Phenylketonuria Market
Number of pages: 100 | Report Format: PDF | Published date: March 10, 2023
Historical Years – 2021 | Base Year – 2022 | Forecasted Years – 2023-2031
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Report Attribute |
Details |
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Market Size Value in 2022 |
US$ 592.9 million |
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Revenue Forecast in 2031 |
US$ 1,697.7 million |
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CAGR |
12.4% |
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Base Year for Estimation |
2022 |
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Forecast Period |
2023-2031 |
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Historical Year |
2021 |
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Segments Covered |
Treatment, Distribution Channel, and Region |
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Regional Scope |
North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa |
According to the deep-dive market assessment study by Growth Plus Reports, the global phenylketonuria market was valued at US$ 592.9 million in 2022 and is expected to register a revenue CAGR of 12.4% to reach US$ 1,697.7 million by 2031.
Phenylketonuria Market Fundamentals
Phenylketonuria (PKU) is an inherited metabolic disorder—a mutation in the gene responsible for producing phenylalanine hydroxylase results in PKU. Those affected have a higher level of phenylalanine in their blood. PKU can have mild to severe symptoms and effects. Infants with exceptional PKU initially appear normal until they are a few months old. Without therapy, these children develop lifelong intellectual impediments. In the first few weeks of life, newborn screening programs are used to diagnose the disease. All cases are then tested for tetrahydropterin (BH4) responsiveness. The foundation of newborn screening programs and the prompt implementation of dietary treatment have averted intellectual disability.
However, neurophysiological and neuropsychological impairments may still exist in PKU patients who have received treatment. Additionally, dietary treatment frequently comes with issues such as nutritional deficiencies and non-compliance brought on by poor palatability. Large neutral amino acids (LNAA) and glycomacropeptide (GMP) have been investigated as more cutting-edge medical foods and formulas with better palatability. However, long-term studies are still required to fully understand these treatments’ effects. In BH4-responsive patients, BH4 therapy has been effective; however, only about 30% of PKU patients can benefit from this therapy. Recently, enzyme substitution treatment, in which phenylalanine ammonia lyase is used as an alternative to phenylalanine hydroxylase, and enzyme replacement therapy using viral delivery of protein fusions targeting the liver have shown promising treatment outcomes for PKU. In addition, the use of tetrahydropterin (BH4) as a pharmaceutical treatment has been found to effectively lower blood phenylalanine levels in certain individuals with PKU. It is considered a viable alternative treatment strategy. Sapropterin hydrochloride functions as a molecular chaperon at therapeutic levels, assisting in the phenylalanine hydrolase enzyme’s proper folding and stability.
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Phenylketonuria Market Dynamics
The phenylketonuria market is expected to grow due to the expanding disease burden associated with the condition and the innovative therapies emerging to treat it. Every ethnic group has been affected by PKU, and its prevalence varies worldwide, affecting 1 in every 10,000 Caucasians, with the highest incidence being in Northern Europe. Finland has the lowest incidence in Europe, with 1 case per 100,000 live births. In contrast, Turkey has the highest, with 1 case per 4000 births, due to high consanguinity in the population. Each year, approximately 25 babies in Australia are diagnosed with PKU.
The market revenue growth has been supported by collaborations among players and the improvement in PKU pipeline drugs. The treatment of PKU is evolving at a rapid pace. Various novel treatments, frequently combined with other new or existing medications, are emerging from the research and development activities. Many pharmaceutical firms are funding research in new drug development for treatment. For instance, there are currently seven clinical studies for treating PKU, of which three are in phase III.
The development of the phenylketonuria market is stimulated by rising interest in intensified genomics research and growth in awareness of the best ways to treat the condition. Furthermore, a rise in efforts by government and non-government organizations to raise public awareness of the disease and the advantages of its treatment is anticipated to increase the rate of early diagnosis and, consequently, the treatment. In 2015, the Government of Prince Edward Island started the PKU supplement program based on the dietitian’s recommendation for special low-protein formulas. The program covered all costs and specialty food items from the National Food Distribution Centre. Expenses covered up to a maximum of US$ 3,600 annually.
However, despite the development trajectory, the high expense of medications and therapies for rare diseases may have a negative impact on the market outlook. Patients with PKU also need readily available, sufficient health insurance to maintain their health for extended periods. These higher prices, primarily due to delayed medication imports, hinder the development of the global phenylketonuria market.
Phenylketonuria Market Ecosystem
The global phenylketonuria market is analyzed from three perspectives: treatment, distribution channel, and region.
Phenylketonuria Market by Treatment
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Based on the treatment, the global phenylketonuria market is segmented into drugs and protein ingredients. Sapropterin (Kuvan) and pegvaliase (Palynziq) are the key drug types. Also, the protein ingredients segment is sub-segmented into essential and non-essential amino acids, glycomacropeptide (GMP), and others.
The drugs segment accounted for the largest revenue share of the global phenylketonuria market in 2022. Further, sapropterin is the most frequently used drug for treating PKU. Kuvan (sapropterin dihydrochloride) is a prescription drug that reduces blood Phenylalanine levels in adults and kids over one month with a specific form of PKU. A Phenylalanine restricted diet should be used in conjunction with Kuvan, which is the active synthetic version of the cofactor for PAH (6R)-L-erythro-5,6,7,8-tetrahydrobiopterin (BH4). It has been given Orphan Drug status and Fast Track designation for treating PKU. According to phase II and phase III clinical results, Kuvan was a safe and effective therapy in a subset of patients with mild-to-moderate PKU and mild hyperphenylalaninemia who responded to a BH4 loading test. According to the estimation of BioMarin Pharmaceutical Inc., based on clinical data, 30% to 50% of the expected 50,000 patients with PKU in developed countries who have received a diagnosis could benefit from Kuvan treatment.
Phenylketonuria Market by Distribution Channel
Based on the distribution channel, the global phenylketonuria market is segmented into hospital pharmacies, retail pharmacies, and online pharmacies.
The hospital pharmacies segment accounted for the largest revenue share of the global phenylketonuria market in 2022. Hospital pharmacies are critical in maximizing patient care by collaborating with doctors, nurses, and other healthcare workers to provide the appropriate medications and resources following established treatment procedures. Hospital pharmacists provide better patient care and offer more medication-related patient transition-of-care interventions, hourly medication order reviews, and medication counseling, according to a national survey on pharmacy practice conducted by the American Society of Health-System Pharmacists. A hospital pharmacy has a significant influence on the economy of medical expenses. Health has a direct effect on the socioeconomic growth of the country and the welfare of its people. A hospital pharmacy offers patients access to the drugs, tools, and other materials required for treating, diagnosing, and mitigating injuries, illnesses, and diseases. Pharmaceutical treatment, compounding, and delivery are all services provided by hospital pharmacies. From the patient’s perspective, hospital pharmacy services provide medications, information, and advice to inpatients, outpatients, medical personnel, and others offering care. In addition, several government initiatives, such as the discounted drug program, help patients buy expensive medications for rare diseases regardless of their financial situation. As a result, patients favor hospital pharmacies due to rising drug prices and the lack of medications in retail pharmacies, resulting in a significant revenue increase.
The most important treatment option for PKU is nutrient supplements that can be easily available in retail pharmacies and online pharmacies. Therefore, the retail and online pharmacies segments are also growing significantly due to the easy availability of drugs and supplements.
Phenylketonuria Market by Region
Based on the region, the global phenylketonuria market is segmented into North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa.
North America accounted for the largest revenue share of the global phenylketonuria market in 2022. The market in North America is likely to be influenced by rising disease prevalence. In the U.S., PKU affects 1 in 10,000 to 15,000 infants. Moreover, increased awareness is the key factor influencing market revenue growth. Additionally, the conference was sponsored by several renowned companies, such as Homology Medicine Inc., Nestle Health Sciences, PTC Therapeutics, and Abbott Laboratories. North America dominates the global phenylketonuria market due to a larger patient pool, an increase in the number of diagnostic procedures, the existence of top manufacturers, and rapid drug development.
Asia Pacific is expected to offer lucrative opportunities in the phenylketonuria market during the forecast period. The global phenylketonuria market is anticipated to be driven by increased demand for supportive care therapeutics due to increased healthcare costs in the region, a rise in medical tourism, augmentation of the pharmaceutical industry, and a high acceptance rate for novel products.
Phenylketonuria Market Competitive Landscape
Prominent companies are concentrating on strategies such as alliances, drug launches, collaborations, and mergers and acquisitions to obtain a gain edge. Additionally, they are running and supporting initiatives that aid and care for patients with uncommon diseases, such as PKU. For instance, the Rare Caregiver Respite Program received its inaugural funding from Retrophin, Inc. in May 2019. The National Organization for Rare Disorders (NORD) started the program, seeking to offer patients various services and financial support.
The prominent companies holding the largest revenue share of the global phenylketonuria market are:
Phenylketonuria Market Strategic Developments
Phenylketonuria is an autosomal recessive disorder that produces phenylalanine hydroxylase, causing an extreme phenylalanine level in the blood.
Europe is the fastest-progressing market for phenylketonuria owing to the increased prevalence of the disease.
Key companies operating the global phenylketonuria market are BioMarin Pharmaceuticals Inc., Censa Pharmaceutical, Synlogic Inc., Erytech Pharma SA, and Codexis Inc.
The development of new treatment strategies, such as enzyme and gene therapy, are revenue growth opportunities in the phenylketonuria market.
The global phenylketonuria market is expected to grow at a revenue CAGR of 12.4% during the forecast period from 2023 to 2031.
*Insights on financial performance are subject to the availability of information in the public domain