Krabbe Disease Treatment Global Market Report 2025

The krabbe disease treatment market size has grown strongly in recent years. It will grow from $2.31 billion in 2024 to $2.47 billion in 2025 at a compound annual growth rate (CAGR) of 6.7%. The growth during the historic period can be attributed to the rising demand for rare disease treatments, increased research efforts in enzyme replacement therapy, expanded government funding for rare disease management, the proliferation of newborn screening programs, and strengthening patient advocacy initiatives.

The krabbe disease treatment market size is expected to see strong growth in the next few years. It will grow to $3.16 billion in 2029 at a compound annual growth rate (CAGR) of 6.4%. The growth in the forecast period can be attributed to increasing research in gene therapy, rising investments in orphan drug development, enhanced regulatory incentives for rare diseases, expanding clinical trial activities, and growing healthcare expenditure. Major trends include the wider adoption of gene and enzyme replacement therapies, a focus on personalized treatment approaches, advancements in stem cell transplantation, integration of artificial intelligence in diagnosis and therapy development, and improvements in diagnostic technologies.

The growing implementation of newborn screening programs is anticipated to boost the expansion of the krabbe disease treatment market in the future. These programs are health initiatives that test infants shortly after birth for potentially serious yet treatable genetic, metabolic, and developmental disorders. Advances in medical technology are contributing to the rise of newborn screening by allowing earlier, more accurate detection and timely prevention of critical health problems. Newborn screening facilitates the early identification of krabbe disease, enabling swift interventions before symptoms appear. This early diagnosis supports treatments like stem cell transplantation, which enhances patient outcomes and slows disease progression. For example, in November 2023, the Michigan Department of Health and Human Services, a US state agency, reported that by 2022, over 7.7 million infants had been screened, with more than 8,000 diagnosed through the NBS blood spot panel. Specifically, out of 100,176 infants screened in 2022, mostly Michigan residents, 322 (0.3%) were diagnosed with a condition. Hence, the rising prevalence of newborn screening programs is fueling growth in the krabbe disease treatment market.

Key companies in the krabbe disease treatment market are concentrating on developing advanced gene therapy programs to improve treatment efficacy and expand global availability. Gene therapy involves altering or replacing genes within a patient’s cells to treat or prevent illnesses. These therapies seek to correct genetic defects by introducing, removing, or modifying specific genes, offering potential cures for genetic disorders. For example, in March 2024, Forge Biologics, a US-based gene therapy firm, launched FBX-101, a novel adeno-associated viral (AAV) gene therapy. This treatment received the Innovation Passport designation from the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA), allowing it to enter the Innovative Licensing and Access Pathway (ILAP). ILAP aims to expedite regulatory approval and market access for therapies targeting severe or life-threatening diseases. FBX-101 has shown promising safety and effectiveness in early clinical trials and functions by delivering a functional GALC gene to patients. It is designed to enhance clinical outcomes for children suffering from this rare and fatal neurodegenerative disorder.

In November 2023, Ajinomoto Co., Inc., a biotechnology and food company based in Japan, acquired Forge Biologics Holdings, LLC for $554 million. This acquisition allows Ajinomoto to expand its footprint in the gene therapy sector and enhance its biopharma services business. It supports Ajinomoto’s ASV Initiatives 2030 Roadmap, which aims to position healthcare as a key growth area by integrating advanced gene therapy development and manufacturing capabilities. Forge Biologics Holdings LLC is a US-based company specializing in gene therapy for krabbe disease.

Major players in the krabbe disease treatment market are Labcorp Holdings Inc., Ajinomoto Co. Inc., Fulgent Genetics, Mayo Clinic Laboratories, Myriad Genetics Inc., Passage Bio Inc., PreventionGenetics, Greenwood Genetic Center, Magenta Therapeutics Inc., Gain Therapeutics Inc., Cells4Life Group LLP, DNA Labs India, M6P Therapeutics, MediciNova Inc., Neurogene Inc., Polaryx Therapeutics Inc., Vascumab LLC, APTEEUS, Agilus Diagnostics, and Medicover Genetics.

North America was the largest region in the krabbe disease treatment market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in krabbe disease treatment report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the krabbe disease treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Krabbe disease treatment involves medical interventions aimed at managing symptoms and slowing the progression of this rare, often fatal genetic disorder affecting the nervous system. Key treatment options include hematopoietic stem cell transplantation (HSCT), which is most effective when performed early, alongside supportive care such as physical therapy, seizure control, nutritional support, and pain management.

The main types of treatment comprise medications, enzyme replacement therapy, gene therapy, and stem cell therapy. Medications like anticonvulsants and muscle relaxants help manage symptoms such as seizures and muscle stiffness to improve patients’ quality of life. Diagnosis is commonly performed through newborn screening and genetic testing. Treatments are distributed via retail pharmacies, e-commerce platforms, and hospital pharmacies. Krabbe disease treatments apply to various forms, including infantile, late-onset, and adult-onset, and are utilized by hospitals, clinics, research centers, and laboratories.

The krabbe disease treatment market research report is one of a series of new reports that provides krabbe disease treatment market statistics, including the krabbe disease treatment industry global market size, regional shares, competitors with the krabbe disease treatment market share, detailed krabbe disease treatment market segments, market trends, opportunities, and any further data you may need to thrive in the krabbe disease treatment industry. This krabbe disease treatment market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.

The krabbe disease treatment market consists of revenues earned by entities providing services such as genetic counseling, enzyme activity testing, hematopoietic stem cell transplantation (HSCT), and supportive care management. The market value includes the value of related goods sold by the service provider or included within the service offering. The krabbe disease treatment market also includes sales of viral vectors, infusion equipment, diagnostic kits, and neurological monitoring systems. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD unless otherwise specified).

The revenues for a specified geography are consumption values and are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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