Propionic Acidemia Market Report 2026

The propionic acidemia market size has grown steadily in recent years. It will grow from $2.07 billion in 2025 to $2.15 billion in 2026 at a compound annual growth rate (CAGR) of 4.1%. The growth in the historic period can be attributed to improvements in newborn screening programs, increased awareness of inherited metabolic disorders, availability of supportive drug therapies, expansion of specialized metabolic clinics, growth in rare disease research funding.

The propionic acidemia market size is expected to see steady growth in the next few years. It will grow to $2.53 billion in 2030 at a compound annual growth rate (CAGR) of 4.1%. The growth in the forecast period can be attributed to increasing development of gene and enzyme therapies, rising investments in orphan drug development, expansion of precision medicine approaches, growing adoption of telemedicine for chronic care, increasing collaboration between research institutions. Major trends in the forecast period include increasing focus on early diagnosis and screening, rising adoption of personalized treatment protocols, growing research in rare metabolic disorders, expansion of specialized drug therapies, enhanced patient monitoring and management.

The increasing prevalence of genetic disorders is expected to propel the growth of the propionic acidemia market going forward. Genetic disorders are conditions caused by mutations or abnormalities in an individual’s DNA that may be inherited or arise spontaneously, affecting normal growth, metabolism, and overall health. The rising prevalence of genetic disorders is partly attributed to consanguineous marriages, which increase the likelihood of inheriting recessive genetic mutations when both parents carry the same defective gene. This significantly raises the risk of rare metabolic conditions such as propionic acidemia. Treatments for propionic acidemia support genetic disorder management by addressing metabolic imbalances caused by enzyme deficiencies, helping reduce disease complications and improve patient outcomes. For instance, in February 2024, according to Osservatorio Malattie Rare (OMAR), an Italy-based independent journalistic agency, in 2023 Italian medical centers identified 18 new disease-causing genes and followed more than 18,000 patients with rare diseases through regional healthcare networks. Therefore, the increasing prevalence of genetic disorders is driving the growth of the propionic acidemia market.

Leading companies in the propionic acidemia market are focusing on the development of innovative mRNA-based treatments to directly target the genetic causes of the disease. mRNA therapies function by using messenger RNA to instruct cells to produce proteins that can prevent or treat diseases at the genetic level. For example, in May 2023, Moderna, a US-based pharmaceutical and biotechnology company, shared interim data from the Phase 1/2 trial of mRNA-3927, an investigational mRNA therapy for propionic acidemia. This therapy delivers functional copies of the PCCA and PCCB genes via mRNA, restoring enzyme activity in the liver and potentially reducing metabolic decompensations. This cutting-edge approach aims to enhance patient outcomes by addressing the genetic root cause of the disease, offering new hope for those affected by this rare metabolic disorder.

In May 2023, Ironwood Pharmaceuticals Inc., a US-based biotechnology company, acquired VectivBio for approximately $1 billion. Through this acquisition, Ironwood aims to strengthen its pipeline for rare diseases by integrating VectivBio’s innovative therapies, including investigational treatments for propionic acidemia. VectivBio, a Switzerland-based biotechnology company, specializes in developing treatments for rare gastrointestinal and metabolic disorders.

Major companies operating in the propionic acidemia market report are Recordati Rare Diseases Inc, Horizon Therapeutics plc, Travere Therapeutics (Retrophin Inc), Mitsubishi Tanabe Pharma Corporation, Ultragenyx Pharmaceutical Inc, Moderna Inc, Krystal Biotech Inc, Astellas Pharma Inc, Synlogic Inc, Encoded Therapeutics Inc, BridgeBio Pharma Inc, VectivBio AG, Agios Pharmaceuticals Inc, CoA Therapeutics Inc, Imbria Pharmaceuticals Inc, Comet Therapeutics Inc, Swedish Orphan Biovitrum AB (Sobi), Regenacy Pharmaceuticals, Audentes Therapeutics, Vertex Pharmaceuticals.

North America was the largest region in the propionic acidemia market in 2025. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the propionic acidemia market report are Asia-Pacific, South East Asia, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the propionic acidemia market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Taiwan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The propionic acidemia market includes revenues earned by entities through enzyme replacement therapy (ERT), nutritional therapy services, personalized care management services, patient support programs, and pharmacogenomic services. The market value includes the value of related goods sold by the service provider or included within the service offering. Only goods and services traded between entities or sold to end consumers are included.

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 that 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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