The global Phenylketonuria Treatment Market was valued at USD 0.81 billion in 2023. The Phenylketonuria Treatment industry is projected to grow from USD 0.85 billion in 2024 to USD 1.20 billion by 2032, exhibiting a compound annual growth rate (CAGR) of 5.00% during the forecast period (2024 - 2032). The rising incidence of phenylketonuria and increasing advancements in diagnostic technologies are the primary market drivers propelling the market's expansion.
Source: Secondary Research, Primary Research, MRFR Database and Analyst Review
June 2024: BioMarin Pharmaceutical is launching a Phase III clinical trial for their gene therapy candidate BMN 307, aiming to provide a long-term solution for PKU patients by addressing the genetic cause.
May 2024: PTC Therapeutics' Phase II study of PTC923, an investigational drug for PKU patients, showed significant improvements in phenylalanine management, suggesting it could be a potential new treatment option.
April 2024: Synlogic completed a Phase I clinical trial for SYNB1618, a synthetic biotic medicine designed to metabolize phenylalanine in the gastrointestinal tract, showing safety and tolerability in PKU patients.
Kuvan is the main medication present for the productive treatment of phenylketonuria, which BioMarin Drugs is making. It adds more BH4 mixtures and helps in animating the phenylalanine hydroxylase chemical, which then breaks down the phenylalanine protein. At the same time, the expanding launches and advancements are further driving the development of the market. For instance, In April 2021, Dr. Reddy's Research centers launched the Sapropterin Dihydrochloride Powder for Oral Arrangement, 100 mg), a therapeutic identical conventional variant of Kuvan (sapropterin dihydrochloride) Powder for Oral Arrangement, 100 mg, USP, supported by the US Food and Medication Organization. For example, In April 2021, Dr. Reddy's Research facilities disclosed Sapropterin Dihydrochloride Powder for Oral Arrangement, 100 mg of a therapeutic comparable conventional variant of Kuvan (sapropterin dihydrochloride) Powder for Oral Arrangement, 100 mg, USP, in the US.
Kuvan permitted patients with PKU who were not on a restricted eating routine to build their day-to-day phenylalanine consumption by 17.5 mg per kg body weight following ten weeks compared to 3.3 mg/kg with placebo. At the point when Kuvan, in addition to count calories limitation, was contrasted and diet limitation alone, the normal phenylalanine admission that was endured following 26 weeks was 81 mg/kg in the Kuvan gathering and 50 mg/kg in the gathering on diet limitation alone. For the treatment of BH4 deficiency, which is an exceptionally uncommon condition, In August 2021, the public library of medication introduced the consequences of three examinations from the distributed literature in which a few patients were treated with sapropterin for a normal of 15.5 months. In these examinations, patients showed an improvement in blood phenylalanine levels and different markers of the illness when they were taking the medication.
Furthermore, Personalized medication permits medical care suppliers to tailor therapy plans for PKU patients in light of their special hereditary cosmetics, metabolic profiles, and individual reactions to treatment. This customization can streamline treatment adequacy and limit adverse impacts. Pharmacogenomic testing predicts how patients will answer explicit prescriptions in light of their hereditary variations. With regards to PKU treatment, pharmacogenomic experiences can direct medicine choice and dosing, advancing restorative results and reducing the risk of adverse medication responses.
Dysregulation of neurotransmitter pathways, especially dopamine and serotonin, is implicated in the neuropsychiatric indications related to PKU. Focusing on neurotransmitter frameworks through pharmacological mediations offers expected helpful advantages for overseeing cognitive and behavioral symptoms in PKU patients, which is driving the market CAGR. BH4 is a cofactor essential for PAH action, and deficiencies in BH4 lead to diminished enzymatic capability and raised phenylalanine levels in PKU patients. Balancing out BH4 or advancing its synthesis represents a therapeutic target to reestablish PAH movement and further develop phenylalanine digestion in BH4-responsive PKU patients. Thus driving the Phenylketonuria Treatment market revenue.
Based on Drug, the global Phenylketonuria Treatment market segmentation includes kuvan, playnziq, and other drugs. In 2023, the kuvan segment dominated the market. Kuvan is primarily shown for the treatment of BH4-responsive PKU, a subset of PKU patients who have a deficiency in tetrahydrobiopterin (BH4), a cofactor essential for the movement of phenylalanine hydroxylase (PAH), the chemical liable for processing phenylalanine. Kuvan works by expanding the action of the PAH catalyst, in this way upgrading the change of phenylalanine to tyrosine, a cycle that is impeded in PKU patients. By expanding PAH action, Kuvan helps reduce blood phenylalanine levels, which are normally raised in PKU.
The Playnziq category is anticipated to be the fastest growing. Palynziq is a type of catalyst substitution treatment that gives the PAH enzyme, which is deficient or dysfunctional in PKU patients. By providing an exogenous PAH enzyme, Palynziq helps metabolize phenylalanine to tyrosine, diminishing blood phenylalanine levels and avoiding its accumulation in the body. Treatment with Palynziq might be considered for the relaxation of dietary limitations in PKU patients by lowering the reliance on strict dietary control for phenylalanine management. Nonetheless, dietary adjustments and observing phenylalanine intake are as yet important to ensure optimal metabolic control.
Figure 1: Global Phenylketonuria Treatment Market by Drug, 2023 & 2032 (USD Billion)
Source: Secondary Research, Primary Research, MRFR Database and Analyst Review
Based on Distribution Channel, the global Phenylketonuria Treatment market segmentation includes hospital pharmacies, retail pharmacies, online pharmacies, and others. In 2023, the hospital pharmacies category generated the most income. Hospital pharmacies professionally prescribe prescriptions, including PKU medicines like Kuvan (sapropterin dihydrochloride) and Palynziq (pegvaliase), to patients who are getting care inside the clinic setting. Drug specialists and drug store staff give instruction and guidance to PKU patients and their parental figures on the proper administration, dosing, and likely symptoms of PKU meds. This education guarantees protected and effective drug use.
The Phenylketonuria Treatment retail pharmacies segment market is anticipated to grow the fastest during the forecast period. Retail drug stores help patients explore protection coverage and repayment for PKU prescriptions. Drug specialists give direction on protection necessities, earlier approval cycles, and copayment help projects to assist patients with getting reasonable treatment choices.
The study provides market insights into North America, Europe, Asia-Pacific, and the Rest of the World by region. The North American Phenylketonuria Treatment market will dominate this market because of a higher occurrence rate of phenylketonuria and the development of novel treatments. For example, In June 2022, according to information from the Public Library of Medicine, the general frequency of phenylketonuria in the US is around 1/15,000. This frequency is more prominent for Caucasian and Local American populaces and less for African American, Hispanic, and Asian populaces. A similar source expressed that in the US, PKU is often discovered by newborn screening tests, and dietary treatment begins in conference with a dietitian and geneticist/digestion-trained professional. Gentle types of PKU in an infant can, be that as it may, go undetected, assuming the mother is released too early or, on the other hand, in the event that the infant has not consumed any protein.
Further, the major countries studied in the market report are the US, Canada, Germany, France, the UK, Italy, Spain, China, Japan, India, Australia, South Korea, and Brazil.
Figure 2: Global Phenylketonuria Treatment Market Share By Region 2023 (USD Billion)
Source: Secondary Research, Primary Research, MRFR Database and Analyst Review
Europe's Phenylketonuria Treatment market accounts for the second-largest market share. Europe has dynamic patient promotion associations devoted to bringing issues to light, offering help, and supporting the requirements of people and families impacted by PKU. These gatherings assume a significant part in impacting medical care strategies and advancing examination drives. Further, the German Phenylketonuria Treatment market held the largest market share, and the UK Phenylketonuria Treatment market was the fastest-growing market in the European region.
The Asia-Pacific Phenylketonuria Treatment Market is expected to grow at the fastest CAGR from 2024 to 2032. Monetary expansion in numerous nations inside the Asia Pacific area has prompted expanded medical services spending and further developed access to clinical benefits. This Increased spending on healthcare opens up options to fund the management and treatment of PKU. Moreover, China’s Phenylketonuria Treatment market held the largest market share, and the Indian Phenylketonuria Treatment market was the fastest-growing market in the Asia-Pacific region.
The industry's leading players will keep spending a lot of money on research and development to increase their product lines, which will propel the Phenylketonuria Treatment market's growth. Contractual agreements, mergers and acquisitions, increased investments, collaboration with other companies, and the production of new products are examples of significant market developments. To increase their worldwide reach, market players also take part in a variety of strategic initiatives. In order to survive and expand in a more competitive and dynamic market, the Phenylketonuria Treatment industry must offer reasonably priced products and services.
One of the main strategies manufacturers use in the worldwide Phenylketonuria Treatment market is local manufacturing, which expands the market sector and helps customers by lowering operating costs. The Phenylketonuria Treatment industry has recently provided some of the great medical advantages. Major players in the Phenylketonuria Treatment market, including Cambrooke Therapeutics, American Gene Technologies International, Inc., BioMarin Pharmaceutical, Inc., Codexis, Inc., Agios Pharmaceuticals Inc., PTC Therapeutics, Synlogic, Pristine Organics, Galen Pharma, Relief Therapeutics Holding SA., and others, are making investments in R&D activities in an effort to boost market demand.
BioMarin Drug Inc (BioMarin) is a biotechnology organization that creates and popularizes imaginative medications for the therapy of rare hereditary infections and ailments like Duchenne solid dystrophy (DMD), achondroplasia, phenylketonuria (PKU), late juvenile neuronal ceroid lipofuscinosis (CLN2) and hemophilia A. The organization centers around creating advanced medicines for crippling and hazardous sicknesses. Its item class comprises numerous clinical and preclinical contributions, which incorporate Naglazyme-Mucopolysaccharidosis Type VI (MPS VI), Aldurazyme-MPS I, and others. September 2020, BioMarin Drug has been leading a Stage II clinical examination study to assess the well-being, effectiveness, and decency of BMN 307 in grown-up PKU members with PAH deficiency.
PTC Therapeutics Inc (PTC Therapeutics) finds, creates, and markets drugs for the treatment of rare problems. Its advertised items incorporate Evrysdi (risdiplam) for spinal strong decay, Translarna (ataluren), and Emflaza (deflazacort) for the treatment of Duchenne solid dystrophy. The organization's pipeline applicants focus on the treatment of Huntington's sickness, youngsters with acquired mitochondrial infection, leiomyosarcoma, intense myelogenous leukemia, and sweet-smelling L-amino corrosive decarboxylase inadequacy. PTC Therapeutics works as a team with different biotechnology and drug organizations to propel its pipeline candidates. February 2022, PTC Therapeutics has been running a Stage III clinical trial review to evaluate the drawn-out well-being of PTC923 in phenylketonuria patients and to evaluate changes from baseline in dietary phenylalanine (Phe)/protein consumption.
July 2022: Preclinical findings for Jnana Therapeutics' flagship program, which might be the first oral therapy of its kind for phenylketonuria (PKU), have been presented. Information has been displayed at the Vancouver, Washington, National PKU Alliance Conference in 2022.
August 2021: The USD 50 million Series B funding for Janana Therapeutics' Lead Phenylketonuria Program and Small Molecule Pipeline, which is based on the Next-Gen Chemoproteomic Platform, has closed.
| Report Attribute/Metric | Details |
| Market Size 2023 | USD 0.81 Billion |
| Market Size 2024 | USD 0.85 Billion |
| Market Size 2032 | USD 1.20 Billion |
| Compound Annual Growth Rate (CAGR) | 5.00% (2024-2032) |
| Base Year | 2023 |
| Market Forecast Period | 2024-2032 |
| Historical Data | 2019-2022 |
| Market Forecast Units | Value (USD Billion) |
| Report Coverage | Revenue Forecast, Market Competitive Landscape, Growth Factors, and Trends |
| Segments Covered | Drug, Distribution Channel, and Region |
| Geographies Covered | North America, Europe, Asia Pacific, and the Rest of the World |
| Countries Covered | The US, Canada, Germany, France, UK, Italy, Spain, China, Japan, India, Australia, South Korea, and Brazil |
| Key Companies Profiled | Cambrooke Therapeutics, American Gene Technologies International, Inc., BioMarin Pharmaceutical, Inc., Codexis, Inc., Agios Pharmaceuticals Inc, PTC Therapeutics, Synlogic, Pristine Organics, Galen Pharma, and Relief Therapeutics Holding SA |
| Key Market Opportunities | The expansion of newborn screening programs reg, regulatory incentives, and orphan drug designation |
| Key Market Dynamics | The rising incidence of phenylketonuria and increasing advancements in diagnostic technologies |
Frequently Asked Questions (FAQ) :
The global Phenylketonuria Treatment market size was valued at USD 0.81 Billion in 2023.
The global market is projected to grow at a CAGR of 5.00% during the forecast period, 2024-2032.
North America had the largest share of the global market
The key players in the market are Cambrooke Therapeutics, American Gene Technologies International, Inc., BioMarin Pharmaceutical, Inc., Codexis, Inc., Agios Pharmaceuticals Inc, PTC Therapeutics, Synlogic, Pristine Organics, Galen Pharma, and Relief Therapeutics Holding SA.
The Kuvan category dominated the market in 2023.
Hospital Pharmacies have the largest share of the global market.
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Assistant Manager He holds an experience of about 7+ years in market research and business consulting, working under the spectrum of life sciences and healthcare domains. rahul conceptualizes and implements a scalable business strategy and provides strategic leadership to the clients. his expertise lies in market estimation, competitive intelligence, pipeline analysis, customer assessment, etc. in addition to the above, his other responsibility includes strategic tracking of high growth markets & advising clients on the potential areas of focus they could direct their business initiatives
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