Global Maple Syrup Urine Disease Treatment Market - 2022-2029

Global Maple Syrup Urine Disease Treatment Market - 2022-2029

Market Overview

The global maple syrup urine disease treatment market size was valued at US$ XX million in 2021 and is estimated to reach US$ XX million by 2029, growing at a CAGR of XX % during the forecast period (2022-2029).

MSUD is a life-threatening metabolic disorder caused by maple syrup. Metabolic disorders are diseases in which the body cannot function normally because it is unable to convert food into energy in a healthy manner.

Market Dynamics

The factors influencing the global maple syrup urine disease treatment market are the rise in demand for disease-specific treatment and the increasing prevalence of maple syrup urine disease.

The rise in demand for disease-specific treatment is expected to drive the market growth in the forecast period

MSUD is a rare genetic disorder marked by a lack of an enzyme complex (branched-chain alpha-keto acid dehydrogenase) needed to break down (metabolize) the three branched-chain amino acids (BCAAs) leucine, isoleucine, and valine in the body. As a result of this metabolic failure, all three BCAAs and a number of their toxic byproducts (specifically, their respective organic acids) build up abnormally. Within a few hours of birth, plasma BCAA concentrations begin to rise in the classic, severe form of MSUD. If left untreated, symptoms appear within the first 24-48 hours of life. There are three main components to treating classic, intermediate, intermittent, and thiamine-responsive MSUD: Ongoing dietary therapy; ongoing maintenance of normal metabolic conditions, including BCAA levels in the body; ongoing medical intervention for metabolic crises. MSUD patients must follow a protein-restricted diet that restricts the amount of branched-chain amino acids they can consume. To promote proper growth and development, protein restriction should begin as soon as possible after birth. Synthetic (artificial) formulas provide all of the nutrients required for proper growth and development but lack leucine, isoleucine, and valine.

The side effects associated with treatment are expected to hamper the market growth

Changes in blood sugar levels are one of the side effects of IV glucose and insulin. Doctors will keep a close eye on these levels in the hospital to make sure they stay within the normal range. Muscle cramps, low blood pressure (hypotension), and changes in blood electrolyte contents are all side effects of hemodialysis and hemofiltration that must be closely monitored. Individuals with classic MSUD have had great success with liver transplants. Complications of a liver transplant are similar to those of any surgical procedure, including bleeding, infection, and blood clots. Other potential complications of transplantation include organ rejection and a weakened immune system (beyond what is needed to prevent organ rejection). Patients can live normal lives with good monitoring and without fear of MSUD problems if they better understand the transplant process.

COVID-19 Impact Analysis

MSUD patients typically live into adulthood if diagnosed and treated early with a BCAA-restricted diet to prevent muscle protein catabolism. Despite the adequate dietary modification, a metabolic crisis in children and adults can be triggered by insults such as infection, surgery, trauma, and fasting. Encephalopathy, gastrointestinal disturbances, and lethargy are all symptoms of a metabolic crisis in these people.

According to the Centers for Disease Control and Prevention (CDC), the coronavirus disease 2019 (COVID-19) pandemic has infected nearly 80 million Americans and killed over 900,000, posing a serious threat to MSUD patients prone to metabolic derangements. Hence, covid-19 is expected to impact the global maple syrup urine disease treatment market.

Segment Analysis

The classic maple syrup urine disease segment is expected to dominate the market growth

Classic maple syrup urine disease (classic MSUD) is the most severe and probably most common form of MSUD, characterized by a maple syrup odor in the cerumen at birth, poor feeding, lethargy, and focal dystonia, which can progress to progressive encephalopathy and central respiratory failure if left untreated. In neonates, classic MSUD is a medical emergency. Acute management necessitates glucose plus insulin, intravenous lipids, plasma amino acid monitoring, and isoleucine and valine supplements to boost protein anabolism. Dialysis is frequently required. Stabilized infants require high-calorie BCAA-free formulas, dietary leucine restriction, and outpatient metabolic clinic monitoring. Patients must follow a strict diet for the rest of their lives and be closely monitored during pregnancy. The use of orthotopic liver transplantation is also a viable option. Within the first 24–48 hours, infants with classic MSUD will show subtle symptoms. Poor feeding, whether by bottle or breast, lethargy, and irritability are subtle symptoms. The infant's focal neurologic signs will then become more prominent. Athetoid, hypertonia, spasticity, and opisthotonus are some of the neurologic signs that can lead to convulsions and coma. If MSUD is not treated, it will cause central neurologic dysfunction and respiratory failure, which will result in death. Although MSUD can be stabilized, there is still a risk of metabolic decompensation and bone loss, resulting in osteoporosis, pancreatitis, and intracranial hypertension. Other signs and symptoms associated with classic MSUD include intellectual disability and behavioral issues.

Geographical Analysis

North America region is expected to hold the largest market share in the global maple syrup urine disease treatment market

The increasing prevalence of maple syrup urine disease and better healthcare facilities in this region is expected to drive the market growth.

MSUD is a very uncommon condition. It affects about 1 in every 185,000 births around the world. When cousins and other close relatives have children together, it is more common in populations with a small gene pool. MSUD affects about 2,000 people in the United States. It affects both men and women equally. It affects approximately one newborn out of every 180,000 live births in the United States. However, in populations where consanguinity is more common, such as the Mennonites in Pennsylvania or the Amish, MSUD is significantly higher, with one newborn out of every 176 live births.

Moreover, on 1st February 2022, Relief Therapeutics Holding SA collaborated with Acer Therapeutics and was granted a new patent by the United States Patent and Trademark Office (USPTO) for certain claims related to ACER-001 (sodium phenylbutyrate). The FDA is currently reviewing Acer Therapeutics' New Drug Application (NDA) for ACER-001 to treat UCDs, with a target action date of June 5, 2022, under the Prescription Drug User Fee Act (PDUFA).

Competitive Landscape

The global maple syrup urine disease treatment market is moderately competitive with mergers, acquisitions, and product launches. Some of the key players in the market are Acer Therapeutics Inc., HemoShear Therapeutics, LLC, Recordati SpA, Jo Mar, Codexis, Inc.

Acer Therapeutics Inc.

Overview: Acer Therapeutics Inc. is a pharmaceutical company focused on acquiring, developing, and commercializing Intermediate MSUD for rare and life-threatening diseases with significant unmet medical needs.

Product Portfolio: ACER-001 (sodium phenylbutyrate) is a nitrogen-binding agent in development to treat inborn metabolic errors such as UCDs and MSUDs. MSUD is a rare inherited disorder caused by defects in the mitochondrial branched-chain ketoacid dehydrogenase complex, resulting in elevated blood levels of the branched-chain amino acids (BCAA), leucine, valine, and isoleucine, as well as the associated branched-chain ketoacids (BCKA).

Key Development: On 1st February 2022, Relief Therapeutics Holding SA collaborated with Acer Therapeutics and has been granted a new patent by the United States Patent and Trademark Office (USPTO) for certain claims related to ACER-001 (sodium phenylbutyrate). The FDA is currently reviewing Acer Therapeutics' New Drug Application (NDA) for ACER-001 to treat UCDs, with a target action date of June 5, 2022, under the Prescription Drug User Fee Act (PDUFA).

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