Propionic Acidemia Market Report 2025

The propionic acidemia market size has grown steadily in recent years. It will grow from $2.0 billion in 2024 to $2.09 billion in 2025 at a compound annual growth rate (CAGR) of 4.8%. The growth during the historic period can be attributed to factors such as the increasing prevalence of the disease, expansion of newborn screening programs, rising healthcare expenditures, a higher rate of pediatric diagnoses, and the advancement of rare disease initiatives.

The propionic acidemia market size is expected to see steady growth in the next few years. It will grow to $2.49 billion in 2029 at a compound annual growth rate (CAGR) of 4.4%. The projected growth during the forecast period can be attributed to factors such as expanding awareness campaigns, a rising number of metabolic disorder cases, increased adoption of telemedicine, growing demand for personalized treatments, and increased healthcare investments. Key trends expected in this period include technological advancements in diagnostics, the development of gene therapies, innovations in metabolic treatments, the integration of AI in healthcare, and the creation of novel therapeutics.

The increasing prevalence of genetic disorders is expected to drive the growth of the propionic acidemia market. Genetic disorders are conditions caused by mutations or abnormalities in an individual’s DNA, which may be inherited or occur spontaneously, impacting health and development. One of the key factors contributing to the rise in genetic disorders is consanguineous marriages, which increase the likelihood of inheriting recessive genetic mutations. This occurs when defective genes are passed down from both parents, significantly raising the risk of genetic conditions. Propionic acidemia plays a crucial role in managing genetic disorders by providing targeted treatments that address metabolic imbalances and improve patient outcomes. For example, in July 2022, a report published by the Cystic Fibrosis (CF) Foundation, a US-based non-profit organization, indicated that the prevalence of cystic fibrosis had increased, affecting nearly 40,000 children and adults in the United States in 2022, with over 105,000 individuals diagnosed globally. Consequently, the growing prevalence of genetic disorders is fueling the expansion 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 players in the propionic acidemia market are Moderna Therapeutics, Synlogic Therapeutics, Invitae Corporation, Global Calcium, BridgeBio Pharma, AdvaCare Pharma, HemoShear Therapeutics, DNA Labs, Velnex Medicare, Phytologix Life Scieneces, Liaoning Koncepnutra Co. Ltd., Propionic Acidemia Foundation, Agex Pharma, Gladfem, CSL Behring, Octapharma AG, Sanquin, Kedrion SpA, Shire plc, Vitane Pharmaceuticals Inc.

North America was the largest region in the propionic acidemia market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in propionic acidemia report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the propionic acidemia market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Propionic acidemia (PA) is a rare inherited metabolic disorder caused by a deficiency of the enzyme propionyl-CoA carboxylase, which is crucial for breaking down specific amino acids and fatty acids. This deficiency results in the accumulation of toxic compounds in the blood, leading to severe symptoms such as metabolic crises and neurological damage. If left untreated, the condition can be life-threatening, particularly during infancy.

The primary drug classes used for treating propionic acidemia include L-carnitine and antibiotics. L-Carnitine is a naturally occurring compound that plays a vital role in transporting long-chain fatty acids into mitochondria, where they undergo oxidation to generate energy. Treatment can be administered through various routes, including oral, intravenous, and intramuscular methods. These medications are distributed through multiple channels, such as hospital pharmacies, retail pharmacies, and online pharmacies.

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

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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