Maple Syrup Urine Disease - Pipeline Insight, 2024

This “Maple Syrup Urine Disease- Pipeline Insight, 2024” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Maple Syrup Urine Disease 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.

Geography Covered

• Global coverage

Maple Syrup Urine Disease: Understanding

Maple Syrup Urine Disease: Overview

Maple syrup urine disease (MSUD) is a rare genetic metabolic disorder called an inborn error of metabolism (IEM). For people with MSUD, the body does not have enough of a properly working enzyme needed to break down certain amino acids (building blocks of protein).This enzyme is called the branched chain alpha ketoacid dehydrogenase (BCKD) complex. MSUD is caused by mutations in the genes involved in making the BCKD complex, which breaks down the amino acids leucine, isoleucine, and valine (also called branched-chain amino acids, BCAA). These amino acids are found in protein-rich foods. The amino acids and their by-products build up in the bodies of people with MSUD, and can result in damage to the brain and other organs. MSUD is inherited in an autosomal recessive pattern, which means that both parents have one defective copy of the gene, which is passed on to the affected child. MSUD occurs in approximately 1 in 185,000 infants, but is more common in Old Order Mennonites, where it has a frequency of 1 in 380. It is equally likely to affect males and females. MSUD is sometimes subdivided into 4 types: classic, intermediate, intermittent and thiamine-responsive. Diagnosis of MSUD is based on symptoms and biochemical testing of blood and urine. It is confirmed with molecular genetic testing for mutations in the genes that encode BCKD. Tests may also be done to measure enzyme activity but this testing is becoming less readily available. MSUD can also be detected using newborn screening, which is now being performed in the U.S. and other developed countries. There are two main aspects to managing all types of MSUD: chronic treatment with a special diet and acute treatment of a metabolic crisis (where the stress of fasting, dehydration, and/or a serious illness triggers severe symptoms) when it occurs.

“Maple Syrup Urine Disease- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Maple Syrup Urine Disease pipeline landscape is provided which includes the disease overview and Maple Syrup Urine Disease treatment guidelines. The assessment part of the report embraces, in depth Maple Syrup Urine Disease 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, Maple Syrup Urine Disease 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 Maple Syrup Urine Disease R&D. The therapies under development are focused on novel approaches to treat/improve Maple Syrup Urine Disease.

Maple Syrup Urine Disease Emerging Drugs Chapters

This segment of the Maple Syrup Urine Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.

Maple Syrup Urine Disease Emerging Drugs

ACER 001: Acer Therapeutics ACER-001 is a taste-masked, immediate-release formulation of sodium phenylbutyrate (Na PB) developed using a microencapsulation process. Treatment with NaPB is currently being studied as a treatment for people with maple syrup urine disease (MSUD). NaPB is approved for people with urea cycle disorders (UCDs) to control their ammonia levels in conjunction with a restricted diet. ACER-001 was granted orphan drug designation as a potential treatment for MSUD in2014.

Maple Syrup Urine Disease: Therapeutic Assessment

This segment of the report provides insights about the different Maple Syrup Urine Disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Maple Syrup Urine Disease

There are approx. 5+ key companies which are developing the therapies for Maple Syrup Urine Disease. The companies which have their Maple Syrup Urine Disease drug candidates in the most advanced stage, i.e. phase II include, Acer Therapeutics.

Phases

This report covers around 5+ 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

Maple Syrup Urine Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

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

Maple Syrup Urine Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Maple Syrup Urine Disease 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 Maple Syrup Urine Disease drugs.

Maple Syrup Urine Disease Report Insights

• Maple Syrup Urine Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Maple Syrup Urine Disease 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 Maple Syrup Urine Disease drugs?
• How many Maple Syrup Urine Disease drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Maple Syrup Urine Disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Maple Syrup Urine Disease 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 Maple Syrup Urine Disease and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Acer Therapeutics
• ASC Therapeutics
• APR Applied Pharma Research
• LyGenesis

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Key Products

• ACER 001
• Gene therapy
• APR OM33
• REC 0545