Phenylketonuria Treatment

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

The global market for Phenylketonuria Treatment estimated at US$1.0 Billion in the year 2024, is expected to reach US$1.6 Billion by 2030, growing at a CAGR of 6.9% over the analysis period 2024-2030. Palynziq, one of the segments analyzed in the report, is expected to record a 7.3% CAGR and reach US$918.2 Million by the end of the analysis period. Growth in the Kuvan segment is estimated at 6.5% CAGR over the analysis period.

The U.S. Market is Estimated at US$282.5 Million While China is Forecast to Grow at 11.0% CAGR

The Phenylketonuria Treatment market in the U.S. is estimated at US$282.5 Million in the year 2024. 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 11.0% over the analysis period 2024-2030. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at a CAGR of 2.9% and 6.9% respectively over the analysis period. Within Europe, Germany is forecast to grow at approximately 4.3% 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.

SCOPE OF STUDY:

The report analyzes the Phenylketonuria Treatment market in terms of units by the following Segments, and Geographic Regions/Countries:

Segments:
Drug Type (Palynziq, Kuvan, Other Drug Types)

Geographic Regions/Countries:
World; United States; Canada; Japan; China; Europe (France; Germany; Italy; United Kingdom; Spain; Russia; and Rest of Europe); Asia-Pacific (Australia; India; South Korea; and Rest of Asia-Pacific); Latin America (Argentina; Brazil; Mexico; and Rest of Latin America); Middle East (Iran; Israel; Saudi Arabia; United Arab Emirates; and Rest of Middle East); and Africa.

Select Competitors (Total 43 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.

TARIFF IMPACT FACTOR

Our new release incorporates impact of tariffs on geographical markets as we predict a shift in competitiveness of companies based on HQ country, manufacturing base, exports and imports (finished goods and OEM). This intricate and multifaceted market reality will impact competitors by artificially increasing the COGS, reducing profitability, reconfiguring supply chains, amongst other micro and macro market dynamics.

We are diligently following expert opinions of leading Chief Economists (14,949), Think Tanks (62), Trade & Industry bodies (171) worldwide, as they assess impact and address new market realities for their ecosystems. Experts and economists from every major country are tracked for their opinions on tariffs and how they will impact their countries.

We expect this chaos to play out over the next 2-3 months and a new world order is established with more clarity. We are tracking these developments on a real time basis.

As we release this report, U.S. Trade Representatives are pushing their counterparts in 183 countries for an early closure to bilateral tariff negotiations. Most of the major trading partners also have initiated trade agreements with other key trading nations, outside of those in the works with the United States. We are tracking such secondary fallouts as supply chains shift.

To our valued clients, we say, we have your back. We will present a simplified market reassessment by incorporating these changes!

APRIL 2025: NEGOTIATION PHASE

Our April release addresses the impact of tariffs on the overall global market and presents market adjustments by geography. Our trajectories are based on historic data and evolving market impacting factors.

JULY 2025 FINAL TARIFF RESET

Complimentary Update: Our clients will also receive a complimentary update in July after a final reset is announced between nations. The final updated version incorporates clearly defined Tariff Impact Analyses.

Reciprocal and Bilateral Trade & Tariff Impact Analyses:

USA
CHINA
MEXICO
CANADA
EU
JAPAN
INDIA
176 OTHER COUNTRIES.

Leading Economists - Our knowledge base tracks 14,949 economists including a select group of most influential Chief Economists of nations, think tanks, trade and industry bodies, big enterprises, and domain experts who are sharing views on the fallout of this unprecedented paradigm shift in the global econometric landscape. Most of our 16,491+ reports have incorporated this two-stage release schedule based on milestones.

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