FDA grants orphan drug designation for potential scleroderma treatment
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Key takeaways:
- BLR-200 has yielded “promising” results in ongoing research.
- The therapy targets multiple drivers of scleroderma and treats affected organs.
The FDA has granted orphan drug designation to BLR-200, an investigational therapy for the treatment of scleroderma, according to a press release from manufacturer.
The drug, a CCN3-derived peptide, is being developed by BLR Bio as part of the Helix 51 Bioscience Incubator at Rosalind Franklin University of Science and Medicine, in North Chicago, Illinois.
“BLR-200 has the potential to establish first-in-class clinical benefits by addressing multiple elements driving scleroderma and treating multiple organs negatively impacted by the disease,” Ronald S. Kaplan, PhD, executive vice president for research at the university, said in the release.
BLR-200 has yielded “promising in vivo results” in ongoing research funded by a $780,000 grant from the Canadian Institutes of Health and Research. The research is aimed at revealing scleroderma’s mechanisms and pathways, as well as the therapy’s ability to influence them, according to a previous press release.
“Our therapy addresses multiple disease-supporting elements or redundancies in the pathways to initiation and progression, and targets a key scar-forming cell population of myofibroblasts,” BLR Bio CEO Bruce Riser, PhD, said in the release.
Orphan drug designation allows for development assistance, tax credits, FDA fee exemptions and 7 years of post-approval marketing exclusivity for therapies targeting rare diseases.
References:
Rosalind Franklin University company BLR Bio awarded $780,000 grant from the Canadian Institutes of Health Research. https://www.prnewswire.com/news-releases/rosalind-franklin-university-company-blr-bio-awarded-780-000-grant-from-the-canadian-institutes-of-health-research-301745853.html. Published Feb. 14, 2023. Accessed April 15, 2024.
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