Phenylketonuria (PKU) is a rare genetic disorder caused due to increased level of phenylalanine in the blood. The excess amount of phenylalanine can cause intellectual disability and other serious health problems. The most severe condition of this disorder is termed as classic PKU and symptoms related to it are seizures, tremors, or trembling and shaking, stunted growth, hyperactivity, skin conditions such as eczema, and musty odor of their breath, skin, or urine. PKU can be managed by maintaining phenylalanine free diet. KUVAN (sapropterin dihydrochloride) is a U.S. Food and Drug Administration approved drug which is majorly used in the treatment of Phenylketonuria.
Phenylketonuria Treatment Market Drivers
Frequent research for development of novel drugs for the treatment of phenylketonuria is expected to drive growth of global phenylketonuria treatment market in the near future. For instance, in 2017, BioMarin Pharmaceutical Inc. submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its new Pegvaliase, indicated for the treatment of patients with Phenylketonuria (PKU). In 2018, European Medicines Agency (EMA) accepted BioMarin’s submission of a Marketing Authorization Application (MAA) for pegvaliase for the treatment of adults with phenylketonuria (PKU) who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l). In 2017, Synlogic, Inc. received Orphan Drug Designation for its SYNB1618 from U.S. Food and Drug Administration. SYNB1618, an orally administered synthetic biotic medicine indicated for the treatment of PKU. Furthermore, increasing acquisitions and collaborations between key players is also expected to create a lucrative environment for growth of global phenylketonuria treatment market. For instance, in 2015, BioMarin Pharmaceutical Inc. acquired all global rights of Kuvan (sapropterin dihydrochloride) and pegvaliase from Merck Serono (Merck). Kuvan is an oral therapy indicated for the treatment of hyperphenylalaninemia (HPA) due to phenylketonuria (PKU). Pegvaliase (PEGylated recombinant phenylalanine ammonia lyase) is an investigational enzyme substitution therapy for the treatment of phenylketonuria (PKU). In 2016, Dimension Therapeutics, Inc. entered into a gene therapy research collaboration with the University of Pennsylvania for research and development of novel treatment for phenylketonuria.
Phenylketonuria Treatment Market Regional Analysis
North America is expected to hold a dominant position in global phenylketonuria treatment market, owing to initiatives taken by governments for the diagnosis and treatment of phenylketonuria in the region. For instance, in 2017, The National PKU Alliance and Baby Genes launched a program to allow easy access to genetic testing for people with phenylketonuria, and their families. The program aims to help in diagnosis and assist the people for treatment of phenylketonuria. Furthermore, launches of novel products by U.S.-based key players is also expected to drive growth of North America phenylketonuria treatment market. For instance, in 2017, Vitaflo USA, LLC launched its new PKU Sphere, a low phenylalanine medical food used in dietary management of Phenylketonuria (PKU). Europe is also expected to exhibit significant growth in the near future, owing to increasing approval of new drugs in the region. For instance in 2015, Merck Group, a Germany-based company, received EU-approval to extend the use of Kuvan in children below four years of age suffering from Phenylketonuria.
Phenylketonuria Treatment Market Restraint
According to data published by The ZB Foundation in 2017, Phenylketonuria is a rare inherited disease that affects only about 1 in 10,000 to 15,000 newborn children. The lack of awareness about the rare disease is a major factor, which is expected to restrain growth of the market in the near future.
Phenylketonuria Treatment Market Key Players
Key players operating in global phenylketonuria treatment market include BioMarin Pharmaceutical Inc., Dimension Therapeutics, Inc., American Gene Technologies International Inc., Synthetic Biologics, Inc., Codexis, Inc., SOM Innovation Biotech SL, Daiichi Sankyo Company, Limited, and Erytech Pharma SA. Key Players in global phenylketonuria treatment market are focusing on strategic collaborations for the development of novel therapies for the treatment of phenylketonuria. For instance, in January 2018, Retrophin, Inc. and Censa Pharmaceuticals collaborated for the development and evaluation of CNSA-001 for the treatment of phenylketonuria (PKU). CNSA-001, is an orally bioavailable proprietary form of sepiapterin, serves as a natural precursor of tetrahydrobiopterin (BH4) that is converted by an endogenous enzymatic pathway to BH4.
Phenylketonuria Treatment Market Taxonomy:
The global Phenylketonuria treatment market is segmented on the basis of treatment type, route of administration, distribution channel, and region:
By Treatment Type-
By Route of Administration-
By Distribution Channel-
Coherent Market Insights followsa comprehensive research methodology focused on providing the most precise market analysis. The company leverages a data triangulation model which helps company to gauge the market dynamics and provide accurate estimates. Key components of the research methodologies followed for all our market reports include:
In addition to this, Coherent Market Insights has access to a wide range of the regional and global reputed paid data bases, which helps the company to figure out the regional and global market trends and dynamics. The company analyses the industry from the 360 Degree Perspective i.e. from the Supply Side and Demand Side which enables us to provide granular details of the entire ecosystem for each study. Finally, a Top-Down approach and Bottom-Up approach is followed to arrive at ultimate research findings.
Coherent Market Insights desk research is based on a principle set of research techniques:
Coherent Market Insights has a large amount of in-house repository of industry database. This is leveraged as a burner for initiating a new research study. Key secondary sources include:
Preliminary Data Mining
The raw data is obtained through the secondary findings, in house repositories, and trade surveys. It is then filtered to ensure that the relevant information including industry dynamics, trends, and outlook is retained for further research process.
Holistic approach is used to ensure that the granular and uncommon parameters are taken into consideration to ensure accurate results. The information from the paid databases are further combined to the raw data in order to standardize it.
Coherent Statistical model
We arrive at our final research findings through simulation models. Coherent Data Analytics Model is a statistical tool that helps company to forecast market estimates. Few of the parameters considered as a part of the statistical model include:
Once the findings are derived from the statistical model, large volume of data is process to confirm accurate research results. Data analytics and processing tools are adopted to process large chunk of collected informative data. In case, a client customizes the study during the process, the research finding till then are benchmarked, and the process for new research requirement is initiated again.
This is the most crucial stage of the research process. Primary Interviews are conducted to validate the data and analysis. This helps in achieving the following purposes:
The primary research is conducted with the ecosystem players including, but not limited to:
Finding it difficult to find the research that would cater to your business demands? Give us a chance to help you. One of our Research Consultants will connect to provide a customized solution.[email protected]
Talk to our research consultant to design an exclusive report as per your research needs.
We aim to fulfil client's research demands with tailored research solutions.
We aim to provide research studies in quickest turnaround time and in a much cost effective manner.
We cover each industry from supply and demand side with an aim to provide a most holistic research study.
We strive to provide most accurate and reliable research findings in our research reports.