Global Orphan Drugs Market to Grow at a CAGR of 6.98% Through 2032, Backed by Rising Rare Disease Awareness and Regulatory Incentives | DelveInsight

The global orphan drugs market is poised for substantial growth through 2032, fueled by increasing awareness of rare diseases, growing demand for novel treatments, and supportive regulatory frameworks such as orphan drug designations and market exclusivity incentives. These drugs, specifically developed for rare diseases affecting a small percentage of the population, are gaining attention as governments, healthcare organizations, and pharmaceutical companies prioritize unmet clinical needs. Additionally, technological advancements in genomics, improved diagnostic capabilities, and patient advocacy efforts are accelerating the development pipeline of orphan therapies.

DelveInsight's Orphan Drugs Market Insights report offers a comprehensive analysis of current and projected market trends, key players, strategic collaborations, and breakthrough drug developments. It also provides insight into evolving regulatory landscapes, challenges in pricing and reimbursement, and competitive dynamics shaping the market outlook through 2032.

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Key Takeaways from the Orphan Drugs Market Report
• The global orphan drugs market was valued at USD 208.61 billion in 2024 and is expected to grow at a CAGR of 6.98%, reaching USD 357.26 billion by 2032.
• As per DelveInsight estimates, North America is anticipated to dominate the global orphan drugs market during the forecast period.
• Notable Orphan Drugs companies such as Sanofi, Biogen, Ionis Pharmaceuticals, Inc., Novartis AG, Alnylam Pharmaceuticals, Inc., F. Hoffmann-La Roche Ltd., AstraZeneca, Gilead Sciences, Inc., Johnson & Johnson Services, Inc., Mitsubishi Tanabe Pharma Corporation, BioMarin Pharmaceutical Inc., Vertex Pharmaceuticals Incorporated, Ultragenyx Pharmaceutical Inc., Kyowa Kirin Co., Ltd., Catalyst Pharmaceuticals, Inc., Chugai Pharmaceutical Co., Ltd., Sarepta Therapeutics, Inc., Astellas Pharma Inc., Pfizer Inc., Novo Nordisk A/S, and several others are currently operating in the Orphan Drugs market.
• In June 2025, Oncovita announced that the FDA granted Orphan Drug Designation to its lead investigational therapy, MVdeltaC, for the treatment of pleural mesothelioma, a rare and aggressive cancer with limited treatment options.
• In May 2025, Beam Therapeutics (Nasdaq: BEAM) announced the FDA granted orphan drug designation to BEAM-302, a liver-targeted lipid nanoparticle therapy using base editing to correct mutations in alpha-1 antitrypsin deficiency (AATD) patients.
• In May 2025, RELIEF THERAPEUTICS Holding SA announced that the FDA granted Rare Pediatric Disease designation to RLF-TD011 for treating epidermolysis bullosa (EB), a rare genetic skin disorder. The drug had already received Orphan Drug Designation for this indication.
• In May 2025, Mesoblast (ASX: MSB; Nasdaq: MESO) announced that the FDA granted seven years of orphan drug exclusivity for Ryoncil® (remestemcel-L) to treat steroid-refractory acute graft-versus-host disease (SR-aGvHD) in pediatric patients aged 2 months and older.
• In May 2025, Thermosome announced that the FDA granted Orphan Drug Designation to its lead compound THE001 for treating soft tissue sarcomas (STS), complementing an existing EMA designation and highlighting its clinical potential.
• In May 2025, Infinant Health announced that the FDA granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to its investigational drug candidate, INF108, for the prevention of necrotizing enterocolitis in preterm infants.
• In April 2025, Esperion (NASDAQ: ESPR) announced it reached an agreement with the FDA to begin Phase III trials of bempedoic acid-both as monotherapy and in combination with ezetimibe-for pediatric patients with heterozygous and homozygous familial hypercholesterolemia (HeFH and HoFH). The trials are expected to launch this year, and bempedoic acid holds orphan drug designation for HoFH.
• In April 2025, NS Pharma announced that the FDA granted Orphan Drug Designation to NS-229, a selective JAK1 inhibitor being developed for eosinophilic granulomatosis with polyangiitis (EGPA). The designation supports NS-229's potential in regulating immune cell function to prevent tissue damage in this rare disease.
• In January 2025, Johnson & Johnson announced that its Nipocalimab Biologics License Application (BLA) received Priority Review designation from the FDA for the treatment of antibody-positive generalized myasthenia gravis (gMG) patients. The drug had previously been granted Orphan Drug Designation by both the FDA and European Medicines Agency (EMA).
• In January 2025, NMD Pharma A/S revealed that the FDA has granted Orphan Drug Designation (ODD) to NMD670, a novel oral small-molecule inhibitor targeting the skeletal muscle-specific chloride ion channel ClC-1, for the treatment of Charcot-Marie-Tooth disease (CMT).
• In January 2025, Elicera Therapeutics AB (publ), a clinical-stage cell and gene therapy company, announced that its drug candidate ELC-100 has received Orphan Drug Designation from the FDA for the treatment of pancreatic neuroendocrine tumors. This designation provides substantial regulatory benefits during the continued development and potential marketing approval of the treatment.
• In January 2025, KalVista Pharmaceuticals, Inc. reported that Japan's Ministry of Health, Labour and Welfare (MHLW) granted Sebetralstat Orphan Drug Designation for the on-demand treatment of hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and older.

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Orphan Drugs Overview
Orphan drugs are pharmaceutical agents developed specifically to treat, prevent, or diagnose rare diseases, also known as orphan or rare conditions, which typically affect a small percentage of the population. Defined in the U.S. by the Orphan Drug Act as affecting fewer than 200,000 people, these diseases often lack effective treatments due to limited commercial incentives for drug developers. To address this, regulatory authorities such as the FDA and European Medicines Agency (EMA) offer various incentives, including tax credits, market exclusivity, reduced fees, and funding support, to encourage innovation in this field.

Orphan drugs have transformed the therapeutic landscape for conditions such as cystic fibrosis, Duchenne muscular dystrophy, and certain rare cancers, significantly improving patient outcomes. With growing awareness, advances in genomics, and an increasing number of drug designations, the orphan drug market is witnessing steady growth and investment, positioning itself as a vital segment within the global pharmaceutical industry.

Orphan Drugs Market Insights
North America is expected to remain the leading region in the orphan drugs market in 2024, fueled by the rising prevalence of rare and genetic diseases, strong government support, and an active pharmaceutical pipeline. Policies like the U.S. Orphan Drug Act of 1983, offering incentives such as tax credits and market exclusivity, continue to drive innovation. The region also benefits from robust R&D activity, advancements in biotechnology, and frequent new product launches.

According to the CDC (2024), around 35,000 individuals in the U.S. live with cystic fibrosis, while the Rare Disease Day Organization estimates that 30 million Americans are affected by rare diseases, 80% of which have a genetic basis. Rare disorders such as SMA, Batten disease, hemophilia, and DMD highlight the urgent need for novel therapies. Similarly, Canada reports that Batten disease affects between 1 and 35 births per 500,000.

The growing demand for targeted therapies has prompted increased investment from key players. For instance, Rigel Pharmaceuticals received FDA Orphan Drug Designation (ODD) in January 2025 for R289 to treat myelodysplastic syndromes, while Tempest Therapeutics secured ODD for amezalpat in hepatocellular carcinoma. These efforts reflect North America's continued commitment to expanding treatment options for underserved rare disease populations.

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Orphan Drugs Market Dynamics
According to the World Economic Forum (2023), around 400-475 million people globally are affected by rare diseases, impacting one in 20 individuals during their lifetime. In the European Union alone, over 36 million people were living with rare conditions in 2023 (EU, 2024). These often severe and under-treated diseases are fueling strong demand for targeted therapies.

Regulatory bodies like the FDA and EMA have introduced incentives such as market exclusivity, tax credits, and fast-track designations through policies like the Orphan Drug Act, making rare disease drug development increasingly attractive to pharmaceutical companies. As a result, investment and innovation in this space are on the rise.

In the UK, about 1 in 2,000 people live with bleeding disorders like hemophilia and von Willebrand disease, requiring advanced and often expensive treatments. Similarly, spinal muscular atrophy (SMA) affects 10,000 to 25,000 people in the U.S., with 1 in 6,000 to 10,000 children born with the condition each year. Recent breakthroughs in gene therapies and ASOs have positioned SMA as a major focus in the orphan drug sector.

New drug approvals further underscore market growth. In October 2023, BioMarin's VOXZOGO® received FDA approval for expanding use in pediatric patients with achondroplasia, with orphan drug designation for hypochondroplasia.

Despite strong momentum, challenges like high drug costs, limited patient pools, and complex clinical development remain barriers to broader market expansion.

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Coverage: Global
Study Period: 2022 to 2032
Orphan Drugs Market CAGR: ~6.98%
Key Orphan Drugs Companies: Sanofi, Biogen, Ionis Pharmaceuticals, Inc., Novartis AG, Alnylam Pharmaceuticals, Inc., F. Hoffmann-La Roche Ltd., AstraZeneca, Gilead Sciences, Inc., Johnson & Johnson Services, Inc., Mitsubishi Tanabe Pharma Corporation, BioMarin Pharmaceutical Inc., Vertex Pharmaceuticals Incorporated, Ultragenyx Pharmaceutical Inc., Kyowa Kirin Co., Ltd., Catalyst Pharmaceuticals, Inc., Chugai Pharmaceutical Co., Ltd., Sarepta Therapeutics, Inc., Astellas Pharma Inc., Pfizer Inc., Novo Nordisk A/S, and others.

Orphan Drugs Market Segmentation
Market Segmentation By Drug Type: Small Molecule and Biologics.
Market Segmentation By Route of Administration: Oral and Parenteral.
Market Segmentation By Indication: Oncology, Hematology, Ophthalmology, Neurology, and others.
Market Segmentation By Distribution Channel: Hospital and Retail Pharmacies, and Online Pharmacies
Market Segmentation By Geography: North America, Europe, Asia-Pacific, and Rest of the World.

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Table of Contents
1. Orphan Drugs Market Report Introduction
2. Orphan Drugs Market Executive Summary
3. Competitive Landscape
4. Regulatory Analysis
5. Orphan Drugs Market Key Factors Analysis
6. Orphan Drugs Market Porter's Five Forces Analysis
7. Orphan Drugs Market Layout
8. Orphan Drugs Market Company and Product Profiles
9. KOL Views
10. Project Approach
11. About DelveInsight
12. Disclaimer & Contact Us

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This release was published on openPR.