Phenylketonuria Treatment Market to grow with a CAGR of 11.20%
Increasing
healthcare infrastructure and a supportive regulatory environment are the major
drivers for the Global Phenylketonuria Treatment Market.
According
to TechSci Research report, “Global Phenylketonuria Treatment Market Industry
Size, Share, Trends, Competition, Opportunity and Forecast, 2018-2028”, Global Phenylketonuria
Treatment Market has valued at USD 502.18 million in 2022 and is anticipated to
witness an impressive growth in the forecast period with a CAGR of 11.00%
through 2028. This can be due to
collaborations and partnerships among leading companies with a diverse approach
to merge the expertise of individual companies and to strengthen their position
in the market.
Advancements
in the treatment of Phenylketonuria (PKU) have been ongoing, with a focus on
improving the management of this genetic disorder. PKU is characterized by the
inability to metabolize phenylalanine, an amino acid found in
protein-containing foods. The treatment aims to maintain safe blood
phenylalanine levels while ensuring adequate nutrition. Glycomacropeptide (GMP)
based protein substitutes have been developed as an alternative to traditional
amino acid-based medical foods. GMP is a naturally low-phenylalanine protein
derived from cheese whey. Advances in the formulation of protein substitutes
have led to better-tasting and more convenient products, improving patient
compliance. Companies have introduced low-protein food products specifically
designed for PKU patients, making it easier to adhere to dietary restrictions.
PEGylated Phenylalanine Ammonia Lyase (PAL) has been developed to help break
down excess phenylalanine in the blood. These therapies are administered by
injection and can be effective in managing PKU.
An
uncommon hereditary condition called phenylketonuria (PKU) causes elevated
blood levels of the amino acid phenylalanine. Proteins derived from food and
nutrition include the amino acid phenylalanine. In the human body, the
phenylalanine hydroxylase enzyme transforms phenylalanine into the amino acid
tyrosine. Seizures, mental illnesses, and several other untreated disorders can
be brought on by an excess of phenylalanine in the blood.
In
May 2020, PTC Therapeutics, Inc. announced that it has reached a deal to buy
Censa Pharmaceuticals, Inc., a biopharmaceutical company specialising in the
development of CNSA-001 (sepiapterin), a clinical-stage investigational therapy
for orphan metabolic diseases, including phenylketonuria (PKU) and other
conditions linked to defects in the tetrahydrobiopterin (BH4) biochemical
pathways that can be identified at birth. The boards of both corporations gave
their approval to the deal. CNSA-001 has being investigated as a potential
therapy for rare metabolic disorders linked to flaws in the biochemical
processes that produce tetrahydrobiopterin. As a precursor to intracellular
tetrahydrobiopterin, which is a vital enzyme cofactor involved in the
metabolism and synthesis of various metabolic products, sepiapterin, a
synthetic substance, is used in CNSA-001, an oral formulation. The main and
secondary objectives of the Phase 2 study testing CNSA-001 for PKU were reached
in December 20191, and the programme is now ready for Phase 3.
Browse over XX market data Figures and spread through 110 Pages and an
in-depth TOC on "Phenylketonuria Treatment Market”
Patient
adherence to treatment is a significant challenge in the Global Phenylketonuria
(PKU) Treatment Market. PKU is a lifelong condition that requires strict
dietary management and, in some cases, pharmacological interventions. Adherence
to treatment is crucial for maintaining safe blood phenylalanine levels and
preventing the adverse effects of elevated phenylalanine, including
intellectual and developmental disabilities. The cornerstone of PKU treatment
involves adhering to a strict low-phenylalanine diet. This diet restricts the
consumption of protein-containing foods such as meat, dairy, and grains.
Dietary limitations can be challenging to maintain over a lifetime. Many
medical foods and supplements designed for PKU patients have a unique taste and
texture, which can be unappealing. This can make it difficult for patients,
especially children, to consume these products regularly. The dietary
restrictions and special dietary needs of PKU can lead to feelings of isolation
and frustration. Patients may experience social challenges when dining with
friends or family, leading to non-compliance.
Global Phenylketonuria Treatment Market segmentation
is based on
Route of Administration, Product, Distribution
Channel, Region and Company
Based on Route of Administration, Global Phenylketonuria
Treatment Market is segmented into Oral and Parenteral. The parenteral route of
administration refers to the method of delivering medications or substances
directly into the body through means other than the digestive tract. Unlike
oral or enteral administration, which involves ingestion through the mouth and
digestion in the gastrointestinal tract, parenteral administration bypasses the
digestive system entirely. Instead, it delivers substances directly into the
bloodstream or other tissues through various methods, such as injections or
infusions. Parenteral administration is commonly used when rapid, precise, or
reliable delivery of medication is required.
Based on Region, North America dominated the Global Phenylketonuria
Treatment Market. North America had relatively good access to PKU
treatments, including enzyme replacement therapies and specialized low-protein
medical foods. Health insurance coverage and reimbursement policies in the
region also supported access to these treatments. Patient advocacy groups in
North America were active in raising awareness, advocating for research
funding, and promoting policies that benefited PKU patients. Their efforts
played a role in driving progress in the field. North America was a hub for
clinical trials and research related to PKU treatments. This research
contributed to advancements in treatment options and expanded the knowledge
base. The presence of a robust pharmaceutical and biotechnology industry in
North America facilitated the development and commercialization of PKU
treatments. This industry's expertise and resources were significant drivers of
market dominance.
Asia-pacific region to fastest growth in
the Global Phenylketonuria Treatment Market. Awareness of rare genetic
disorders like PKU has been growing in the Asia-Pacific region. As awareness
increases, more individuals may be diagnosed with PKU through newborn screening
programs and genetic testing, driving demand for treatment. The Asia-Pacific
region is home to a significant portion of the world's population. As the
population grows, so does the number of individuals potentially affected by
PKU, contributing to increased demand for PKU treatments. Many countries in the
Asia-Pacific region are experiencing rapid economic growth, leading to
increased healthcare infrastructure and resources. This growth can improve
access to specialized care and PKU treatments
Some
of the major companies operating in the Global
Phenylketonuria Treatment Market include:
- BioMarin
Pharmaceutical Inc.
- Synlogic,
Inc.
- Retrophin,
Inc.
- Daiichi
Sankyo Company, Limited
- Codexis,
Inc.
- SOM
Innovation Biotech SL
- Homology
Medicines, Inc.
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“Certain areas, particularly in
North America, are projected to exert significant demand for Phenylketonuria
Treatment. The growth in the competitive landscape and the presence of
well-established companies in the market, committed to enhance the overall
wellbeing of people each year, are expected to contribute to a remarkable
growth of the Global Phenylketonuria Treatment Market in the forecast period,"
said Mr. Karan Chechi, Research Director with TechSci Research, a
research-based Global management consulting firm.
Phenylketonuria Treatment Market – Global
Industry Size, Share, Trends, Opportunity, and Forecast, 2018-2028 Segmented by
Route of Administration (Oral,
Parenteral), by Product (Dietary Supplement, Drugs), by Distribution Channel (Hospital Pharmacies,
Online Pharmacies, Pediatric Clinics), by region, and
Competition evaluated the future growth potential of Global Phenylketonuria
Treatment Market and provides statistics & information on market size, structure,
and future market growth. The report intends to provide innovative market
intelligence and help decision makers take sound investment decisions. Besides,
the report also identifies and analyzes the emerging trends along with
essential drivers, challenges, and opportunities in Global Phenylketonuria
Treatment Market.
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