Achondroplasia
therapeutics currently exhibits a considerably strong pipeline with seven
active drug candidates.
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.
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Detailed Report Summary:
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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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
By Phase
By Molecule
Type
By Route of
Administration
By Company
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