Medical Foods for Orphan Disease Market Projected to Hit USD 14.65 Billion by 2034 at 5.2% CAGR: Towards Healthcare Report

The global medical foods for orphan disease market size is calculated at USD 9.3 billion in 2025 and is expected to reach around USD 14.65 billion by 2034, growing at a CAGR of 5.2% for the forecasted period.

Ottawa, Oct. 22, 2025 (GLOBE NEWSWIRE) -- The global medical foods for orphan disease market size was valued at USD 8.84 billion in 2024 and is predicted to hit around USD 14.65 billion by 2034, rising at a 5.2% CAGR, a study published by Towards Healthcare a sister firm of Precedence Research.

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Key Takeaways

The medical foods for orphan disease market will likely exceed USD 8.84 billion by 2024.
Valuation is projected to hit USD 14.65 billion by 2034.
Estimated to grow at a CAGR of 5.2% from 2025 to 2034.
North America accounted for the major share of the market in 2024.
Europe is expected to be the fastest-growing region during 2025-2034.
By disease type, the inborn errors of metabolism (IEM) segment led the market in 2024.
By disease type, the neurological & neurodevelopmental disorders segment is expected to witness rapid expansion in the coming years.
By product type, the amino acid-based formulas segment was dominant in the medical foods for orphan disease market in 2024.
By product type, the ketogenic medical foods segment is expected to grow at a rapid CAGR during the forecast period.
By route of administration, the oral segment held the largest share of the market in 2024.
By route of administration, the enteral segment is expected to grow rapidly in the upcoming years.
By age group, the pediatric patients segment registered dominance in the market in 2024.
By age group, the adult segment is expected to be the fastest-growing during 2025-2034.
By distribution channel, the hospitals & specialty clinics segment dominated the market in 2024.
By distribution channel, the home delivery & online pharmacies segment is expected to register the fastest growth in the predicted timeframe.

What are the Medical Foods for Orphan Diseases?

The global medical foods for orphan disease market mainly comprises diverse specialized foods for the dietary management of a concern, available through a physician's guidance. A rise in cases of rare diseases, demand for specialized, convenient, and easily administered products, including powders and those delivered through enteral feeding, is propelling the market expansion. Currently, the globe is increasingly using 3D printing for customized products, mobile apps for patient management, and joint ventures for the development of new formulas for specific issues, such as pediatric eosinophilic GI disorders.

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Key Metrics and Scope

Metric	Details
Market Size in 2025	USD 9.3 Billion
Projected Market Size in 2034	USD 14.65 Billion
CAGR (2025 - 2034)	5.2	%
Leading Region	North America Share by 47%
Market Segmentation	By Disease Type, By Product Type, By Route of Administration, By Age Group, By Distribution Channel
Top Key Players	Nestlé Health Science, Abbott Laboratories, Danone Nutricia, Mead Johnson Nutrition, Cambrooke Therapeutics, Vitaflo International Ltd, BioMarin Pharmaceutical Inc., MedDay Pharmaceuticals, Relmada Therapeutics, B. Braun Melsungen AG, Ajinomoto Cambrooke Inc., Targeted Medical Pharma, Inc., Mevalia, Nutricia Metabolics, Cymbiotika, Metagenics Inc., KetoCal (Nutricia), Vitafriends (distributor & service provider), Solace Nutrition, Nutricia North America

What are the Key Drivers in the Medical Foods for Orphan Disease Market?

Apart from the growing rare disease cases, there is a rise in newborn screening programs for earlier detection, & intervention is also impacting the market progress. Also, the global market is widely fueled by the increasing awareness and diagnosis of rare genetic issues and the transformation of precision nutritional compounds. The globe is bolstering home healthcare, and digital tools are fulfilling the raised demand for convenient, ready-to-use medical food products.

What are the Drifts in the Medical Foods for Orphan Disease Market?

In July 2025, ZOIA, a Pentec Company, acquired Lil’s Low Carb Inc., to boost its commitment to the rare metabolic disease community.
In April 2025, EveryONE Medicines and Mendelian partnered to revolutionize the UK landscape for rare disease diagnosis and personalized treatment.

What is the Evolving Limitation in the Market?

The medical foods for orphan disease market has a vital limitation in inconsistencies in insurance plans covering medical foods, as they are not classified as traditional drugs, resulting in major financial hurdles for patients. Alongside, the market is facing limitations in conducting clinical trials due to a lack of patient populations.

Regional Analysis

Why did North America Dominate the Market in 2024?

By capturing the biggest share, North America dominated the market share by 47% in 2024. The accelerating demand for specialized medical food for newborns and ongoing innovations in genetic research and personalized medicine are boosting the development of highly targeted therapies. Involvement of leading players, such as Cambrooke Therapeutics and Nestlé, is leveraging their medical foods with digital tools and apps for robust patient management.

For instance,

In May 2025, the U.S. Food and Drug Administration (FDA) and the National Institutes of Health (NIH) introduced a new joint nutrition research initiative.:

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How did Europe Grow Notably in the Market in 2024?

In the upcoming era, Europe is anticipated to witness rapid expansion in the medical foods for orphan disease market. The regional market will eventually be fueled by growing advances in diagnostics, which further support early and more precise detection of increasingly rare disease instances. Additionally, the European regulatory landscape fosters the support of the development of Foods for Special Medical Purposes (FSMP) for rare diseases. Diverse EU regulations exploring advances in food science that are developing more specific, bioavailable, and stable nutritional compounds personalized to individual patient needs.

Medical Foods for Orphan Disease Market: Notable Efforts in 2024-2025

Indian Government	Secured ₹974 crore for the fiscal year 2025-26 to the National Fund for Rare Diseases (NFRD).
European Union amendments	In 2025, new regulations governing foods for special medical purposes (FSMPs) include modifications to protein requirements and authorized ingredients.
NIH-Funded Paper	Described a trial from January 2025 on the effect of a ketogenic diet on metabolic dysfunction.

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Segmental Insights

By disease type analysis

Which Disease Type Led the Medical Foods for Orphan Disease Market in 2024?

The inborn errors of metabolism (IEM) segment held a major share of the market in 2024. The segment is primarily driven by the rising public and professional awareness of IEM and the significance of early intervention with medical foods. Alongside the accelerating newborn screening by employing techniques, especially mass spectrometry, the transformation of digital health tools for patient management with inexpensiveness is also impacting the comprehensive adoption. The emergence of digital resources, including Treatable ID and IEMbase, is offering clinicians comprehensive, up-to-date information,

Moreover, the neurological & neurodevelopmental disorders segment will expand rapidly in the coming era. Continuous research activities in these cases, led by major investment, are supporting the detection of innovative pathways for intervention and escalating recognition of the role of factors, such as the gut microbiome. In September 2025, the FDA approved leucovorin calcium tablets for cerebral folate deficiency, which is a neurological condition. Recent studies are exploring probiotics and prebiotics are potential to impact neurotransmitter balance and enhance mood and memory.

By product type analysis

Why did the Amino Acid-Based Formulas Segment Dominate the Market in 2024?

In 2024, the amino acid-based formulas segment captured the largest share of the medical foods for orphan disease market. A rise in severe cow's milk allergy (CMA), eosinophilic esophagitis, and food protein-induced enterocolitis syndrome (FPIES) conditions is widely fueling demand for these products. Recently, Abbott unveiled EleCare Jr with DHA & Lutein, an amino acid-based medical food for children aged one and older, which supports the management of severe food allergies, gastrointestinal conditions, and protein malabsorption.

On the other hand, the ketogenic medical foods segment is predicted to witness the fastest growth. In this era, it mainly encompasses supplements, such as medium-chain triglycerides (MCTs), ketone esters, and modified diets that provide more protein and carbohydrates, and expand adherence and lower side effects. Studies in 2025 have demonstrated that the ketogenic diet improves cognition, sociability, and overall neurological function and also unveiled new research avenues for autism spectrum disorder, addiction, and other psychiatric concerns.

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By route of administration analysis

What Made the Oral Segment Dominant in the Market in 2024?

The oral segment accounted for the dominating share of the medical foods for orphan disease market in 2024. The widespread advantages of this route include easy use, mainly for at-home management, chronic conditions and patients of all ages. The latest innovations in pharmaceutical approaches are employing nanoparticles, microspheres, and hydrogels for designing mucoadhesive (adhering to mucous membranes) oral delivery systems.

Whereas the enteral segment is estimated to witness rapid expansion during 2025-2034. The market is putting efforts into advances in genomics and metabolic analysis, which accelerate precision in personalizing enteral medical foods. The exploration of alternative proteins, plant-based products, and specialized peptides and amino acids, are being incorporated into formulas are also merged in this route. In 2025, the FDA strengthened the approval of the RELiZORB cartridge, a device that mimics natural lipase to hydrolyze fats in enteral formulas.

By age group analysis

Which Age Group Led the Medical Foods for Orphan Disease Market in 2024?

In 2024, the pediatric patients segment held the biggest share of the market. The segment is mainly driven by Advocacy groups, especially the National Organization for Rare Disorders (NORD), which expands awareness among clinicians and caregivers. The latest innovation comprises the launch of a hypoallergenic enteral formula for Food Protein-Induced Enterocolitis Syndrome (FPIES) approved for neonatal ICUs by Mead Johnson Nutrition.

Moreover, the adult segment will expand rapidly in the coming era. The growing instances of rare diseases and the diagnosis of the same conditions are bolstering demand for advanced medical foods. Ongoing groundbreakings in medical foods are also being created to eliminate inflammation or neutralize free radicals connected with chronic pain and autoimmune disorders, including fibromyalgia and rheumatoid arthritis.

By distribution channel analysis

How did the Hospitals & Specialty Clinics Segment Dominate the Market in 2024?

The hospitals & specialty clinics segment accounted for a dominant share of the medical foods for orphan disease market in 2024. The increasing requirement for specialized nutritional assistance under physician supervision, and the major role of these facilities in diagnosis, treatment, and complex therapies, is fueling the global expansion. Moreover, the accelerating demand for advanced therapies, like complex gene-therapy infusions, necessitates the specialized infrastructure that hospitals and specialty clinics offer.

Although the home delivery & online pharmacies segment will expand rapidly. The wider demand for online platforms and telemedicine services is boosting the development of tailored nutritional interventions. This further resolves geographical barriers and facilitates personalized nutritional support remotely. Alongside, online pharmacies in India are leveraging their services beyond general medicine to incorporate health foods and nutritional products. Also, the globe is shifting towards at-home care management and e-commerce services for diverse rare diseases.

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Transforming Developments in the Medical Foods for Orphan Disease Market

In October 2025, TRYNGOLZA (olezarsen) was approved by the U.S. Food and Drug Administration, used in reducing triglycerides in adults with familial chylomicronemia syndrome (FCS)..
In October 2025, GeneDx partnered with Florida’s Sunshine Genetics Network, unveiled the nation’s first state-backed genomic newborn screening program.
In February 2025, UCLA Health launched a study on the ketogenic diet for bipolar disorder in youth.

Medical Foods for Orphan Disease Market Key Players List

Nestlé Health Science
Abbott Laboratories
Danone Nutricia
Mead Johnson Nutrition
Cambrooke Therapeutics
Vitaflo International Ltd
BioMarin Pharmaceutical Inc.
MedDay Pharmaceuticals
Relmada Therapeutics
B. Braun Melsungen AG 
Ajinomoto Cambrooke Inc. 
Targeted Medical Pharma, Inc.
Mevalia
Nutricia Metabolics
Cymbiotika
Metagenics Inc. 
KetoCal (Nutricia)
Vitafriends (distributor & service provider) 
Solace Nutrition 
Nutricia North America

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Segments Covered in the Report

By Disease Type

Inborn Errors of Metabolism (IEM)
Phenylketonuria (PKU) 
Maple Syrup Urine Disease (MSUD)
Homocystinuria
Methylmalonic Acidemia
Mitochondrial Disorders
Neurological & Neurodevelopmental Disorders
Rett Syndrome 
Alzheimer’s
Autism Spectrum Disorders
Gastrointestinal Disorders
Cancer Cachexia

By Product Type

Amino Acid-Based Formulas
Phenylalanine-free, leucine/isoleucine/valine-free, etc.
Ketogenic Medical Foods
Fat-Modified Formulas
Carbohydrate-Controlled Medical Foods
Nucleotide/Nucleoside-Based Compounds 
Liquid/Ready-to-Drink Formulas
Powdered Formulas

By Route of Administration

Oral
Enteral
Parenteral

By Age Group

Pediatric Patients
Adults
Neonates/Infants

By Distribution Channel

Hospitals & Specialty Clinics 
Home Delivery & Online Pharmacies
Retail Pharmacies
Specialty Nutrition Distributors

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