On September 28, 2023, Regeneron published the results of their Phase 2 LUMINA-1 Clinical Trial of the drug garetosmab in Nature Medicine, a peer-reviewed publication.
Regeneron also published clinical pharmacology results from the LUMINA-1 Trial in the Journal of Clinical Pharmacology.
Also on September 28, Vanderbilt University Medical Center, one of the sites for the LUMINA-1 Trial, issued this press release announcing the peer-reviewed publication of Phase 2 results and sharing the story of Sharon Kantanie, a trial participant.
The IFOPA is grateful to the 44 individuals with FOP who enrolled in the LUMINA-1 Phase 2 Trial, including the five community members who passed during the open-label period. The willingness of families to participate in clinical trial research is the only way we will ever learn if drugs are safe and effective treatments for FOP. We also appreciate the work of the trial investigators and their commitment to FOP families. We are also grateful to Regeneron for their commitment to the FOP community and the development of garetosmab.
The investigation of garetosmab as a treatment for FOP has now moved forward in the Phase 3 OPTIMA trial. You can learn more at ifopa.org/regn2477.
From a readership of 11 in 1988 to more than 2,000 in 2023, the IFOPA newsletter has evolved to inform and connect friends across the world
Together with her sister-in-law, Anne, and fellow IFOPA founding member, Nancy Sando, Jeannie Peeper published the first edition of the FOP Connection in 1988. Back then, it looked quite a bit different from the version you’re reading today.
“It was very basic,” said Jeannie. “It was done on a typewriter, copied and mailed out.”
Jeannie got the idea to bring people living with FOP together after Dr. Michael Zasloff gave her the names of 18 other people with FOP.
“I wanted to help end the isolation many of us felt,” she said.
Jeannie wrote to the others to gather details like names, birthdays and hobbies, and 11 of the 18 responded. Jeannie invited those that wrote back to be Founding Members of the IFOPA. Information they sent back helped create the first newsletter.
“It was a way to share everyone’s information, connect and build friendships,” she said.
In 2010, FOP Connection moved online.
Thirty-five years later, the FOP Connection still functions in a similar manner, just on a massively different scale. A readership of 11 in 1988 has exploded to more than 2,000 in 2023.
Just like our organization, it’s remarkable to see how the newsletter has evolved to serve a growing audience yet remained committed to providing a personal connection and welcoming community for everyone.
FOP Connection provides people across the world with the latest research and clinical trial news, information about IFOPA programming, community updates, support and more to better navigate life with FOP.
It’s been so special to see FOP Connection blossom into what it is today.
Thank you, Jeannie, for creating such a wonderful way for our community to connect!
L.I.F.E. Award Grant makes it easier for professional with FOP to work from home
Kathleen Degenhardt works from home. She also has FOP, which makes it difficult for her to perform some tasks. She learned about the Harold & Elaine Kaplan Quality of L.I.F.E. Award program while reading the IFOPA monthly newsletter and applied.
The program provides grants to people living with FOP. Recipients can use the assistance to purchase tools and make improvements to help them perform daily tasks. Whether that be in the form of home modifications or comfort, school, workplace, communication or mobility aids is up to them. Kathleen used it to purchase an adjustable-height desk.
“It’s made working from home easier because instead of using books to make my keyboard the correct height, I’m able to press a button to do it,” she said. “As my body changes, I can easily adjust it so I can continue to work.”
Now that she can sit straighter and be more comfortable while working, Kathleen said that many tasks have become easier and are more enjoyable. She is grateful for the improvement and hopes the program can help others.
“As members of the FOP community, we live challenging lives and struggle to do everyday tasks,” she said. “Sometimes simple things can make such a big difference and make life easier.”
As the IFOPA continues to expand and reach new communities around the world, demand for this type of assistance is growing. For those looking to help people living with FOP, supporting the Quality of L.I.F.E. Awards is one of the best ways to have a direct impact.
“Your support of the program means a lot and provides all sorts of items to FOP members,” Kathleen said. “This kind of immediate, personalized assistance has been life-changing.”
Learn more about the L.I.F.E. Award program or donate to provide direct support to someone living with FOP.
Connect with FOP researchers, meet new families and more!
You’ll notice a few things are brand new for this year’s In Pursuit of a Cure campaign. But the most important part of the campaign is the same — you! Read on for a quick update on some exciting changes and what they mean to you in the weeks ahead…
We’ve supercharged In Pursuit of a Cure 2023 with some updates that make it easier for you to:
- Support FOP research
- Share videos, social posts and good news
- Ask questions and get information direct from FOP researchers
Changes for 2023 include:
- A signature campaign video available to watch right NOW instead of a one-hour video to watch at the end of the campaign
- A LIVE webinar on August 30 (3 viewing times offered) with leading FOP researchers where you can ask questions and interact in real-time. Live translation in Spanish and AI (artificial intelligence) translation available in 27 languages. Register at ifopa.org/curefop_webinar
- Brand new videos from members of the worldwide FOP community created for you to share with your family and friends
We still have the match to double the first $25,000 we receive in donations!
There’s no reason to wait if you plan to make a gift to help advance the next phase of transformational FOP research. The campaign is full speed ahead right now!
Please join us in the relentless pursuit of a cure today to:
We’re on this path together for as long as it takes. Together, we are Relentless!
MONTREAL, Feb. 11, 2019 (GLOBE NEWSWIRE) — Clementia Pharmaceuticals Inc. (Nasdaq: CMTA), a clinical-stage biopharmaceutical company innovating treatments for people with ultra-rare bone disorders and other diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to palovarotene for the treatment of fibrodysplasia ossificans progressiva (FOP). Palovarotene, an investigational therapy for FOP, has previously been granted Orphan Drug, Fast Track and Breakthrough designations by the FDA for FOP.
“FOP is a rare, devastating disease that begins to irreversibly impact individuals in childhood,” said Clarissa Desjardins, Ph.D., founder and chief executive officer of Clementia. “The receipt of this designation highlights the urgent need for a treatment for people with FOP. The entire Clementia team is working towards an NDA submission in the second half of 2019 in hopes of achieving our first regulatory approval, an important step toward achieving our mission of bringing innovative and effective treatments to individuals who currently have none.”
In October 2018, based on a meeting with the FDA, Clementia announced that the Agency was supportive of the company’s plan to submit a New Drug Application (NDA) for palovarotene for the prevention of heterotopic ossification (HO) associated with flare up symptoms in patients with FOP. The FDA based its assessment on the efficacy and safety data generated in the completed Phase 2 clinical program. The company’s NDA preparations are underway, with a submission targeted for the second half of 2019. If approved, Clementia expects a first commercial launch for palovarotene in 2020.
The Rare Pediatric Disease Designation qualifies investigational palovarotene for a Rare Pediatric Disease Priority Review Voucher (PRV), granted at the time of NDA approval. The PRV Program is intended to encourage development of therapies to prevent and treat rare pediatric diseases. This PRV can be redeemed to receive a priority review of a subsequent marketing application or sold to a third party.
Source : Clementia Pharmaceuticals Inc.
NDA Submission Planned for the Second Half of 2019
MONTREAL, Oct. 23, 2018 (GLOBE NEWSWIRE) — Clementia Pharmaceuticals Inc. (Nasdaq: CMTA), a clinical-stage biopharmaceutical company innovating treatments for people with ultra-rare bone disorders and other diseases, today announced that it plans to submit a New Drug Application (NDA) for palovarotene to the U.S. Food and Drug.
Administration (FDA) in the second half of 2019. Based on recent interactions between Clementia and the FDA, including a Type B meeting held earlier today as part of palovarotene’s Breakthrough Therapy Designation, the FDA has agreed that available data would support filing of an NDA for palovarotene for the prevention of heterotopic ossification (HO) associated with flare up symptoms in patients with fibrodysplasia ossificans progressiva (FOP). The FDA based its assessment on the efficacy and safety data available from the completed Phase 2 clinical program, which showed a statistically significant reduction in mean new HO volume, or bone volume, associated with flare-ups of FOP at 12 weeks as compared to placebo or untreated flare-ups.
The data provided to the FDA included the results from Clementia’s Phase 2 studies, which were recently presented at ASBMR 2018. In these studies, 92 palovarotene-treated flare-ups in 62 patients across three different dosing regimens were evaluated compared to 46 placebo or untreated flare-ups in 41 patients from the Company’s natural history study. Patients treated with palovarotene at the time of a flare-up demonstrated a greater than 70 percent reduction in mean new HO volume at 12 weeks compared to the untreated group. Further, the reduction in mean new HO volume among those treated with the episodic 20/10 mg dosing regimen (20 mg for 4 weeks starting at the time of a flare-up followed by 10 mg for 8 weeks) was statistically significant (p=0.02). Palovarotene was generally well tolerated across all dosing regimens of the Phase 2 clinical program. There were dose-related increases in retinoid-associated adverse events (AEs) with most being mild or moderate in severity, and only one patient discontinued participation in the study because of an AE.
Following the completion of standard non-clinical, clinical pharmacology and CMC studies to be agreed upon with the FDA, Clementia plans to submit its NDA in the second half of 2019 to seek approval of the palovarotene 20/10 mg episodic dosing regimen.
“The identification of a path to an NDA submission in the second half of 2019 is a significant milestone for Clementia and for patients with this ultra-rare and devastating genetic bone disease,” said Clarissa Desjardins, Ph.D., founder and chief executive officer of Clementia. “We are thankful for the collaboration with the FDA’s Division of Bone, Reproductive and Urologic Products, potentially bringing the first approved treatment option to individuals affected by FOP. We are also grateful to the patients and their families, as well as the investigators and clinical sites, without whom none of this work would have been possible.”
Clementia’s ongoing Phase 3 MOVE Trial will continue as planned, evaluating a dosing regimen of palovarotene which includes a chronic 5 mg daily dose in addition to the episodic 20/10 mg dosing regimen at the time of a flare-up. The Company believes that, if successful, the data from the MOVE Trial may provide the basis for a supplemental NDA for an additional treatment regimen option for patients with FOP.
Clementia also plans to engage international regulatory authorities in 2019 to discuss the registration strategy for palovarotene and anticipates providing an update at a later stage.
Source : Clementia Pharmaceuticals Inc.