What is Propionic Acidemia?
Propionic Academia (PA) is a very rare genetic disease or disorder which basically caused by dis-functionality and deficiency of propionyl-CoA carboxylase. propionyl-CoA carboxylase is a type of enzyme within human body which is responsible for breaking down of amino acids of proteins. Propionic Acidemia is categorised as Metabolic Disorder, this also leads up to build up of toxic content within the tissues as organic acids increases within body. Propionic Acidemia is mostly found in New-born infants due to faulty genes passed over by parents. The symptoms of PA may be vomiting, weak muscle tone to more severe such as seizures, coma, and even the possibly of death. Although there are no particular drugs completely treating PA, but there does exist certain therapies and passive drugs which can limit the spread and severity of PA. It is highly found in Middle East, and Europe.
The market study is broken down by Type (Neonatal-Onset PA and Late-Onset PA), by Application (Hospitals & Clinics and Medicinal & Pharmaceutical Stores) and major geographies with country level break-up.
Established and emerging Players should take a closer view at their existing organizations and reinvent traditional business and operating models to adapt to the future.
Moderna Inc. (United States), Recordati S.p.A (Italy), CSL Behring (United States), HemoShear Therapeutics, Inc. (United States), BRIDGEBIO PHARMA, INC. (United States) and GeneDx, Inc. (United States) are some of the key players that are part of study coverage.
Segmentation Overview
AdvanceMarketAnalytics has segmented the market of Global Propionic Acidemia market by Type, Application and Region.
On the basis of geography, the market of Propionic Acidemia has been segmented into South America (Brazil, Argentina, Rest of South America), Asia Pacific (China, Japan, India, South Korea, Taiwan, Australia, Rest of Asia-Pacific), Europe (Germany, France, Italy, United Kingdom, Netherlands, Rest of Europe), MEA (Middle East, Africa), North America (United States, Canada, Mexico). If we see Market by Treatment Type, the sub-segment i.e. Carnitine Supplementation will boost the Propionic Acidemia market. Additionally, the rising demand from SMEs and various industry verticals gives enough cushion to market growth.
In 2019, Moderna, Inc., one of world’s largest pharmaceutical and Biotechnology Company received FDA clearance for fast track designation for its messenger therapeutic called mRNA-3927, to treat propionic acidemia (PA). The Mrna-3927 is aimed at helping body restore dysfunctional proteins and it includes two mRNA’s consisting of PCC and LNP to treat PA., and In 2021, United States FDA has approved Carbaglu as an adjunctive treatment therapy for acute hyperammonemia which is caused primarily by PA.
Biotin has not Been Approved by FDA for Medicinal Use. According to US FDA, “Metronidazole” is used to reduce the development of drug-resistant bacteria and maintain the effectiveness of metronidazole and other antibacterial drugs, metronidazole should be used only to treat infections that are proven to be caused by bacteria
Market Trend
- Intense Research and Development in Bio-Medicine Specific to Genetic Disorders
Market Drivers
- Rising Genetic Disorders
- Improvement in Medical Infrastructure
Opportunities
- Surge in Medical Insurance Coverages
- Augmenting Awareness and Government Funding's has Created Growth Opportunities for Market
Restraints
- High Cost of Treatment
- Less Awareness regarding Genetic Disorders in Emerging Countries of South America & Middle East
Challenges
- Suitability of Proper Treatment
Key Target Audience
Propionic Acidemia Treatment Providers, Venture Capitalists and Private Equity Firms, New Entrants/Investors, Analysts and Strategic Business Planners, Raw Material Suppliers, Government Regulatory and Research Organizations and End-Use Industries
About Approach
To evaluate and validate the market size various sources including primary and secondary analysis is utilized. AMA Research follows regulatory standards such as NAICS/SIC/ICB/TRCB, to have the better understanding of the market. The market study is conducted on basis of more than 200 companies dealing in the market regional as well as global areas with purpose to understand companys positioning regarding market value, volume and their market share for regional as well as global.
Further to bring relevance specific to any niche market we set and apply number of criteria like Geographic Footprints, Regional Segments of Revenue, Operational Centres, etc. The next step is to finalize a team (In-House + Data Agencies) who then starts collecting C & D level executives and profiles, Industry experts, Opinion leaders etc. and work towards appointment generation.
The primary research is performed by taking the interviews of executives of various companies dealing in the market as well as using the survey reports, research institute, and latest research reports. Meanwhile, analyst team keeps preparing set of questionnaires and after getting appointee list; the target audience are then tapped and segregated with various mediums and channels that are feasible for making connection that includes email communication, telephonic, skype, LinkedIn Group & InMail, Community Forums, Community Forums, open Survey, SurveyMonkey etc.