Global Propionic Acidemia Drugs Market -2023-2030

Overview

Propionic acidemia is an inherited disorder in which the body is unable to process certain parts of proteins and lipids (fats) properly. It is classified as an organic acid disorder, which is a condition that leads to an abnormal buildup of particular acids known as organic acids.

Propionic acidemia is caused by a deficiency of propionyl-CoA carboxylase (PCC), the enzyme that catalyzes the conversion of propionyl-CoA to methyl malonyl-CoA. Newborns with PA tested by expanded newborn screening have elevated C3 (propionylcarnitine). Testing of urine organic acids in persons who are symptomatic or those detected by newborn screening reveals elevated 3-hydroxypropionate and the presence of methylcitrate, tiglylglycine, propionylglycine, and lactic acid.

Market Dynamics: Drivers and Restraints

Rise in the prevalence of propionic acidemia

The rise in the prevalence of propionic acidemia is one of the significant factors that helps the market to grow during the forecast period. For instance, according to the article published in NCBI Propionic acidemia affects about 1 in 100,000 people in the United States.

The condition appears to be more common in several populations worldwide, including the inuit population of Greenland, some amish communities, and saudi arabians. Furthermore, the rise in the advancements in treatment options, the rise in the prevalence of ketotic hyperglycinemia and others will also drive the market growth during the forecast period.

Market Dynamics: Restraint

One of the restraint factors of the propionic acidemia market is it is highly complex to enroll and conduct a clinical trial of a new drug treatment for propionic acidemia, due to the rarity, heterogeneity, mortality, and unpredictable outcomes associated with these inborn errors of metabolism. Selecting clinical endpoints for these trials is particularly challenging. Thus, there is a clear need to identify which biomarkers for propionic acidemia could be surrogate endpoints to support the investigation of new therapies for these rare diseases.

Segment Analysis

The global propionic acidemia drugs is segmented based on drug class, route of administration distribution channel and region.

The L-carnitine from the drug class segment accounted for approximately 40.2 % of the market share

The L-carnitine from the drug class segment accounted for 40.2% and it is expected to be dominated during the forecast period. L-carnitine is a chemical that is made in the human brain, liver, and kidneys. It helps the body turn fat into energy. L-carnitine is important for heart and brain function, muscle movement, and many other body processes. The body can convert L-carnitine to other chemicals called acetyl-L-carnitine and propionyl-L-carnitine

Treatment with L-carnitine greatly enhanced the formation and excretion of short-chain acylcarnitines in three patients with propionic acidemia and in three normal controls. The use of fast atom bombardment mass spectrometry and linked scanning at constant magnetic (B) to electric ? field ratio identified acylcarnitine as propionylcarnitine in patients with propionic acidemia. The normal children excreted mostly acetylcarnitine.

Geographical Analysis

North America accounted for approximately 38.4% of the market share in 2022

North America is estimated to hold about 38.4% of the total market share throughout the forecast period, owing, to the improved healthcare infrastructure, FDA approvals, rise in the prevalence of propionic acidemia, collaborations and acquisitions with the key players and others expected to dominate by this region during the forecast period.

For instance, in May 2022, the U.S. Food and Drug Administration (FDA) granted the National Institutes of Health (NIH)'s National Center for Advancing Translational Sciences (NCATS) an orphan drug designation for AAV9-hPCCA (NCATS-BL0746), investigational gene therapy for the treatment of propionic acidemia resulting from a deficiency of Propionyl-CoA Carboxylase, alpha subunit (PCCA).

COVID-19 Impact Analysis

COVID-19 had a substantial impact on the propionic acidemia market where evidence suggests that patients with propionic acidemia who contract COVID-19 may be at risk of metabolic decompensation, but the clinical course of the infection is generally mild and recovery is possible without severe clinical consequences.

Another case report published in NCBI it describes those two patients with underlying metabolic disorders, including propionic acidemia, who contracted COVID-19. The report suggests that individuals with an underlying inborn error of metabolism (IEM) are at risk of metabolic decompensation in the setting of a COVID-19 infection. However, the virus does not appear to cause a more severe metabolic deterioration than is typical.

Competitive Landscape

The major global players in the propionic acidemia drugs market include: Leadiant, Pfizer, ScotMedcare, Cambridge Healthcare, Teva Pharmaceuticals, Wellona Pharma, Merck, AdvaCare Pharma, serve pharmaceuticals, Vitane Pharmaceuticals, Inc and among others.

Key Developments

• In May 2023, Moderna, Inc, a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, stated that an abstract reporting on interim data from the Phase I/II trial of mRNA-3927, an investigational mRNA therapy for propionic acidemia (PA) has been accepted for presentation at the 2023 American Society of Gene + Cell Therapy (ASGCT) Annual Meeting being held May 16-20, 2023 in Los Angeles, CA.
• In August 2022, BridgeBio Pharma stated the Dosing of the First Patient in the Phase I Trial of BBP-671, a Potential best-in-class treatment for propionic acidemia (PA) and methylmalonic acidemia (MMA)BBP-671 is an investigational oral therapy intended to increase CoA levels by allosterically modulating pantothenate kinases, key enzymes in the CoA biosynthesis pathway.
• In January 2021, Food and Drug Administration (FDA) approved a new indication for Carbaglu for use as adjunctive therapy in the treatment of acute hyperammonemia due to propionic acidemia (PA).

Why Purchase the Report?

• To visualize the global propionic acidemia drugs market segmentation-based drug class, route of administration, distribution channel and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development
• Excel data sheet with numerous data points of propionic acidemia drugs market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global propionic acidemia drugs market report would provide approximately 69 tables, 70 figures, and 185 Pages.

Target Audience 2023

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies