The leading analysts and researchers at Reports and Insights compiled and outlined the assessment of the influence of recommendations on market operations and exercises. The research comprises data driven by the market’s historical and present circumstances, as well as other elements impacting the market’s development process. It comprises the leading-age and up-to-date information on the global Achondroplasia Treatment Market outlook. The evaluation goes into information about revenue forecasts, volume, demand, growth, share, kinds, applications, and sales, among other things. The research analyzes a range of critical restrictions, comprising manufacturing capacity, item pricing, profit and loss figures, and distribution approaches.
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Achondroplasia Treatment Market Dynamics
The rising prevalence of achondroplastic dwarfism with no cure or prevention remedies is expected to drive the growth of the achondroplasia treatment market around the globe. For instance, according to the National Organization of Rare Disorders, 2018, achondroplasia is the most commonly occurring abnormality of bone growth, occurring in approximately 1 in 20,000-30,000 live births. Further, a short-term treatment of the growth hormone is an effective way to treat achondroplasia which is beneficial for the growth of the bones.
Further, leg-lengthening surgeries are also done in special cases to prevent loss of function. Moreover, there is a large number of symptoms that are caused due to achondroplasia which can be treated by using drugs and hence increasing the global achondroplasia treatment market.
Furthermore, increasing government funding, for such abnormalities also drives the revenue growth of the global achondroplasia treatment market in the coming years. The rising development of drugs combined with the growing awareness of this abnormality in developing countries is expected to create lucrative growth opportunities for the growth of the target market globally in the coming years.
Additionally, short-duration effectiveness of available therapy creates an opportunity for the key players for the development of new treatment therapies. However, a lack of awareness regarding achondroplastic dwarfism is expected to act as a barrier in the growth of the global achondroplasia treatment market.
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Achondroplasia Treatment Market Segmentation
The Achondroplasia Treatment market is segmented on the basis of
By Product Type:
By Drugs
RBM-007
TA-46
B-701
Others
By Therapy
Growth Hormone Therapy
Surgery
Supportive Therapy
By End User:
Hospitals
Clinics
Others
By Region:
North America
Europe
Asia Pacific
Latin America
Middle East
Africa
Achondroplasia Treatment Market Pipeline Drugs and Therapies Overview
Investigation of a C-type natriuretic peptide (CNP) analog is one of the most advanced therapeutic options for the treatment of achondroplasia, which acts a counterbalance to the effect of FGFR3. It was assumed that increasing CNP activity could respond to the excess negative signal of FGFR3 mutations which appeared to be true. An analog was developed by BioMarin Pharmaceuticals with greater stability, and animal experiments have been completed using this peptide, called BMN-111 or Vosoritide. Human trials for this drug have begun and are in phase 2 trials in young children and active recruitment for phase 3 trials.
Furthermore, Meclizine, a drug that improves the growth of the long bone in mouse through inhibition of the downstream effectors of FGFR3 have been sent for the phase 1 clinical trial. Other suggested therapies include the use of mesenchymal stem cells for the source of native CNP. Also, stain treatment of the rescued stem cells would help to treat achondroplasia.
Moreover, some suggested experimental protocols for the treatment of achondroplasia is inhibition of chaperone proteins of FGFR3, which help in the degradation of FGFR3 and, in turn, decrease its signaling. Use of decoy, soluble FGFR3 which is used to decrease binding of FGFs and consequently decrease the signaling of the protein. This therapy has entered early clinical trials. Other experimental protocols include direct blocking of the FGFR3 binding domain and reducing the activity of FGFR3 using a tyrosine kinase inhibitor.
Achondroplasia Treatment Market Competitive Landscape
Some of the key participating players in the achondroplasia treatment market are
Ascendis Pharma A/S
BioMarin Pharmaceutical Inc.
Ribomic Inc.
BridgeBio Pharma Inc.
QED Therapeutics
BioMarin Pharmaceutical Inc.
Pfizer Inc.
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