Krabbe Disease Treatment Market is to grow at a 7.22 % CAGR, with the United States leading the market through 2032

The Krabbe Disease Treatment Market (BPO) will reach USD 5.03 billion by 2032. The Krabbe Disease Treatment Market is driven by increased investment in rare disease therapies and improved healthcare infrastructure.

Krabbe Disease Treatment Market to Hit USD 5.03 Bn by 2032  

The Krabbe Disease Treatment Market was USD 2.88 Bn in 2024 and is expected to expand at a CAGR of 7.22 % from 2024 to 2032, reaching USD 5.03 Bn by 2032. Forge Biologics has reported positive news about its FBX-101 clinical study, a gene treatment for the adeno-associated virus serotype rh10 (AAVrh10). FBX-101, which is administered intravenously after hematopoietic stem cell transplantation (HSCT), attempts to treat the peripheral and cerebral neuropathy linked to Krabbe disease. In February 2024, Minnesota began screening newborns for Krabbe disease. The U.S. Food and Drug Administration (FDA) has designated Passage Bio’s PBKR03 as an Orphan Drug and a Rare Pediatric illness for the treatment of Krabbe disease.  

To cure hereditary illnesses like Krabbe disease, gene-based medicines like enzyme replacement therapy (ERT) and stem cell-based therapies are showing encouraging promise. Organizations like the National Institute of Neurological Disorders and Stroke (NINDS) advocate for and educate people about Krabbe illness and its impact. Businesses and academic organizations are investigating a variety of therapeutic alternatives, including as cell-based therapeutics, gene editing technologies (such as CRISPR-Cas9), and small molecule medications. Agencies like the FDA and the European Medicines Agency (EMA) provide tax benefits, increased market exclusivity, and expedited approval procedures for orphan disease therapies. The availability of more treatments and better patient outcomes may result from the approval of ERTs designed especially for Krabbe disease. In 2024, the United States Krabbe disease treatment market is projected to hold a 27.8% share of the global market.

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Krabbe Disease Treatment Market Segmentation

By Disease Type: Infantile Krabbe Disease typically presents in the first 6 months of life and progresses rapidly, leading to severe neurological damage. Newer medicines like gene therapy and treatments like hematopoietic stem cell transplantation (HSCT) have shown promise in improving new outcomes. Late-onset Krabbe Disease is a less common kind that usually manifests after the age of two, however, symptoms may manifest in infancy or adulthood. Enzyme replacement therapy (ERT), symptomatic treatments, and genetic therapies may be used in conjunction to treat this type. 

By Treatment Insight: Anticonvulsants are frequently recommended to treat seizures, which are a typical sign of Krabbe illness, especially in young children and newborns with the infantile type. Muscle Relaxants are recommended to treat symptoms of infantile and late-onset Krabbe disease, including muscular rigidity and spasticity. Hematopoietic Stem Cell Transplantation (HSCT) is a well-established treatment for infantile Krabbe disease. A new therapeutic option for Krabbe disease is gene therapy.

Krabbe Disease Treatment Market Regional Analysis

United States: The Krabbe disease treatment market in the US is expected to grow at a CAGR of 9.77% from 2024 to 2032. A leading player, Passage Bio, Inc., in the US, focuses on creating gene treatments for uncommon genetic conditions that affect the central nervous system. Passage Bio is developing PBKR03, a gene therapy medicine intended to treat Krabbe disease. To finance its clinical studies for Krabbe disease and other uncommon neurological illnesses, the business raised about $234 million during its first public offering (IPO).

Europe: The market for Krabbe disease in Europe was estimated to be worth $0.25 billion in 2023 and is expected to grow at a CAGR of 8.5% from 2024 to 2032, reaching $0.45 billion by 2032. HSCT is a key therapy strategy used in Europe, aiming to halt disease progression if administered early. Forge Biologics, Novartis, Pfizer Inc., Sanofi S.A., GlaxoSmithKline Plc (GSK), and Biogen Inc. are the key players that dominate the European Krabbe disease treatment market.

China: The Chinese Krabbe disease market is expected to reach USD 6.82 billion by 2032, with a CAGR of 8.2% from 2024 to 2032. ​WuXi AppTec is a prominent global pharmaceutical, biotechnology, and medical device company in China. Its Net profit surpassed RMB 10 billion, demonstrating excellent operational success. WuXi supported the FDA’s February 2024 approval of the BLA application for the first-ever TIL-based therapy.

Krabbe Disease Treatment Market Competitive Landscape

Forge Biologics: In November 2023, Forge Biologics was acquired by Ajinomoto Co., Inc., a world leader in the manufacturing of food and amino acids, for approximately $620 million. In Columbus, Ohio, Forge Biologics runs a 200,000-square-foot facility called “the Hearth” that employs more than 300 staff members.

Genzyme (Sanofi): Genzyme was acquired by Sanofi-Aventis, a French pharmaceutical business, for approximately $20.1 billion. Sanofi-Aventis offered $74.00 per Genzyme share in cash. Novazyme was acquired by Genzyme for $137.5 million.

Biogen Inc.: In July 2024, Biogen acquired HI-Bio to broaden its immunology offering for approximately $1.8 billion, encompassing prospective milestones as well as a $1.15 billion initial payment. In January 2025, in an unsolicited purchase attempt, Biogen offered $7.22 per share, which was 30% more than Sage Therapeutics’ closing price.

Recent Developments:

• Forge Biologics is a well-known gene therapy business that is developing therapies for uncommon illnesses, including Krabbe disease. ” They are developing FBX-101, a gene therapy treatment that aims to deliver the GALC gene and correct the genetic defect in patients with Krabbe disease.
• HSCT has gained interest, particularly when used early in the course of the illness. Stem cell transplantation is used in this treatment to stop or delay the course of the illness. Its effectiveness and safety have increased due to advancements in transplant procedures.

Conclusion

• Important advancements in gene treatments have shown promise in early-stage clinical studies, especially those that deliver the defective GALC gene using adeno-associated viral (AAV) vectors.
• The goal of enzyme replacement treatments is to reduce symptoms by making up for the loss of essential enzymes, but HSCT is still a good choice for early intervention.

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