Phenylketonuria Treatment

Phenylketonuria Treatment

Global Phenylketonuria Treatment Market to Reach US$1.6 Billion by 2030

The global market for Phenylketonuria Treatment estimated at US$975.0 Million in the year 2023, is expected to reach US$1.6 Billion by 2030, growing at a CAGR of 7.0% over the analysis period 2023-2030. Palynziq Drug, one of the segments analyzed in the report, is expected to record a 7.4% CAGR and reach US$918.2 Million by the end of the analysis period. Growth in the Kuvan Drug segment is estimated at 6.6% CAGR over the analysis period.

The U.S. Market is Estimated at US$265.6 Million While China is Forecast to Grow at 11.2% CAGR

The Phenylketonuria Treatment market in the U.S. is estimated at US$265.6 Million in the year 2023. China, the world`s second largest economy, is forecast to reach a projected market size of US$343.9 Million by the year 2030 trailing a CAGR of 11.2% over the analysis period 2023-2030. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at a CAGR of 3.0% and 6.9% respectively over the analysis period. Within Europe, Germany is forecast to grow at approximately 4.4% CAGR.

Global Phenylketonuria Treatment Market - Key Trends and Drivers Summarized

Phenylketonuria (PKU) is a rare inherited metabolic disorder caused by a deficiency in the enzyme phenylalanine hydroxylase (PAH), which is necessary for the metabolism of the amino acid phenylalanine. When PAH is deficient or absent, phenylalanine accumulates in the blood and brain, leading to severe cognitive impairments and neurological issues if left untreated. The cornerstone of PKU management has traditionally been a strict, lifelong diet low in phenylalanine, which involves the exclusion of high-protein foods and the inclusion of specially formulated medical foods and phenylalanine-free amino acid supplements. Early diagnosis through newborn screening programs has been critical in preventing the severe outcomes associated with untreated PKU, allowing for the timely initiation of dietary management and close monitoring of phenylalanine levels.

In recent years, advances in PKU treatment have expanded beyond dietary management, providing new therapeutic options that improve patient outcomes and quality of life. One significant development is the use of pharmacological treatments such as sapropterin dihydrochloride, a synthetic form of the cofactor tetrahydrobiopterin (BH4), which can enhance the residual activity of PAH in some patients with mild to moderate PKU. Additionally, pegvaliase, an enzyme substitution therapy, has been approved for adults with PKU who have uncontrolled blood phenylalanine levels despite dietary management. Pegvaliase works by breaking down phenylalanine in the bloodstream, offering a new avenue for those who struggle to maintain low phenylalanine levels through diet alone. Gene therapy and other innovative treatments are also under investigation, aiming to provide more permanent solutions by addressing the underlying genetic cause of PKU.

The growth in the phenylketonuria treatment market is driven by several factors. The increasing awareness and understanding of PKU among healthcare providers and patients have led to earlier and more accurate diagnoses, boosting the demand for effective treatments. Technological advancements and ongoing research have facilitated the development of new pharmacological therapies and innovative treatments, expanding the options available for PKU management. The rise of personalized medicine, which tailors treatments to individual genetic profiles, is also driving market growth by offering more targeted and effective therapies. Additionally, the expansion of newborn screening programs globally has increased the detection rates of PKU, further fueling the need for comprehensive treatment solutions. The growing availability of specialized medical foods and supplements, coupled with supportive healthcare policies and funding for rare disease treatments, are creating a favorable market environment. As these trends continue to evolve, the phenylketonuria treatment market is poised for significant growth, driven by advancements in medical science and a heightened focus on improving patient outcomes.

Select Competitors (Total 86 Featured) -

• Ajinomoto Cambrooke, Inc.
• American Gene Technologies Inc.
• Arla Foods Ingredients Group P/S
• BioMarin Pharmaceutical, Inc.
• Codexis, Inc.
• Metropolis Healthcare Ltd.
• Nestle Health Science SA
• Nutricia
• PTC Therapeutics
• SOM Biotech
• Synlogic
• Travere Therapeutics, Inc.
• Ultragenyx Pharmaceutical, Inc.

Please note: Reports are sold as single-site single-user licenses. Electronic versions require 24-48 hours as each copy is customized to the client with digital controls and custom watermarks. The Publisher uses digital controls protecting against copying and printing is restricted to one full copy to be used at the same location.

The latest version of Adobe Acrobat Reader is required to view the report. Upon ordering an electronic version, the Publisher will provide a link to download the purchased report.

Prior to fulfillment of an order, the client will be required to sign a document detailing the purchase terms for a publication from this publisher.