Phenylketonuria Treatment Market

The global phenylketonuria (PKU) treatment market, which recorded a whooping valuation of US$ 446.7 million in the year 2020, is expected to witness significant growth in the coming years.

Market Overview

The phenylketonuria treatment market is projected to grow at a CAGR of 11% from 2022 to 2027. This sector was moderately affected by COVID-19, as PKU patients adapted dietary restrictions to changing environments. Adolescents and adults exhibited better metabolic control during home confinement due to teleconsultancy. The pandemic underscored a conducive environment and accessible medical connections for optimal metabolic compliance among PKU patients.

The market is also fueled by increasing clinical trials and major research and development activities. In August of the year 2022, BioMarin Pharmaceutical Inc. and PTC Therapeutics conducted clinical trials investigating the safety and efficacy of treatments like BMN 307 and PTC923 in lowering blood phenylalanine levels (less than 2 milligrams per deciliter (mg/dL) among phenylketonuria patients.

Rising awareness programs about PKU by local and national governmental organizations and healthcare systems also stimulate market growth. For instance, the National Phenylketonuria Alliance, a collaborative community, raises PKU awareness and supports local efforts for PKU education and cures. However, the high cost could inhibit market growth in the forecast period.

Key Report Findings

• The PKR treatment market is expected to grow at a CAGR of 11% from 2022 to 2027.
• The COVID-19 pandemic impacted PKU patients, forcing adaptations in dietary restrictions and treatments. Teleconsultancy emerged as a beneficial tool for better metabolic control.
• Research and clinical trials have escalated, with companies like BioMarin Pharmaceutical Inc. and PTC Therapeutics conducting studies to increase treatment efficacy.
• PKU awareness programs, provided by various organizations, governments, and regulatory bodies, are driving the growth of the phenylketonuria treatment market.
• The high cost of PKU treatment could potentially limit market growth.
• Kuvan, produced by BioMarin Pharmaceuticals, is a dominant drug in the market. Clinical trials and studies have affirmed its efficacy in reducing blood phenylalanine levels.
• The phenylketonuria treatment market is segmented by Drugs (Kuvan, Palynziq, and others) and Geography (North America, Europe, Asia-Pacific, and the Rest of the World).
• North America is anticipated to hold a significant market share due to higher PKU incidence rates and development in treatment types.
• Increasing cases of phenylketonuria in the U.S. have driven market growth in the region.
• Research activities in phenylketonuria treatment are supported by governmental bodies, leading to potential advances in therapy.

Market Drivers

Impact of COVID-19 on PKU Treatment Adherence         

The pandemic triggered adaptations in dietary restrictions and treatments among phenylketonuria patients. Teleconsultancy emerged as an essential tool for treatment adherence and better metabolic control during lockdowns. This signifies the potential of remote healthcare services to improve phenylketonuria treatment, which is expected to remain a driver post-pandemic.

Increasing Research and Clinical Trials

Key players like BioMarin Pharmaceutical Inc. and PTC Therapeutics are conducting clinical trials to explore new treatment methods to yield more effective treatments, thus driving the market. For instance, the investigation of BioMarin's BMN 307, a promising gene transfer approach, demonstrates this active research effort. The increased intensity of these activities is set to unearth new, more effective treatments, stimulating market growth and providing fresh hope for those afflicted by PKU.

Rising PKU Awareness Programs

Governments, regulatory bodies, and organizations are amplifying efforts to enhance global awareness about PKU. Notably, alliances like the National Phenylketonuria Alliance are pioneering such initiatives. By educating patients about available treatments and emphasizing the importance of early intervention, they're helping to increase the visibility of this condition. These sustained efforts are expected to fuel demand for PKU treatments and, in turn, significantly stimulate market growth.

High Incidence Rates of PKU in North America

North America, particularly the U.S., is witnessing high incidence rates of PKU, contributing to a significant market share in this region. Through the systematic implementation of newborn screening tests, PKU is often detected at an early stage, facilitating prompt initiation of diet therapy. These measures, coupled with advanced medical infrastructure and research initiatives, are playing a crucial role in driving the growth of the phenylketonuria treatment market in this region.

Efficacy of Current Treatment Options

The demonstrated effectiveness of drugs like Kuvan (sapropterin dihydrochloride) in managing PKU plays a significant role in the growth of the market. Kuvan, with its proven ability to reduce blood phenylalanine levels, provides confidence to patients and healthcare providers. This, coupled with the ongoing development of its therapeutic equivalents, has secured Kuvan's market dominance, thereby driving substantial growth in the overall phenylketonuria treatment market worldwide.

Market Opportunities

Technological Advancements in Teleconsultancy: The introduction of Dr. Reddy’s Laboratories' generic version of Kuvan signals a potential shift towards more affordable phenylketonuria treatments. If more pharmaceutical companies invest in developing generic versions of PKU drugs, it could make treatment more accessible for PKU patients, thereby expanding the market.

Rising Investments in Gene Therapy: Companies like BioMarin Pharmaceutical Inc. are investing in gene therapy (a technique that modifies genes to treat or cure disease) research for PKU treatment. If successful, this new approach could revolutionize the market and offer a curative solution for PKU. As such, the rising investment in gene therapy presents a significant market opportunity.

Increased Demand for Generic Drugs: The introduction of Dr. Reddy’s Laboratories' generic version of Kuvan signals a potential shift towards more affordable phenylketonuria treatments. If more national and international pharmaceutical companies invest in developing generic versions of PKU drugs, it could make treatment more accessible for patients, thereby expanding the market.

Untapped Markets in Developing Regions: While the incidence of PKU is less in populations in Asia and Africa, the large population sizes in these regions mean there are still a significant number of individuals with PKU. Intensified efforts to improve phenylketonuria awareness, diagnosis, and screening in these regions could open up new potential markets for phenylketonuria treatment.

Government Support for PKU Research: Governmental support and funding for PKU research and caregiver programs, those provided by the National Organization for Rare Disorders (NORD), offer opportunities for further market expansion. As more resources are directed toward understanding PKU, this could lead to the development of innovative treatments that could boost the market.

Market Challenges

High Treatment Costs Limiting Market Growth: Despite the progress made in the field of PKU treatment, the high cost poses a significant challenge. For many patients, especially in developing regions, the financial burden of treatment is unmanageable. Without measures to reduce these costs or improve access to affordable treatment, this barrier will continue to inhibit market growth.

Limited Understanding of PKU in Some Populations: Although PKU is typically identified in infancy in developed regions, the same cannot be said for all areas globally. A lack of knowledge about PKU and inadequate newborn screening programs means many cases go undiagnosed. The lack of awareness and limited diagnostic facilities challenge the market's expansion.

Dietary Compliance and Treatment Adherence: Even when patients have access to treatment, ensuring they adhere to the necessary dietary restrictions is challenging for adolescents and adults, who may find it harder to maintain a strict diet for effective treatment. Failure to adhere to these diets can lead to less effective treatment outcomes and potentially hinder market growth.

Regulatory Hurdles in Drug Approval: New drugs for PKU treatment undergo a rigorous and lengthy approval process. Not all drugs pass through these stages successfully; even those that do can experience delays, limit the number of available options and slow the growth of the market.

Regional Coverage

North America

• United States
• Canada

Europe

• Germany
• United Kingdom
• France
• Italy
• Spain
• Rest of Europe

Asia Pacific

• China
• Japan
• India
• Australia
• South Korea
• Rest of Asia-Pacific

Latin America

• Brazil
• Mexico
• The rest of Latin America

Middle East and Africa

• GCC
• The Rest of the Middle East and Africa

Company Recent Development

Major players operating in the Phenylketonuria treatment market include:

• American Gene Technologies
• Dimension Therapeutics
• BioMarin Pharmaceutical
• Synthetic Biologics
• Codexis
• SOM Innovation Biotech
• Daiichi Sankyo
• Erytech Pharma

The presence of several major players characterizes the phenylketonuria (PKU) treatment market. These companies are actively involved in the research, development, and commercialization of innovative treatments for PKU. With their years of expertise, resources, and commitment to improving patient outcomes, these major players play a crucial role in driving advancements in PKU treatment. Through ongoing research, clinical trials, and strategic collaborations, these companies are dedicated to addressing the challenges and meeting the needs of PKU patients worldwide.