Propionic Acidemia Market

Key Highlights

• The rise in Propionic Acidemia cases can be attributed to improved diagnostic capabilities, expanded newborn screening programs, and a better understanding of the genetic basis of the disease. 
• The leading companies involved in the management of Propionic Acidemia are focusing on providing medical interventions and supportive care to patients.
• The market for Propionic Acidemia is expected to grow steadily, with a consistent CAGR during the forecast period (2024-2034) due to the increasing cases of Propionic Acidemia and better healthcare spending around the world. 
• Emerging therapies hold promise for driving future growth in the Propionic Acidemia treatment market. Several emerging treatments, including mRNA-3927 (Moderna), and others are under investigation for their potential to prevent Propionic Acidemia.

DelveInsight’s comprehensive report titled “Propionic Acidemia— Market Insights, Epidemiology, and Market Forecast – 2034” offers a detailed analysis of Propionic Acidemia. The report presents historical and projected epidemiological data covering total prevalent cases of Propionic Acidemia, total diagnosed prevalent cases of Propionic Acidemia, severity-specific cases of Propionic Acidemia and treated cases of Propionic Acidemia. 

In addition to epidemiology, the market report encompasses various aspects related to the patient population. These aspects include the diagnosis process, prescription patterns, physician perspectives, market accessibility, treatment options, and prospective developments in the market across seven major markets: the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan, spanning from 2020 to 2034.

The report analyzes the existing treatment practices and unmet medical requirements in Propionic Acidemia . It evaluates the market potential and identifies potential business prospects for enhancing therapies or interventions. This valuable information enables stakeholders to make well-informed decisions regarding product development and strategic planning for the market.

Propionic AcidemiaOverview

Propionic Acidemia is a rare metabolic disorder characterized by deficiency of propionyl-CoA carboxylase, an enzyme involved in the breakdown (catabolism) of the chemical “building blocks” (amino acids) of proteins. Symptoms most commonly become apparent during the first weeks of life and may include abnormally diminished muscle tone (hypotonia), poor feeding, vomiting, restlessness (lethargy), dehydration and seizures. Without appropriate treatment, episodes of vomiting, lethargy, dehydration, and accumulation of excessive levels of acids in the blood and bodily tissues (acidosis) may lead to coma and death.

Propionic Acidemia Diagnosis and Treatment Algorithm 

Propionic Acidemia can be identified at birth through expanded newborn screening by measuring levels of certain metabolites such a propionylcarnitine and methionine in a blood sample. Most infants are diagnosed in the first weeks of life based upon a thorough clinical evaluation, a detailed patient and family history, and molecular genetic testing. It may be diagnosed before birth (prenatally) by screening fetal DNA for disease-causing (pathogenic) mutations in the PCCA and PCCB genes.

During acute episodes, the treatment of infants with propionic acidemia may require fluid therapy; measures to provide appropriate nutritional intake (e.g., intravenous glucose, with and without intravenous lipids); administration of certain medications to prevent or treat bacterial infection; and other supportive measures as required. In infants with severe disease (e.g., severe acidosis, hyperammonemia), treatment may require procedures that remove excess waste products from the blood (hemodialysis). 

Propionic Acidemia Epidemiology

The epidemiology section on the Propionic Acidemia market report offers information on the patient populations, including historical and projected trends for each of the seven major markets. Examining key opinion leader views from physicians or clinical experts can assist in identifying the reasons behind historical and projected trends. The prevalence patient pool, their trends, and the underlying assumptions are all included in this section of the report.

This section also presents the data with relevant tables and graphs, offering a clear and concise view of the prevalence of Propionic Acidemia. Additionally, the report discloses the assumptions made during the analysis, ensuring data interpretation and presentation transparency. This epidemiological data is valuable for understanding the disease burden and its impact on the patient population across various regions.

Key Findings

As per the study, the cases of Propionic Acidemia vary widely. The estimated live-birth cases are 1:105,000–1:130,000 in the US, 1:166,000 in Italy, and 1:250,000 in Germany. In Japan, the live birth cases of severe Propionic Acidemia is 1:465,000 but increases to 1:17,400 when combined with an apparently asymptomatic form of identified through newborn screening.

In North America, detection rates of Propionic Acidemia were 0.20 (US, California) per 100,000 newborns. The pooled point estimate indicated a detection rate of 0.33 per 100,000 newborns in North America.

In Europe, the detection rates varied between 0.32 and 2.20 per 100,000 newborns. The pooled point estimate indicated a rate of 0.33 per 100,000 newborns in Europe. Screening programs with 100% population coverage were identified in Italy and Spain, where detection rates were 1.25 (Italy) and 0.95 (Spain) per 100,000 newborns.

Based on the research studies, the prevalence rate of Propionic Acidemia is probably about 1 in 100,000 live births worldwide.

According to a study newborn screening programs provided relatively precise estimates on the birth prevalence of Propionic Acidemia . The overall detection rates of Propionic Acidemia remained under 8.14 cases per 100,000 newborns in all geographic regions. Studies conducted in North America and Europe reported birth prevalence rates lower than 2.2 cases per 100,000 newborns.

Propionic Acidemia Market Outlook

In recent times, there has been a notable increase in interest in propionate biology and understanding the mechanisms responsible for organ dysfunction in Propionic Acidemia. Despite the current limited management options for Propionic Acidemia, which primarily involve strict dietary regimens, carnitine supplementation, and the use of antibiotics like metronidazole to decrease propionate production by intestinal bacteria, there is ongoing exploration into more effective approaches.

Moreover, the utilization of ammonia scavengers such as carglumic acid to manage hyperammonemia episodes has been a significant aspect of Propionic Acidemia treatment. Notably, the approval by the US FDA of CARBAGLU, developed by Recordati, for a new indication to address acute hyperammonemia linked with Propionic Acidemia and Methylmalonic Acidemia represents a substantial breakthrough in tackling the fundamental cause of Propionic Acidemia, for which approved therapies were previously lacking.

The expansion of the Propionic Acidemia market is being driven by various factors, including the high prevalence of the condition, heightened awareness, and the growing demand for improved treatment modalities. These advancements signal a promising trend in the management and comprehension of Propionic Acidemia, particularly in the last decade.

Propionic Acidemia Drug Chapters 

Marketed Propionic Acidemia Drugs

There are no approved therapies that address the underlying root cause of Propionic Acidemia. Current management of the disorder is limited to strict dietary management, carnitine supplementation, antibiotics such as metronidazole to reduce propionate production by intestinal bacteria, and ammonia scavengers such as carglumic acid to control episodes of hyperammonemia.

Note: Detailed marketed therapies assessment will be provided in the final report.

Emerging Propionic Acidemia Drugs

In recent years, the drug market for propionic acidemia is emerging, with several promising investigational treatments in development. Some of the drugs in the pipeline include mRNA-3927 (Moderna), BBP-671 (CoA Therapeutics), and others.

mRNA-3927: Moderna

Moderna is developing mRNA-3927. It act as Methylmalonyl-CoA decarboxylase expression stimulants. It is a combination therapy encoding for PCCA and PCCB protein to form an active PCC enzyme. The drug has been granted Fast Track designation from US FDA in October 2019. The drug is currently in phase II clinical development.

Note: Detailed emerging therapies assessment will be provided in the final report.

Propionic Acidemia Market Segmentation

DelveInsight’s ‘Propionic Acidemia Market Insights, Epidemiology, and Market Forecast – 2034’ report provides a detailed outlook of the current and future Propionic Acidemia market, segmented within countries, by therapies, and by classes. Further, the market of each region is then segmented by each therapy to provide a detailed view of the current and future market share of all therapies. 

Propionic Acidemia Market Size by Countries

The Propionic Acidemia market size is assessed separately for various countries, including the United States, EU4 (Germany, France, Italy, and Spain), the UK, and Japan. In 2023, the United States held a significant share of the overall 7MM (Seven Major Markets) Propionic Acidemia market, primarily attributed to the country's higher prevalence of the condition and the elevated cost of the available treatments. This dominance is projected to persist, especially with the potential early introduction of new products.

Propionic Acidemia Market Size by Therapies

Propionic Acidemia Market Size by Therapies is categorized into current and emerging markets for the study period 2020–2034. One of the emerging drugs anticipated to launch during the forecast period is mRNA-3927 under the developmental pipeline of Moderna. 

Note: Detailed market segment assessment will be provided in the final report.

Propionic Acidemia Drugs Uptake

This section focuses on the sales uptake of potential Propionic Acidemia drugs that have recently been launched or are anticipated to be launched in the Propionic Acidemia market between 2020 and 2034. It estimates the market penetration of Propionic Acidemia drugs for a given country, examining their impact within and across classes and segments. It also touches upon the financial and regulatory decisions contributing to the probability of success (PoS) of the drugs in the Propionic Acidemia market.

The emerging Propionic Acidemia therapies are analyzed based on various attributes such as safety and efficacy in randomized clinical trials, order of entry and other market dynamics, and the unmet need they fulfill in the Propionic Acidemia market .

Note: Detailed assessment of drug uptake and attribute analysis will be provided in the full report on Propionic Acidemia .

Propionic Acidemia Market Access and Reimbursement

DelveInsight’s ‘Propionic Acidemia – Market Insights, Epidemiology, and Market Forecast – 2034’ report provides a descriptive overview of the market access and reimbursement scenario of Propionic Acidemia .

This section includes a detailed analysis of the country-wise healthcare system for each therapy, enlightening the market access, reimbursement policies, and health technology assessments. 

KOL Views

To keep up with current Propionic Acidemia market trends and fill gaps in secondary findings, we interview KOLs and SMEs’ working in the Propionic Acidemia domain. Their opinion helps understand and validate current and emerging therapies and treatment patterns or Propionic Acidemia market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the Propionic Acidemia unmet needs.

Propionic Acidemia : KOL Insights

DelveInsight’s analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. These KOLs were from organizations, institutes, and hospitals such as National Human Genome Research Institute, Maryland in the US, UCSF Department of Pediatrics in the US, University of Washington School of Medicine in the US,  among others.

“Even after advancement in modern sciences, so far now there is no approved therapy to PA. There is development going on to bring new therapies”

“Timely diagnosis of PA remains a challenge due to its rarity and the variability of symptoms, leading to delayed intervention and potential complications. Improving newborn screening programs and increasing awareness among healthcare professionals are crucial to ensure early detection and intervention”

“While dietary management is the mainstay of treatment for PA, it may not be sufficient to prevent all complications or fully normalize metabolic function. There is a need for novel therapies that address the underlying metabolic dysfunction and reduce the accumulation of toxic metabolites more effectively.”

Note: Detailed assessment of KOL Views will be provided in the full report on Propionic Acidemia .

Competitive Intelligence Analysis

We conduct a Competitive and Market Intelligence analysis of the Propionic Acidemia Market, utilizing various Competitive Intelligence tools such as SWOT analysis and Market entry strategies. The inclusion of these analyses is contingent upon data availability, ensuring a comprehensive and well-informed assessment of the market landscape and competitive dynamics.

Propionic Acidemia Pipeline Development Activities

The report offers an analysis of therapeutic candidates in Phase II and III stages and examines companies involved in developing targeted therapeutics for Propionic Acidemia . It provides valuable insights into the advancements and progress of potential treatments in clinical development for this condition.

Pipeline Development Activities

The report covers information on collaborations, acquisition and merger, licensing, patent details, and other information for emerging Propionic Acidemia therapies.

Propionic Acidemia Report Insights

• Propionic Acidemia Patient Population
• Therapeutic Approaches
• Propionic Acidemia Pipeline Analysis
• Propionic Acidemia Market Size and Trends
• Propionic Acidemia Market Opportunities
• Impact of Upcoming Therapies

Propionic Acidemia Report Key Strengths

• 11 Years Forecast
• The 7MM Coverage
• Propionic Acidemia Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Propionic Acidemia Market
• Propionic Acidemia Drugs Uptake

Propionic Acidemia Report Assessment

• Propionic Acidemia Current Treatment Practices
• Unmet Needs
• Propionic Acidemia Pipeline Product Profiles
• Propionic Acidemia Market Attractiveness

Key Questions

• How common is Propionic Acidemia ?
• What are the key findings of Propionic Acidemia epidemiology across the 7MM, and which country will have the highest number of patients during the study period (2020–2034)?
• What are the currently available treatments for Propionic Acidemia ?
• What is the disease risk, burden, and unmet needs of Propionic Acidemia ?
• At what CAGR is the Propionic Acidemia market and its epidemiology expected to grow in the 7MM during the forecast period (2024–2034)?
• How would the unmet needs impact the Propionic Acidemia market dynamics and subsequently influence the analysis of the related trends?
• What would be the forecasted patient pool of Propionic Acidemia in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the UK, and Japan?
• Among EU4 and the UK, which country will have the highest number of patients during the forecast period (2020–2034)?
• How many companies are currently developing therapies for the treatment of Propionic Acidemia ?