Global Osteogenesis Imperfecta Treatment Market- Key Developments
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development and commercialization of novel products, announced that the first patients have been dosed in both of its late-stage clinical trials evaluating setrusumab in pediatric and young adult patients with OI sub-types I, III, and IV. The Phase 3 portion of the pivotal Phase 2/3 Orbit study is evaluating the effect of setrusumab compared to placebo on annualized clinical fracture rate in patients aged 5 to <26 years. The newly initiated Phase 3 Cosmic study is an active-controlled study evaluating setrusumab compared to intravenous bisphosphonate (IV-BP) therapy on annualized total fracture rate in patients aged 2 to <5 years.
In October 2022, Osteogenesis Imperfecta Federation Europe (OIFE) announced the re-establishing Osteogenesis Imperfecta Federation Europe (OIFE) in Belgium. OIFE is re-established as a new legal entity in Belgium and dissolved as a legal entity in the Netherlands.
In October 2021, Mereo BioPharma Group plc, a clinical stage biopharmaceutical company, focused on oncology and rare diseases, the Osteogenesis Imperfecta Federation Europe (OIFE), an association for organizations dealing with the rare disease Osteogenesis Imperfecta (OI) and the Osteogenesis Imperfecta Foundation (OIF), a voluntary national health organization helping people cope with the problems associated with osteogenesis imperfect, announced completion of enrollment in the largest global gathering of data-to-date about the impact that OI has on people with OI, their families, and caregivers. The IMPACT Survey closed with more than 2200 individual responses collected over a 3-month period from some 65 countries. The survey results will be used to support future collaborative work on better diagnosis, treatment, and care and to support the timely evaluation and availability of potential new treatments for OI.
In December 2022, Sanofi, an innovative global healthcare company, initiated a Phase I study to evaluate the safety, tolerability, and activity of a single dose of SAR439459 in adult participants with Osteogenesis imperfecta. Orphan Drug Designation was granted by the FDA for this indication
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