Published: 2022 November 04

Phenylketonuria Treatment Market

SKU : PH2150
180 pages
Report Summary
Table of Contents
List of Tables & Figures

Phenylketonuria Treatment Market is segmented By Product Type (Drugs, Kuvan (sapropterin hydrochloride), Palynziq (pegvaliase-pqpz), Dietary Supplements, Others), By Route of Administration (Oral (Tablets and Capsules, Syrups, Powder), Parenteral (Intravenous, Subcutaneous)), By Distribution Channel (Hospital Pharmacies, Drug Stores, Online Pharmacies, Others), and By Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) – Share, Size, Outlook, and Opportunity Analysis, 2022-2029.

Phenylketonuria Treatment Market is estimated to reach at a CAGR of 9.46% during the forecast period (2022-2029).

Phenylketonuria Treatment Market - Strategic Insights



Market CAGR


Segments Covered

By Product Type, By Route of Administration, By Distribution Channel By End User, and By Region

Report Insights Covered

Competitive Landscape Analysis, Company Profile Analysis, Market Size, Share, Growth, Demand, Recent Developments, Mergers and acquisitions, New Product Launches, Growth Strategies, Revenue Analysis, and Other key insights.

Fastest Growing Region

Asia Pacific

Largest Market Share 

North America

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Phenylketonuria is a rare hereditary condition in which the body accumulates the amino acid phenylalanine. PKU is caused by a mutation in a gene that aids in the production of the phenylalanine-degrading enzyme. Seizures, mental illnesses, and other untreated disorders can result from an excess of phenylalanine in the blood. Although there is no cure for PKU, medication can help to prevent intellectual deficits and other health issues. PKU is treated by adhering to a PKU diet that restricts foods containing phenylalanine.

Market Dynamics

The global phenylketonuria treatment market is growing due to several factors, such as the rising research on new interventions for phenylketonuria.

Clinical trials conducted by the market players to treat phenylketonuria are expected to drive market growth.

BioMarin Pharmaceutical Inc. conducted a Phase 1/2 Open-Label, Dose Escalation research in Phenylketonuria patients with Plasma Phe Levels > 600 mol/L to investigate the safety and efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase. Moreover, PTC Therapeutics also conducted a clinical trial to assess the efficacy of PTC923 in lowering blood phenylalanine (Phe) levels in Phenylketonuria patients, as evaluated by the mean change in blood Phe levels from baseline to Weeks 5 and 6. (that is, the average of each respective treatment dose 2-week period of double-blind treatment). Thus, the market is expected to drive in the forecast period from the above statements.

The lack of awareness and high cost are expected to hinder global phenylketonuria treatment market growth.

Lack of awareness regarding the rare disease, the high cost of PKU treatment, irregular reimbursement policies, and lack of treatment regimen compliance are some factors expected to hamper the market growth in the forecast period.

Industry analysis.

The global phenylketonuria treatment market report will provide an in-depth analysis of the market based on various industry factors such as porter`s five forces, innovations, new product launches, pricing, and pipeline analysis.

COVID-19 Impact Analysis.

The COVID-19 pandemic has moderately impacted healthcare systems and the market. Neurocognitive outcome and phenylketonuria (PKU) prognosis are directly related to lifelong phenylalanine (Phe) levels and treatment adherence. PKU patients had to adjust their dietary restrictions to the new environment. While younger patients may have been less exposed (meals strictly according to diet plan regardless of setting), adolescent and adult patients strongly reflected the obligation to stay home by showing better metabolic control. Multiple factors could have contributed to this, including the availability of teleconsultancy, which allowed for easier connections. Still, findings show how the pandemic and the environment can significantly impact PKU treatment adherence and how removing distance barriers can improve and optimize metabolic compliance. Thus, from the above statements, the market got affected, and it is expected to gain traction quickly with the resumption of economic activities.

Segment Analysis

The dietary supplement segment is expected to hold the largest market share in the phenylketonuria treatment market throughout the forecast period (2022-2029).

The dietary supplement segment is expected to dominate in 2021. The segment's growth is beneficial because diet limits foods with phenylalanine. It means the diet must be low in protein, and people with PKU work closely with a doctor or nutritionist to maintain a healthy food balance while decreasing their phenylalanine intake. They must also keep track of the amount of phenylalanine in the meals they eat throughout the day to track their phenylalanine levels. Moreover, Children with PKU must take PKU formula to ensure they continue obtaining adequate protein. Except for phenylalanine, it contains all of the essential amino acids. At specialty health stores, you can also get low-protein, PKU-friendly foods. For instance, Breast milk is given to infants with PKU. They generally need to take a particular formula called Lofenalac as well. When your infant is old enough to eat solid foods, you should avoid giving them protein-rich foods. Thus, the market segment is expected to hold the largest market share in the forecast period from the above statements.

Geographical Analysis

The North American region holds the largest market share in the global phenylketonuria treatment market.

In 2021, North America accounted for the highest revenue share. The increasing prevalence of phenylketonuria and higher adoption of phenylketonuria treatment-based diets, government support for ongoing clinical research and product launches by the market players in the region are expected to boost in the forecast period. For instance, PKU affects 1 in 10,000 to 15,000 babies in the United States. Most instances of PKU are diagnosed by newborn screening shortly after birth, and treatment is started immediately. Moreover, In terms of phenylketonuria treatment-based diet adoption, the United States is a leader. Patients in the country have taken a lot of PKU substances, including essential and non-essential amino acids.

Palynziq (pegvaliase-pqpz) has also been approved by the Food and Drug Administration (FDA) for adults with PKU. Palynziq is an injectable enzyme therapy for patients with uncontrolled blood phenylalanine levels despite existing medication. BioMarin Pharmaceutical Inc. manufactures Palynziq. Thus, the North American region is expected to hold the largest market share in the forecast period from the above statements.

Competitive Landscape

Major key players in the phenylketonuria treatment market are BioMarin, SOM Innovation Biotech SL, Homology Medicines, Inc., Par Pharmaceutical, PTC Therapeutics, and Synlogic, Inc. The key players are adopting various strategies such as product launches, mergers & acquisitions, partnerships, and collaborations, contributing to the growth of the Phenylketonuria Treatment market globally. For instance, on Dec 16, 2021, BioMarin and Skyline Therapeutics entered a strategic collaboration to develop novel gene therapies for cardiovascular diseases. Also, on June 13, 2022, the U.S. Food and Drug Administration (FDA) lifted the clinical hold previously placed on the pheNIX gene therapy clinical trial with investigational HMI-102 for adults with phenylketonuria (PKU) of Homology Medicines, Inc.

BioMarin Pharmaceutical Inc.


BioMarin Pharmaceutical Inc. is an American biotechnology company headquartered in San Rafael, California. It has offices and facilities worldwide, including in the United States, South America, Asia, and Europe. Enzyme replacement medicines are BioMarin's main business and research focus (ERTs). By manufacturing laronidase, BioMarin was the first company to deliver therapies for mucopolysaccharidosis type I (MPS I) (Aldurazyme, commercialized by Genzyme Corporation). BioMarin was also the first business to develop phenylketonuria treatments (PKU).

Product Portfolio:

 KUVAN (sapropterin dihydrochloride): KUVAN (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution are prescription medicines used to lower blood Phe levels in adults and children over one month of age with a certain type of Phenylketonuria (PKU). It is to be used along with a Phe-restricted diet.

The global phenylketonuria treatment market report would provide access to approximately 40+ market data tables, 45+ figures, and in the range of 200 (approximate) pages.


What is the Projected CAGR value of the Phenylketonuria Treatment Market?

Phenylketonuria Treatment Market is expected to grow at a CAGR of 9.46% during the forecasting period 2022-2029.

Which region controlled the global market during 2022-2029?

North America region Controls the Phenylketonuria Treatment Market during 2022-2029.

Which is the fastest growing region in the Phenylketonuria Treatment Market?

Among all regions, Asia Pacific is the fastest growing market share during the forecast period.

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