Methylmalonic Acidemia Market Growth Anticipated by 2032 | Major Players: LogicBioTherapeutics, HemoShearTherapeutics, VectivBio, are expected to boost the Methylmalonic Acidemia Market

In the market landscape of Methylmalonic Acidemia, an impressive surge is expected during the study period spanning 2019 to 2032, according to latest report titled  “Methylmalonic Acidemia Market Insights, Epidemiology and Market Forecast 2032” from DelveInsight. 

Driving Forces Behind the Methylmalonic Acidemia Market Growth

The methylmalonic acidemia market dynamics are projected to undergo significant changes in the forthcoming years. Factors contributing to this transformation include the approval of Carbaglu and the rising incidence of MMA. The anticipated introduction of emerging therapies like LB-001 (based on LogicBio’s GeneRide technology) and HST5040 (developed by HemoShear Therapeutics) is poised to stimulate market growth within the forecasted period [2023-2032]. The MMA pipeline represents a promising prospect for improved treatment approaches, heralding a positive outlook for the market in the years ahead.

The Methylmalonic Acidemia market report sheds light on Methylmalonic Acidemia current treatment practices, upcoming drugs in the Methylmalonic Acidemia pipeline, market shares of individual therapies, and the anticipated trajectory of the Methylmalonic Acidemia market size from 2019 to 2032 across the 7MM (the United States, the EU-4 comprising Italy, Spain, France, and Germany, the United Kingdom, and Japan).

Discover the Anticipated Evolution and Growth of the Market @ Methylmalonic Acidemia Therapeutics Market Forecast

Therapeutic Advancements and Emerging Treatments:

• Methylmalonic Acidemia Clinical Trial Progression: The market is set to experience significant growth, driven by the progression of emerging therapies expected for launch between 2023 and 2032. Pioneering companies, including LogicBioTherapeutics, HemoShearTherapeutics, VectivBio, and others, are actively engaged in developing novel drugs for potential market entry.

• Methylmalonic Acidemia Innovative Therapies: Ongoing research and development activities are fostering the introduction of innovative therapies designed to address the signs and symptoms of Methylmalonic Acidemia. Therapies such as LB-001, HST5040, VB1197, and others are driving the Methylmalonic Acidemia market.

Methylmalonic Acidemia Market Dynamics

Anticipated shifts are expected to occur within the MMA market in the upcoming years. Key industry players like HemoShear (HST5040) and LogicBio Therapeutics (LB-001) are actively engaged in developing therapies for Methylmalonic Acidemia.

LB-001, an investigational pediatric genome editing therapy, utilizes LogicBio’s GeneRide technology, facilitating site-specific integration and lifelong expression of therapeutic transgenes without the use of exogenous promoters or nucleases. The drug aims to integrate a functional version of the faulty MUT gene into the patient's genome. It has earned orphan drug designation from the US FDA for treating patients with methylmalonic acidemia.

The FDA granted rare pediatric disease designation for MMA treatment in July 2019. An update on screening and enrollment activities for the SUNRISE Phase I/II clinical trial of LB-001 was announced by the company in April 2021. This investigational treatment for MMA is based on LogicBio's proprietary gene insertion platform, GeneRide, with the first patient for the Phase I/II SUNRISE trial anticipated to be enrolled by mid-2021. The company also forged a strategic collaboration with CANbridge Pharmaceuticals and a research partnership with Daiichi Sankyo.

HST5040, developed by HemoShear, is an investigational oral small molecule therapy designed to rectify metabolic abnormalities linked with MMA and PA. Its ability to distribute to multiple affected tissues suggests potential activity throughout the body, including the brain, heart, and muscles. This therapy is formulated for convenient daily administration at home as a liquid, taken orally or through a gastric feeding tube. HemoShear Therapeutics received clearance from the US FDA in January 2020 for an Investigational New Drug (IND) application to conduct a phase II clinical trial of HST5040 for treating patients with MMA and PA.

In March 2021, HemoShear Therapeutics, Inc. initiated the HERO (HElp Reduce Organic acids) study, a Phase II clinical trial exploring HST5040 for the treatment of MMA and PA in patients aged 2 and older.

Methylmalonic Acidemia Treatment Market

There exists a critical need for developing and validating biomarkers and surrogate endpoints in clinical trials related to propionic acidemia (PA) and methylmalonic acidemia (MMA). Potential biomarkers in individuals affected by either PA or MMA include secondary metabolites like ammonium or the mitochondrial disease marker fibroblast growth factor 21.

Further investigation is necessary to validate these biomarkers as surrogate endpoints. It's also essential to explore whether other metabolites or markers indicating organ damage could serve as useful biomarkers in clinical trials evaluating investigational drug treatments for patients with PA or MMA.

To manage bladder pain, bladder instillations involving anesthetic solutions are employed. Heparin agents are believed to aid in the repair of the damaged urothelium. As per the American Urological Association's practice guideline for Methylmalonic Acidemia, bladder instillations are considered a secondary treatment option when adjustments in diet, stress management, and over-the-counter remedies fail to provide sufficient relief. Available instillation types in the market include Alkalinized Lidocaine and Heparin, DMSO, Sodium Hyaluronate, and BCG.

Leading Methylmalonic Acidemia Companies and Emerging Drugs: Pioneering companies such as LogicBioTherapeutics, HemoShearTherapeutics, VectivBio, among others, are actively developing novel drugs for potential entry into the Methylmalonic Acidemia market.

Methylmalonic Acidemia Therapeutic Landscape: Key therapies identified for Methylmalonic Acidemia treatment include LB-001, HST5040, VB1197, and more.

Methylmalonic Acidemia Overview:

Methylmalonic acidemia encompasses a cluster of hereditary conditions where the body struggles to properly process specific proteins and lipids (fats). Individuals with this condition are unable to metabolize methymalonyl-coenzyme A, resulting in an accumulation of methylmalonic acid in their system. Besides genetic causes, deficiencies in vitamin B12, unrelated to genetic origins, can also contribute to the buildup of methylmalonic acid.

Several gene mutations can cause methylmalonic acidemia. Those affected can be categorized into two groups: 1) individuals with isolated MMA, wherein only methylmalonic acid levels are elevated, and 2) those with combined defects who exhibit elevated levels of both methylmalonic acid and homocysteine. Mutations in the MMAA, MMAB, and MUT genes are responsible for isolated methylmalonic acidemia.

This condition presents in two primary forms: isolated methylmalonic acidurias and combined methylmalonic aciduria and homocystinuria, each resulting from distinct gene mutations. Alongside these types, there exist five less common subtypes, including cblD, cblF, cblJ, and cblX deficiencies.

Typically emerging within the first few months of life, though occasionally manifesting in late childhood, symptoms of Methylmalonic Acidemias may involve lethargy, failure to thrive, recurrent vomiting, acidosis, dehydration, respiratory distress, reduced muscle tone, developmental delays, seizures, and/or an enlarged liver.

Diagnosis of Methylmalonic Acidemias can often occur before birth through prenatal measures like analyzing methylmalonic acid concentration in amniotic fluid or assessing deficient enzyme activity in fluid or tissue samples obtained during pregnancy via procedures like amniocentesis or chorionic villus sampling (CVS). Expanded newborn screening with tandem mass spectrometry can also detect the disorder at birth.

Currently, there isn't a specific cure for methylmalonic acidemia. Treatment mainly focuses on symptom management, which involves aggressive management during decompensation events, a specialized diet managed for protein intake, vitamin B12 supplementation for responsive subtypes, medications like carnitine, avoidance of stressors triggering decompensation events (such as fasting or illness), and, in some severe cases, liver and kidney transplants.

Key Facts Methylmalonic Acidemia Market Report:

• Key players such as LogicBioTherapeutics, HemoShearTherapeutics, VectivBio, and others are investigating its candidates for Methylmalonic Acidemia.

• Methylmalonic Acidemia pipeline includes the major therapies such as LB-001, HST5040, VB1197, and others. 

Methylmalonic Acidemia Epidemiology Segmentation:

“According to DelveInsight’s analysis, both genders are affected equally, in case of Methylmalonic Acidemia (MMA).”

• As per the research conducted by An et al. (2017) in their paper titled "Systemic Messenger RNA Therapy for the Treatment of Methylmalonic Acidemia," isolated methylmalonic acidemia/aciduria (MMA) stands as a severe metabolic disorder, demonstrating poor outcomes despite current medical interventions. This condition affects an estimated 1 in 50,000–100,000 individuals.

• In the United States, Therrell et al. (2014), in their study titled "Inborn Errors of Metabolism Identified via Newborn Screening: Ten-Year Incidence Data and Costs of Nutritional Interventions for Research Agenda Planning," presented aggregated data from newborn screening programs covering infants born between 2001 and 2010. Their research revealed the identification of 158 cases of MMA mutations, equating to a detection rate of 0.63 per 100,000 newborns.

To know more in detail, download our report @ https://www.delveinsight.com/report-store/methylmalonic-acidemia-market