Achondroplasia therapeutics currently exhibits a considerably strong pipeline with seven active drug candidates.
Overview of Achondroplasia Therapeutics Pipeline in 2018
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.
Insights on Pipeline Segments
According to the research findings, majority of the drug candidates for the treatment of achondroplasia are being developed to be administered by the subcutaneous route. Some of the key advantages of subcutaneous route are sustained release of drug, improved patient condition, lower risk of systemic infections, less painful administration, and less-expensive nature of the administered drugs.
Achondroplasia therapeutics Pipeline is characterized by Positive Clinical Trial Results
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.
Technological Advancements is a key factor marking the Growth of Achondroplasia Therapeutics Pipeline
It has been observed that many pharmaceutical companies are adopting advanced technologies for the development of achondroplasia therapeutics. These technologies are helpful in the development of drugs as either combination therapies or single-agent therapies. For instance, RIBOMIC Inc.’s, RiboART system, is the essential element in drug discovery technology, that helps in the discovery of various new drugs for the treatment of the various diseases including achondroplasia.
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