Propionic Acidemia - Pipeline Insight, 2025

This “Propionic Acidemia - Pipeline Insight, 2025” report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in Propionic Acidemia pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Propionic Acidemia: Understanding

Propionic Acidemia: Overview

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, listlessness (lethargy), dehydration and seizures. Without appropriate treatment, coma and death may result. Rarely, the condition may become apparent later in life and may be associated with less severe symptoms and findings. Propionic acidemia is inherited in an autosomal recessive pattern. Individuals with this condition have to follow a specific diet including a low protein intake and specific food formulas (medical foods).

Symptoms most commonly develop during the first weeks of life and may include vomiting, listlessness (lethargy), low muscle tone (hypotonia), failure to grow and gain weight at the expected rate (failure to thrive), and dehydration. Approximately 30% of affected infants may also develop seizures. The recurrence or worsening of symptoms may be associated with an infection, constipation, or consumption of high amounts of protein. In some affected infants, episodes of symptoms may alternate with periods of apparently normal health and development. This disorder typically manifests with episodic decompensations characterized by symptoms like dehydration, lethargy, nausea, vomiting, and a risk for neurologic sequelae. Neuropathologic findings in propionic acidemia include white matter spongiosis in neonates and a variable appearance in older children. Additionally, a case report described diffuse gray matter vacuolization in a 4-year-old girl postmortem, which is a previously unreported finding in propionic acidemia but has been observed in other fatty acid disorders.

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 with propionic acidemia are diagnosed in the first weeks of life based upon a thorough clinical evaluation, a detailed patient and family history, and molecular genetic testing.

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). Long-term treatment includes administration of a low-protein diet, possibly in combination with medical formula (medical foods) that are low in certain amino acids (i.e., amino acids which give rise to propionate, e.g., isoleucine, valine, threonine, and methionine). Liver transplant is a potential surgical option for individuals with severe symptoms and frequent recurrent acute episodes (decompensation).

'Propionic Acidemia- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Propionic Acidemia pipeline landscape is provided which includes the disease overview and Propionic Acidemia treatment guidelines. The assessment part of the report embraces, in depth Propionic Acidemia commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Propionic Acidemia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Propionic Acidemia R&D. The therapies under development are focused on novel approaches to treat/improve Propionic Acidemia.

Propionic Acidemia Emerging Drugs Chapters

This segment of the Propionic Acidemia report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Propionic Acidemia Emerging Drugs

mRNA-3927: Moderna

mRNA-3927 is designed to instruct the body to restore the missing or dysfunctional proteins that cause PA. mRNA-3927 contains two mRNAs that encode for the alpha and beta subunits of the mitochondrial enzyme propionyl-CoA carboxylase (PCC), encapsulated within Moderna’s proprietary lipid nanoparticle (LNP). mRNA-3927 is intended to treat patients with PA regardless of whether they are missing the alpha or beta subunits. mRNA-3927 uses the same proprietary LNP formulation used in the Company’s antibody against chikungunya virus (mRNA-1944) and MMA (mRNA-3704) programs. In addition to Fast Track designation, mRNA-3927 has also been granted Orphan Drug and Rare Pediatric Disease designations from the FDA and Orphan Designation by the European Medicines Agency (EMA). Currently, the drug is in Phase II stage of its clinical trial.

Propionic Acidemia: Therapeutic Assessment

This segment of the report provides insights about the different Propionic Acidemia drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Propionic Acidemia

• There are approx. 4+ key companies which are developing the therapies for Propionic Acidemia. The companies which have their Propionic Acidemia drug candidates in the most advanced stage, i.e. Phase II include, Moderna.

Phases

The report covers around 4+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Propionic Acidemia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Propionic Acidemia: Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Propionic Acidemia therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Propionic Acidemia drugs.

Propionic Acidemia Report Insights

• Propionic Acidemia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Propionic Acidemia Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Propionic Acidemia drugs?
• How many Propionic Acidemia drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Propionic Acidemia?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Propionic Acidemia therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Propionic Acidemia and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Moderna Therapeutics
• Bridge Bio Pharma
• Agios Pharmaceuticals

Key Products

• mRNA 3927
• BBP 671
• BCAT2 inhibitors

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