Achondroplasia is the most common form of disproportionate short stature or dwarfism. It is caused by a mutation in the FGFR3 gene that inhibits bone growth, resulting in unusually short limbs and torso. Current treatment options include hormone therapy, limb-lengthening procedures, spinal decompression surgery, and genetic counseling. Researchers are investigating drugs that can block FGFR3 signaling to stimulate bone growth in patients.
The achondroplasia treatment market is driven by the rising prevalence of the condition and advancements in treatment options. According to the CDC, Achondroplasia affects about 1 in every 25,000 newborns.Growing research into developing drugs targeting the underlying FGFR3 mutation driving abnormal bone growth is expected to offer hope for effective medical therapies. Additionally, pipeline drugs from companies such as BioMarin and Ascendis Pharma that aim to improve growth and height in patients could positively impact market growth over the forecast period if approved.
The global achondroplasia treatment market is segmented into two types – recombinant human growth hormone therapy and rhGH therapy. The rhGH therapy segment currently dominates the market due to growing number of clinical trials and pipeline drugs in this segment. This therapy involves administration of recombinant growth hormone injections that promote linear growth in children with achondroplasia.
Political: Government regulations around clinical trials and approval processes for novel drugs impact the development of new treatment options for achondroplasia.
Economic: High treatment cost of various therapies poses a challenge for widespread adoption. However, increasing healthcare expenditure is expected to drive market growth.
Social: Rising clinical awareness programs and patient support groups are encouraging the development of improved treatment alternatives.
Technological: Advancements in genetic engineering and Gene therapy are likely to bring more pipeline candidates and novel treatment mechanisms in the forecast period.
The global achondroplasia treatment market is expected to witness high growth, exhibiting CAGR of 36.5% over the forecast period 2023-2030 due to increasing clinical research and strong pipeline of drugs. The market is estimated to reach US$ 127.8 million in 2023.
Regional analysis: North America dominates the global market currently due to presence of leading pharmaceutical companies and favorable regulatory environment for clinical trials. However, Asia Pacific is expected to be the fastest growing market owing to rising healthcare expenditure and growing clinical research activities in countries like China and India.
Key players: Key players operating in the achondroplasia treatment market are BioMarin, RIBOMIC, Ascendis Pharma A/S, BridgeBio Pharma, Inc., Pfizer Inc., PhaseBio Pharmaceuticals, Inc., SiSaf, Novo Nordisk A/S, F. Hoffmann-La Roche Ltd, LG Chem, Ferring B.V., JCR Pharmaceuticals Co., Ltd, KVK TECH, INC., VIVUS LLC., ProLynx Inc., Teva Pharmaceutical Industries Ltd., Eli Lilly and Company, Ipsen Pharma, Novartis AG, and Xiamen Amoytop Biotech Co., Ltd.