally, please reach out to together@ifopa.org.

Thank You to Our Global FOP Awareness Day Sponsors

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