United States Phosphoglucomutase (PGM 1) Deficiency Market is expected to reach US$ 850 million by 2032 | Major Companies - Avalo Therapeutics, Inc., Biosynth, BioSpectra, Inc.

Market Size and Growth:

The Global Phosphoglucomutase (PGM 1) Deficiency Market size reached US$ 145 million in 2024 and is expected to reach US$ 320 million by 2034, growing with a CAGR of 8.2% during the forecast period 2024-2034. The Market is growing due to increasing awareness and diagnosis of PGM 1 deficiency, along with rising investment in novel therapies and supportive treatments.

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The Phosphoglucomutase (PGM 1) Deficiency Market refers to the global industry focused on the diagnosis, treatment, and management of PGM 1 deficiency, a rare genetic metabolic disorder affecting glucose metabolism. The market encompasses therapies, diagnostics, and research initiatives aimed at improving patient outcomes, addressing unmet medical needs, and advancing innovative treatments for this life-impacting condition.

Industry Recent Developments: United States

✅ September 2025: AVTX-801, an oral formulation of D-galactose being developed for PGM1 deficiency treatment, entered late-stage clinical trials with an expedited regulatory pathway.​

✅ October 2025: Increased awareness and genetic testing initiatives have driven growth in the PGM1 deficiency market, supported by emerging therapies and diagnostics.​

✅ November 2025: Continued development of targeted therapies for rare genetic diseases, including PGM1 deficiency, with focus on improving efficacy and patient access.

Industry Recent Developments: Japan

✅ September 2025: Research efforts on rare diseases including PGM1 deficiency are ongoing, with Japan positioned as a key region for clinical trial activities.​

✅ October 2025: Government initiatives to accelerate rare disease drug approvals, including for treatments targeting PGM1 deficiency, reflecting a supportive regulatory environment.​

✅ November 2025: Expansion of patient support programs and increased focus on personalized medicine approaches for congenital disorders of glycosylation in Japan.

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FDA / Regulatory Approvals & Designations in 2025:

✅ Fast Track Designation: Cerecor (now or formerly) had a program CERC‐801, an ultra-pure D‐galactose formulation, which received Fast Track Designation from the FDA.

✅ Orphan Drug Designation & Rare Pediatric Disease Designation: For CERC‐801, Cerecor secured Orphan Drug Designation and Rare Pediatric Disease Designation, making it eligible for a Priority Review Voucher (PRV) upon approval.

✅ Gene Therapy Regulatory Potential: AAV-based therapy is in preclinical development (mouse model). The University of Utah's Technology Licensing Office describes an AAV9‐PGM1 gene therapy as a "potential first FDA approved treatment or cure."

Major Key Players:

Avalo Therapeutics, Inc.
Biosynth
BioSpectra, Inc.
Glentham Life Sciences Limited
ZellBio GmbH
Merck KGaA
Otto Chemie Pvt. Ltd.
SRL Chemical
Fengchen Group Co.,Ltd
Pfanstiehl

Segments Covered in the Phosphoglucomutase Deficiency Market:

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.

Regional Analysis for Phosphoglucomutase Deficiency Market:

⇥ North America (U.S., Canada, Mexico)

⇥ Europe (U.K., Italy, Germany, Russia, France, Spain, The Netherlands and Rest of Europe)

⇥ Asia-Pacific (India, Japan, China, South Korea, Australia, Indonesia Rest of Asia Pacific)

⇥ South America (Colombia, Brazil, Argentina, Rest of South America)

⇥ Middle East & Africa (Saudi Arabia, U.A.E., South Africa, Rest of Middle East & Africa)

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Chapter Outline:

⏩ Market Overview: It contains five chapters, as well as information about the research scope, major manufacturers covered, market segments, Phosphoglucomutase Deficiency market segments, study objectives, and years considered.

⏩ Market Landscape: The competition in the Global Phosphoglucomutase Deficiency Market is evaluated here in terms of value, turnover, revenues, and market share by organization, as well as market rate, competitive landscape, and recent developments, transaction, growth, sale, and market shares of top companies.

⏩ Companies Profiles: The global Phosphoglucomutase Deficiency market's leading players are studied based on sales, main products, gross profit margin, revenue, price, and growth production.

⏩ Market Outlook by Region: The report goes through gross margin, sales, income, supply, market share, CAGR, and market size by region in this segment. North America, Europe, Asia Pacific, Middle East & Africa, and South America are among the regions and countries studied in depth in this study.

⏩ Market Segments: It contains the deep research study which interprets how different end-user/application/type segments contribute to the Phosphoglucomutase Deficiency Market.

⏩ Market Forecast: Production Side: In this part of the report, the authors have focused on production and production value forecast, key producers forecast, and production and production value forecast by type.

⏩ Research Findings: This section of the report showcases the findings and analysis of the report.

⏩ Conclusion: This portion of the report is the last section of the report where the conclusion of the research study is provided.

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Frequently asked questions:

➠ What is the global sales value, production value, consumption value, import and export of Phosphoglucomutase Deficiency market?

➠ Who are the global key manufacturers of the Phosphoglucomutase Deficiency Industry? How is their operating situation (capacity, production, sales, price, cost, gross, and revenue)?

➠ What are the Phosphoglucomutase Deficiency market opportunities and threats faced by the vendors in the global Phosphoglucomutase Deficiency Industry?

➠ Which application/end-user or product type may seek incremental growth prospects? What is the market share of each type and application?

➠ What focused approach and constraints are holding the Phosphoglucomutase Deficiency market?

➠ What are the different sales, marketing, and distribution channels in the global industry?

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