fibrodysplasia ossificans progressiva
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Search complete. 7 mentions across 5 episodes found for "fibrodysplasia ossificans progressiva".
Oct 2, 2026
Graig SuvannavejhHOST
I'm gonna turn it over to Brian again to discuss some new data from Dyne Therapeutics from the 31st Annual World Muscle Society Conference taking place in Hiroshima, Japan.
Graig SuvannavejhHOST
And then additional news this week from, um, Mirum, uh, which we just spoke about, which came a couple days after news of their FOP drug approval.
Graig SuvannavejhHOST
So Brian?
Brian SkorneyHOST
Yeah.
speaker_1HOST
This breakthrough underscores ongoing innovations in neuropharmacology aimed at enhancing symptom management and improving quality of life for patients with this debilitating condition.
speaker_1HOST
In parallel, Merum Pharmaceuticals has also received FDA approval for Atabreaz, a small molecule targeting ALK2, specifically designed for Fibrodysplasia acificans progressiva, FOP, a rare musculoskeletal disorder.
speaker_1HOST
The Phase 2 data supporting Atabreaz's approval provides hope by inhibiting the aberrant bone growth associated with FOP.
speaker_1HOST
This development not only expands therapeutic options for rare diseases but also emphasizes the significance of targeted therapies in addressing specific genetic and molecular pathways implicated in disease pathology.
speaker_1HOST
The business landscape is reflecting a thriving scene with significant licensing deals and partnerships.
Eugene LeeGUEST
Regulators, payers, healthcare systems, and others also need to think differently about their approach when it comes to rare disease.
Madeline SpencerHOST
There are several important FDA decisions occurring as we record this, including decisions for orphan drugs to treat FOP, MCT8 deficiency, as well as SCN2A and SCNA8 deficiencies.
Madeline SpencerHOST
We will have a full report on those decisions next week.
James RadkeHOST
CheckRare is also attending and covering the AANEM conference focused on neuromuscular disorders this week, so we'll have plenty to talk about in our next podcast.
Alex MercerHOST
The FDA approved Mirum's Atebrioz, Xelurgizer Tib, and oral ALK2 inhibitor Mirum Licensed exclusively from Insight.
Alex MercerHOST
The approval is for patients 12 and older with fibrodysplasia ossificans progressiva, or FOP.
Alex MercerHOST
In this disease, bone forms in soft tissue.
Maya PatelHOST
and it came despite a miss.
speaker_0HOST
Retinitis pigmentosa gene therapy has had a rough track record, so a clean, positive, pivotal result here is a real proof point, and Beacon says it plans to start filing with regulators by the end of the year.
speaker_0HOST
On the regulatory side, Merum Pharmaceuticals won FDA approval for an oral drug called Atebriaz for fibrodysplasia ossificans progressiva, or FOP, a devastating ultra-rare disease where soft tissue gradually turns into bone.
speaker_0HOST
This is actually the second FOP drug approved in just about a month after Regeneron's Passatru got the green light in August.
speaker_0HOST
Both target the same biological pathway but work a little differently, and Mirim's pitch is that its once-daily pill and broader age range can compete even though Regeneron got there first.
speaker_0HOST
It's a nice example of the FDA being willing to approve a strong second-in-class drug quickly once a new mechanism is proven out, which is encouraging for any company worried about not being first to market in a rare disease space.