Phenylketonuria Market to Garner Considerable Growth at a 16.30% CAGR During the Study Period [2018-2030], Evaluates DelveInsight
Only one therapy, CNSA-001 (PTC923), is anticipated to launch in the forecast period and targets the overall Phenylketonuria patient pool. Whereas therapies such as SYNB1618, and HMI-102, are only for adult Phenylketonuria patients.
LAS VEGAS, Sept. 29, 2021 /PRNewswire/ -- DelveInsight's "Phenylketonuria (PKU) Market" report provides a thorough comprehension of the Phenylketonuria historical and forecasted epidemiology and the Phenylketonuria market trends in the 7MM [the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan]. The PKU market report also proffers an analysis of the current Phenylketonuria treatment algorithm/practice, market drivers, market barriers, and unmet medical needs.
Some of the necessary takeaways from the Phenylketonuria Market Research Report
- Several key pharmaceutical companies, including Asubio-Pharma, BioMarin-Pharmaceutical, PTC Therapeutics, Homology Medicines, Synlogic, and others, are developing novel products to improve the PKU treatment outlook.
- The FDA approved Palynziq (pegvaliase-pqpz) for Phenylketonuria adults in 2018. Palynziq is an injectable enzyme therapy for PKU patients and is manufactured by BioMarin Pharmaceutical.
- The Phenylketonuria market share of Kuvan is expected to decrease in the forecast period after losing its market exclusivity and entry of generics in the PKU market.
- The Phenylketonuria market will grow as there has been an increase in awareness of the disease with the increasing prevalence. Recently, research and development strategies are being made to produce novel products.
- Nevertheless, the Phenylketonuria market will be impeded by the unclear comorbidities associated with the disease and the limitations related to the current treatment.
- At present, only two therapies are approved for PKU treatment, and the rest of the management depends on neutral amino acid supplementation and enzyme replacement therapy. With the increase in diagnosis rate and awareness of the disease, the prevalent cases of PKU are expected to increase, which will increase the PKU market size. The pipeline possesses potential drugs as monotherapies and gene therapies, due to which the PKU therapeutics market is expected to grow in the forecast period (2021–2030).
- It is expected that PTC-923 will be competing with all the upcoming therapies and already approved therapies in the future. Large pool capture, early market entry, and low safety issues will help PTC-923 capture a significant PKU market share. But compared to HMI-102, the market size of PTC-923 will be less because HMI-102 is gene therapy and will be launched at a much higher price than PTC-923.
For further information on Market Impact by Therapies, visit: Phenylketonuria Drugs Market Analysis
Phenylketonuria (PKU) is an inborn error of metabolism and is characterized by the absence or deficiency of an enzyme called phenylalanine hydroxylase (PAH), responsible for processing the amino acid phenylalanine.
DelveInsight estimates that the total diagnosed Phenylketonuria prevalent cases in the 7MM was 49,887 cases in 2020.
The Phenylketonuria Market Analysis Report provides historical as well as forecasted epidemiological analysis segmented into:
- Diagnosed Prevalent Cases of Phenylketonuria
- Age-specific Diagnosed Prevalent Cases of Phenylketonuria
- Severity-specific Diagnosed Prevalent Cases of Phenylketonuria
- Diagnosed Prevalent Cases of Phenylketonuria by Mutation type
Get a complete epidemiological segmentation breakdown @ Phenylketonuria Epidemiological Analysis
Phenylketonuria Treatment Market
Kuvan was approved by the US FDA and is used to lower blood Phe levels in adults and children over one month of age. However, it was observed that not all PKU patients responded to Kuvan treatment. The drug also causes serious issues and serious side effects such as severe allergic reactions, including anaphylaxis, hyperactivity, and many more. So, there was a requirement for a better PKU treatment option.
Phenylketonuria treatment is quite challenging, and the patient often faces a decrease in adherence to diet and the presence of neurocognitive deficits despite therapy. To overcome these barriers, Palynziq was approved by the US FDA in 2018. This novel enzyme therapy was approved for Phenylketonuria adults patients who have uncontrolled blood Phe concentrations on current treatment and may help maintain lower blood Phe levels with fewer diet restrictions.
Phenylketonuria Emerging Drugs
As Phenylketonuria cases are increasing day by day, several companies are focusing on the therapeutic pipeline. In the upcoming years, many new products such as CNSA-001/PTC923 (PTC Therapeutics), HMI-102 (Homology Medicines), and SYNB1618 (Synlogic) are also expected to enter the PKU market.
Among all the upcoming therapies, PTC-923 is expected to be the most promising drug. It is expected that PTC-923 will be competing with all the forthcoming therapies and already approved therapies in the future. A large pool, early market entry, and low safety issues will help PTC-923 capture a significant Phenylketonuria market share. But compared to HMI-102, the market size of PTC-923 will be less because HMI-102 is gene therapy and will be launched at a much higher price than PTC-923. The available therapeutics treatment options in Phenylketonuria Landscape aim to reduce the disease burden.
Phenylketonuria Market Dynamics
Significant growth will be observed in the Phenylketonuria market; there has been an increasing awareness about the disease among clinicians and patients with the rising PKU prevalence among the growing population, including newborns and an adult that has surged demand for PKU treatment worldwide. Phenylketonuria can now be detected during the first few days of life by two reliable mass screening techniques and its significant consequence. Also, there is government support for ongoing clinical research that has allowed the US to dominate the PKU treatment landscape. In addition to that, Research and Development also provide an outlook on emerging approaches for hyperphenylalaninemia treatment, such as recruiting the microbiome into the therapeutic endeavor and therapies under development such as gene therapy. The market players are leveraging the proliferating demand for other drugs through the different administration modes and focusing on launching novel products.
Nevertheless, PKU treatment options primarily remain limited to dietary restrictions, which leads to many untreated PKU patients experiencing intellectual disability, seizures, and mental disorders. Potential dietary therapy issues have been associated with nutritional deficiencies, especially vitamin D and B12. In early-treated patients with PKU, it is unclear if comorbidities such as overweight, obesity, hypertension, and diabetes are higher than those in the general population and associated with increased cardiovascular risk. Moreover, PKU has significant socioeconomic repercussions which will hinder the Phenylketonuria market growth.
Scope of the Phenylketonuria Market Insight Report
- Geography Covered: The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
- Study Period: 3-year historical and 10-year forecasted analysis (2018-2030).
- Phenylketonuria Markets Segmentation: By Geographies and By PKU Therapies (Historical and Forecasted, Current and Upcoming)
- Dominant Market Companies investigating its candidates for Phenylketonuria: Asubio-Pharma, BioMarin-Pharmaceutical, PTC Therapeutics, Homology Medicines, Synlogic, and several others.
- Analysis: Comparative and conjoint analysis of emerging therapies.
- Case Studies
- KOL's Views
- Analyst's View
Request for a Webex demo of the report @ Phenylketonuria Therapeutics Market
Table of Contents
1 |
Phenylketonuria (PKU) Key Insights |
2 |
Phenylketonuria Report Introduction |
3 |
Phenylketonuria Market Overview at a Glance |
4 |
Executive Summary of Phenylketonuria |
5 |
Phenylketonuria Treatment |
6 |
Phenylketonuria Epidemiology and Patient Population |
7 |
Country Wise-Epidemiology of Phenylketonuria |
7.1 |
The United States |
7.2 |
EU5 Countries |
7.2.1 |
Germany |
7.2.2 |
France |
7.2.3 |
Italy |
7.2.4 |
Spain |
7.2.5 |
The United Kingdom |
7.3 |
Japan |
8 |
Organizations contributing towards Phenylketonuria |
9 |
Phenylketonuria Case Reports |
10 |
Phenylketonuria Patient Journey |
11 |
Phenylketonuria Marketed Therapies |
11.1 |
Kuvan (Sapropterin Hydrochloride): Asubio-Pharma/BioMarin-Pharmaceutical |
11.2 |
Palynziq (pegvaliase-pqpz/rAvPAL-PEG/BMN 165): BioMarin Pharmaceutical |
12 |
Phenylketonuria Emerging Therapies |
12.1 |
CNSA-001 (PTC923, Sepiapterin): PTC Therapeutics |
12.2 |
HMI-102: Homology Medicines |
12.3 |
SYNB1618: Synlogic |
12.4 |
BMN 307: BioMarin Pharmaceuticals |
13 |
Phenylketonuria 7MM Market Analysis |
13.1 |
The United States PKU Market Size |
13.2 |
EU-5 Phenylketonuria Market Size |
13.2.1 |
Germany Market Size |
13.2.2 |
France Market Size |
13.2.3 |
Italy Market Size |
13.2.4 |
Spain Market Size |
13.2.5 |
The United Kingdom Market Size |
13.2.3 |
Japan PKU Market Size |
14 |
Phenylketonuria Market Drivers |
15 |
Phenylketonuria Market Barriers |
16 |
Phenylketonuria SWOT Analysis |
17 |
Phenylketonuria Unmet Needs |
18 |
Phenylketonuria KOL Views |
19 |
Appendix |
20 |
DelveInsight Capabilities |
21 |
Disclaimer |
22 |
About DelveInsight |
Browse full report with detailed TOC with charts, figures, tables @ Phenylketonuria Diagnostics Market Report
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SOURCE DelveInsight Business Research, LLP
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