Osteogenesis imperfecta is an inherited connective tissue disorder characterized by bone fragility and recurrent fractures, most commonly due to pathogenic variants affecting type I collagen.
Medivir's 2025 Annual Report highlights FDA Orphan Drug Designation for MIV-711 in Osteogenesis Imperfecta and promising fostrox liver cancer data showing 13.7-month median survival. The company raised SEK 196 million to fund Phase 2 studies for both programs and reported year-end cash of SEK 119.2 million.
Class action lawsuits allege false statements about clinical trial results and regulatory prospects at Atara Biotherapeutics, Ultragenyx, Mereo BioPharma, and Vistagen Therapeutics. The complaints cite failed Phase 3 studies for setrusumab in Osteogenesis Imperfecta and fasedienol for social anxiety disorder, along with manufacturing issues affecting Atara's tabelecleucel application. Investors have deadlines ranging from April to May 2026 to seek lead plaintiff status.
Ultragenyx will lay off 130 employees as part of restructuring aimed at profitability by 2027. The FDA rejected the company's gene therapy application for Sanfilippo syndrome due to manufacturing concerns.
Ultragenyx Pharmaceutical faces federal securities class action lawsuits following the failure of Phase 3 Orbit and Cosmic studies for setrusumab in Osteogenesis Imperfecta, with stock falling over 42% after December 2025 results announcement.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07412782 |
REMS25: Study on the Use of REMS Technology in Diseases Commonly Associated With Reduced Bone Mineral Density (BMD) |
RECRUITING | NA |
| NCT07366086 |
Pediatric Safety Follow-up Study of Prior Treatment With Romosozumab for Osteogenesis Imperfecta |
RECRUITING | PHASE3 |
| NCT07287241 |
Prospective Observational Cohort Study of Cardiac Structure and Function in Children and Adults With Osteogenesis Imperfecta |
RECRUITING | |
| NCT07173010 |
Pediatric Arthropathy Beyond Inflammation: Clinical Spectrum and Diagnostic Approach at Assiut University Children Hospital |
NOT_YET_RECRUITING | |
| NCT06636071 |
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT06555536 |
e-Health Care Method for Patients With Osteogenesis Imperfecta (Tele OI) |
COMPLETED | NA |
| NCT06397443 |
Ready to Sail: Evaluating Sailing's Feasibility as Ergotherapy |
COMPLETED | NA |
| NCT06193642 |
Increasing Knowledge on Osteogenesis Imperfecta by Collecting Epidemiological Data |
COMPLETED | NA |
| NCT06086613 |
A First-in-Human Study Evaluating AGA2115 in Adult Healthy Volunteers |
COMPLETED | PHASE1 |
| NCT06065111 |
Study of Osteogenesis Imperfecta Tendon |
UNKNOWN |
