Global Phosphoglucomutase (PGM 1) Deficiency Market - 2023-2030

Overview

The phosphoglucomutase (PGM 1) deficiency market trends show rising research owing to the rising technological advancements and research studies. Several therapies are being researched for the treatment of phosphoglucomutase (PGM 1) deficiency. D-Glactose supplements are also utilized for management of phosphoglucomutase (PGM 1) deficiency.

Furthermore, rising treatment for phosphoglucomutase (PGM 1) deficiency due to the rising availability of therapies with positive outcomes and increase in the number of phosphoglucomutase (PGM 1) deficiency cases in the market, and an increase in the research and development is driving up the phosphoglucomutase (PGM 1) deficiency market size.

The market is experiencing a growth in demand for treatment from North American areas as a result of the rising advancements in this field. With significant competitors like Avalo Therapeutics, Inc., Biosynth, BioSpectra, Inc., and Merck KGaA actively operating in the market, the market will grow in upcoming time.

Dynamics

Growing Research and Development by the Researchers Drive the Growth of the Phosphoglucomutase (PGM 1) Deficiency Market

Numerous research and developments are currently taking place by the researchers for finding treatment of phosphoglucomutase 1 (PGM1) deficency. Experiments addressing either the function of the PGM1 gene and protein or the modified metabolism connected to glucose-6-P give comprehensive evidence that galactose treatment for PGM1 deficiency is ineffective. The nucleotide sugars UDP-galactose or UDP-glucose, as well as galactose-1-P, must be replenished for galactose treatment to be effective.

The proper ER- and GA-associated glycosylation, respectively, depends on these UDP-hexoses (Figures 1B and 1C). Additionally, the existence of galactose causes a metabolic rewiring of the manufacture of the two nucleotide sugars. Research has crucially demonstrated that the labeled galactose is integrated into matured N-glycans on the cell surface, demonstrating the route of action of galactose administration in this therapy for people with PGM1-CDG.

The strategy also implies that galactose therapy may not be the most efficient or burdensome way of treating affected people, but rather the direct delivery of nucleotide sugars. The information also opens up new avenues for the research and development of nutritional therapies for associated CDG with other flaws in nucleotide-sugar transport and metabolism.

Rising Collaboration by the Major Players Create a Lucrative Opportunities for Growth of the Market

Currently several major players are developing novel therapies for the treatment of phosphoglucomutase (PGM 1) deficiency. These companies are developing strategic initiatives such as partnerships, mergers, collaborations or product launch for increasing the growth of the market. The Frontiers in Congenital Disorders for Glycosylation Consortium (FCDGC) and Cerecor Inc., a biopharmaceutical firm focused on being a pioneer in the research and marketing of therapies for rare and orphan diseases, have joined forces.

With daily therapeutic levels of CERC-801 administered to about ten patients, this trial will examine the security, tolerability, and effectiveness of the drug in PGM1-CDG patients. Clinically relevant biomarkers and assessment of clinical symptoms are examples of outcome measurements. Loss of function mutations in the PGM1 gene, which codes for the enzyme involved in the intermediate conversion of glucose-6-phosphate to glucose-1-phosphate, result in PGM1 deficiency.

Uridine diphosphate (UDP)-galactose, an amino acid that contributes galactose subunits for glycoprotein synthesis, can be obtained from glucose-1-phosphate. For the time being, there has not been an approved medication to treat PGM1 deficiency. These above mentioned factors are the factors responsible for driving the growth of the market

Adverse Events Associated with the Phosphoglucomutase (PGM 1) Deficiency Treatment will Hamper the Growth of the Market

Only gastrointestinal adverse effects have been noted. Few participants reported modest gastrointestinal problems (mild constipation, unexplained vomiting, or diarrhea 1 week prior to commencing D-gal), according to a current study. One-two patient's therapy with galactose was momentarily discontinued ( 2 weeks) due to an increase in PMM2-CDG-related diarrhea.

As the constipation subsided and the polyethylene-glycol preparation was discontinued, another patient experienced the complete removal of all gastrointestinal problems. These factors may restrain the growth of the market in coming years.

Segment Analysis

The global phosphoglucomutase (PGM 1) deficiency market is segmented based on involvement type, treatment and end user.

Owing to High Utilization, the D-Galactose therapy from Treatment Segment Accounted for Approximately 71.4% of the Phosphoglucomutase (PGM 1) Deficiency Market Share

The D-galactose therapy category from treatment held the largest market share in 2022 and is expected to maintain its dominance over the forecast period due to a growth in incidence of phosphoglucomutase (PGM 1) deficiency worldwide. By replenishing the nucleotide sugar pools UDP-galactose and UDP-glucose required for Golgi-linked glycosylation or ER-linked glycosylation, respectively, D-galactose treatment in PGM1-CDG has the ability to restore glycosylation.

Regarding clinical management, supplementation with D-galactose was tested in more than 20 PGM1-CDG patients, who exhibited significant improvement in the majority of symptoms, including exercise fatigability, intolerance, hypogonadism, rhabdomyolysis, delayed puberty, and the frequency of hypoglycemia.

An increase in laboratory indicators, including as transaminase levels, stabilization of antithrombin III or FSH levels, and a decrease in CK levels, was shown in long-term follow-up studies of PGM1-CDG patients who received D-galactose. The majority of PGM1-CDG patients require D-galactose therapy in addition to dietary enrichment with complex carbs to preserve normal blood glucose levels.

Geographical Penetration

North America Accounted for Approximately 35.4% of the Market Share in 2022, Owing to the Strong Presence of Major Players and Increasing Healthcare Infrastructure Investment

Due to the rising need for phosphoglucomutase (PGM 1) deficiency treatment in healthcare, manufacturers in North America have chances of increasing their operations. There are many producers and suppliers in North America and owing to the quick economic growth of the region, industrial production has expanded, driving the demand of treatment for phosphoglucomutase (PGM 1) deficiency.

Increasing expenditure on healthcare and rising research studies, advancement of technologies for treatment of infectious disease, and increase in pharmaceutical and biotechnology business establishment across the region are also contributing to the growth of phosphoglucomutase (PGM 1) deficiency market share of this region.

It is also anticipated that the main healthcare organizations' and enterprises' collaborative research projects as well as new product development, which constantly seeks to improve available alternatives, will contribute to the expanding demand. Individuals are becoming more aware of various treatment such as D-galactose therapy for management purposes, leading to the expansion of the market in this region. The above-mentioned factors further proves the dominance of North America on a global scale.

Competitive Landscape

The major global players in the phosphoglucomutase (PGM 1) deficiency market include Avalo Therapeutics, Inc., Biosynth, BioSpectra, Inc., Glentham Life Sciences Limited, ZellBio GmbH, Merck KGaA, Otto Chemie Pvt. Ltd., SRL Chemical, Fengchen Group Co.,Ltd and Pfanstiehl among others.

COVID-19 Impact Analysis

By Involvement Type

• Congenital Malformations
• Neurological Involvement
• Ophthalmologic Involvement
• Endocrine Involvement
• Cardiac Involvement
• Gastrointestinal Involvement
• Liver Involvement
• Hematological/vascular Involvement
• Muscular Involvement

By Treatment

• D-Galactose therapy
• Gene Therapy

By End User

• Hospitals
• Ambulatory Surgical Centers
• Specialty Clinics

By Region

• North America
  • U.S.
  • Canada
  • Mexico

• Europe
  • Germany
  • U.K.
  • France
  • Spain
  • Italy
  • Rest of Europe

• South America
  • Brazil
  • Argentina
  • Rest of South America

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

• Middle East and Africa

Key Developments

• In July, 2023, the pathobiology of the cardiac dysfunction seen in PGM1-CDG was investigated using a unique conditional Pgm2 gene (the mouse homolog of human PGM1) knockout animal model (Pgm2 cKO). Studies using echocardiography supported a DCM phenotype with a markedly decreased ejection fraction along with left ventricular enlargement comparable to those found in people with PGM1-CDG. They discovered a reduction in mitochondrial activity in the hearts of KO mice. Hearts from mutant mice underwent transcriptomic analysis, which revealed a gene signature for DCM.
• On March 20, 2021, in 9 PMM2-CDG patients, an open-label pilot study using D-galactose was done. However, some milder individuals did exhibit beneficial clinical changes, and there was a tendency toward better glycosylation. Despite the fact that there was no appreciable improvement overall. To find out whether D-galactose could be employed as a supportive treatment in PMM2-CDG patients, larger placebo-controlled studies are needed.

DMI Opinion:

The global phosphoglucomutase (PGM 1) deficiency market is going to see moderate growth in upcoming years owing to rising usage of D-Galactose therapy for phosphoglucomutase (PGM 1) deficiency. Several researches are taking place worldwide for D-Galactose therapy development. Numerous advances are taking place leading to the growth of market such as gene therapy. According to DMI the phosphoglucomutase (PGM 1) deficiency market will see a decent growth with several novel therapies in the market.

Why Purchase the Report?

• To visualize the global phosphoglucomutase (PGM 1) deficiency market segmentation based on involvement type, treatment, end user and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of phosphoglucomutase (PGM 1) deficiency market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global phosphoglucomutase (PGM 1) deficiency market report would provide approximately 53 tables, 54 figures and 195 Pages.

Target Audience 2023

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies