Weekly Spotlight - 07.11.24

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In the News

Umedaptanib Pegol Shows Promise in Achondroplasia Growth Trial

RIBOMIC's Phase IIa trial of umedaptanib pegol shows promise for children with achondroplasia. The low-dose treatment increased growth rates by up to 4.6 cm/year in some patients, offering hope for new treatment options. No safety concerns were reported, and further studies are underway to optimise dosing.

Achondroplasia Market Growth Predicted to Rise Significantly

The achondroplasia market is set to grow significantly from 2024 to 2034, driven by increased prevalence and awareness. Key companies like Sanofi and BridgeBio are developing promising therapies. The FDA's Breakthrough Therapy Designation for infigratinib highlights potential advancements in treatment, offering hope for improved patient outcomes and support.