Achondroplasia is a common disorder of dwarfism with disproportionate stature. It is an autosomal dominant disorder that occurs due to mutation in fibroblast growth factor receptor 3 (FGFR3) gene, which is located on short arm of chromosome 4. The disorder is characterized by macrocephaly, shortened-limbs (rhizomelic dwarfism), midface retrusion, and frontal bossing. The achondroplasia therapeutics pipeline study covers seven active drug candidates in different stages of development.
Achondroplasia therapeutics pipeline has majority of candidates in the Pre-Clinical stage of development. A peptide-based drug candidate namely, vosoritide, is in the Phase III stage of development by BioMarin Pharmaceutical Inc., for the treatment of achondroplasia.
The drug candidates developed by several companies for the treatment of achondroplasia have shown positive clinical results in the various phases of drug development. For instance, BioMarin Pharmaceutical Inc.’s drug candidate vosoritide, which is under the Phase III stage of development, has been well-tolerated at all doses in the Phase II stage’s study. The majority of adverse events (AEs) were mild and no serious AEs were reported during the study. Across all doses, injection site reactions and hypotension were the most common drug-related AEs.
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Some of the key players involved in the development of achondroplasia therapeutics include BioMarin Pharmaceutical Inc., Ascendis Pharma A/S, and Therachon AG.
Achondroplasia Therapeutics Pipeline Analysis
- By Phase
- By Molecule Type
- By Route of Administration
- By Company
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