Isovaleric Acidemia Treatment Market

Isovaleric Acidemia Treatment Market Snapshot (2023 to 2033)

The global isovaleric acidemia treatment market is expected to reach a valuation of US$ 652.28 Million in 2023 and is projected to reach US$ 1,125.15 by 2033, trailing at a CAGR of 5.6%. Presently, genetic screening using mass spectrometry and DNA testing among all newborns is carried out in almost all of the developed nations of the world and also in developing economies of India, Brazil, and China. This helps uncover inherited metabolic disorders (IMD) at an early stage which is driving the market growth.

However, among many adult populations and in some children, rare genetic metabolic disorders are abruptly presented and often not accurately diagnosed. In such individuals, abnormal metabolic changes are considered to be a type of genetic mutation in routine diagnosis. The treatment of IMDs is broadly classified into dietary restrictions, dietary supplementation, drugs that inhibit or regulate metabolism, transplantation of the concerned organ, gene therapy, and dialysis in severe cases. In the case of familial chylomicronemia, gene therapy includes Alipogene tiparvovec recombinant gene therapy drug, manufactured by UniQure Inc.

This therapy utilizes viruses (adenovirus vector) designed in such a way that upon infection, the gene for producing the lipoprotein lipase is induced into the host cell, thereby producing the enzyme in-vivo. This therapy has shown positive results and is expected to be released into the market soon which presents opportunistic prospects for market expansion. For now, the global inherited metabolic disorders Market lies broadly in the dietary supplements market which is supporting market growth.

The use of genetic markers in T-cell receptor excision circles for severe combined immunodeficiency is one promising technique that augments market expansion. Moreover, the use of tandem mass spectrometry for the diagnosis of inborn errors of metabolism has the potential to expand the newborn screening panel which will propel the diagnostic market for isovaleric acidemia treatment.

2018 to 2022 Isovaleric Acidemia Treatment Demand Analysis vs. Forecast 2023 to 2033

The isovaleric acidemia treatment market was valued at US$ 520 Million in 2018 while exhibiting a CAGR of 4.64%. Adoption of new technology such as mRNA in metabolic testing procedures is expected to shift the pharmaceutical industry to new heights which are augmenting the demand for the Isovaleric Acidemia treatment market. Considering these factors, the market is estimated to be valued at US$ 1,125.15 Million, growing at a CAGR of 5.6% by end of the forecast period.

Which are Some Prominent Drivers of the Isovaleric Acidemia Treatment Market?

The advent of new technologies in IVA fuelling market growth

The advent of tandem mass spectrometry expanded newborn screening (NBS) and has become a mandatory public health strategy in most developed and developing countries, which will drive market growth. Moreover, this technology allows inexpensive simultaneous detection of more than 30 different metabolic disorders like Isovaleric Acidemia Treatment, in one single blood spot specimen at a cost of about US$ 10 per baby, with commendable analytical accuracy and precision, which will bolster the demand for Isovaleric Acidemia Treatment drug and therapeutics during the forecast period. studies have confirmed that the savings achieved through the use of expanded NBS programs are significantly greater than the costs of implementation.

Autonomy for local screening agencies to develop their screening programs in order to keep pace with international advancements is expected to support market expansion. Integration of existing clinical IEM services with the expanded NBS program to enable close communication between the laboratory, clinicians, and allied health parties.

The adjuvant therapy with sapropterin is also helpful for PKU treatment. In recent years, private medical insurers in the US and a few other countries are also offering reimbursement programs for the consumption of certain medical foods. Thus, the rising adoption of medical food and nutrient supplements is expected to foster market growth during the forecast period.

Sabyasachi Ghosh

Principal Consultant

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What are the Challenges Projected to be Faced by the Market?

High cost of diagnostic treatment restraining market expansion

Inadequate reimbursement scenario, high cost of treatment and delayed adoption of new emerging diagnostic and therapeutic technologies will restrain the market growth during the forecast period.

Region-Wise Insights

North America dominates the market with the highest market share

North America was the largest market for Isovaleric Acidemia Treatment therapeutics with a market share of 41.8% in 2022. The market revenue in this region is projected to be valued at around US$ 520 Million by 2033. It is likely to show prominent growth while retaining its dominance during the forecast period. This is due to the increase in demand for sapropterin and kuvan for amino acid metabolism disorders treatments in the region. Moreover, the region boasts of a wide availability of treatments and a well-established healthcare infrastructure.

The extensive presence of several end-users focusing on treatment and studies of chronic disease makes use of metabolomics, and this is the primary driver of market growth in the region. The active participation of the government in the form of investment and world-class resources for research activities is driving the growth of the metabolomics market in the region of Canada.

Mounting Investments in APAC to drive the market with the fastest CAGR

The market in Asia Pacific is estimated to be valued at US$ 350 Million, trailing at a CAGR of 4.9% during the forecast period. Growing investments in diagnostic healthcare, increasing adoption of new technologies, rising prevalence of inherited metabolic disorders in other regions, and increasing awareness regarding health are fuelling the demand for isovaleric acidemia disorder therapeutics in the region. China is expected to dominate the Inborn errors of metabolism market with more than 41% market share in this region owing to the rapid adoption of mRNA technology in diagnostics and surging advancements in the medical sector.

Category wise Insights

L-Carnitine Supplements to dominate the market by treatment type

The supplements carnitine and glycine can reduce the toxicity of isovaleric acid and aid in its removal from the body. L-Carnitine supplements are expected to dominate the treatment segment with a share of 79% and are projected to retain the highest weight during the forecast period.

Administration of equimolar amounts of glycine or L-carnitine separately with leucine demonstrated that isovaleryl-coenzyme A is removed by supplemental L-carnitine in the form of isovalerylcarnitine as effectively as it is by glycine, in the form of isovalerylglycine. Treatment with L-carnitine alone has proven effective in preventing further hospitalizations in a patient with this disorder, which will drive the segment growth by end of the forecast period.

Start-ups in Isovaleric Acidemia Treatment Market

• Founded in 2013, ‘Aeglea Biotherapeutics’ is a US-based startup that is developing novel treatments for inborn errors of metabolism and cancer by using engineered human amino acid degrading enzymes. Aeglea's lead program AEB1102 is in pre-clinical development and is expected to lower elevated arginine in blood as enzyme replacement therapy for Arginase 1 deficiency and treatment of arginine-dependent tumors. It received funding of USD 56 million from Novartis Venture Fund, Lilly Ventures, and 7 more investors.
• Founded in 2015, Fulcrum Therapeutics is a US-based company, focused on discovering and developing small molecules that modulate the on/off control mechanisms that regulate genes. The Fulcrum product engine integrates insights from four scientific disciplines (Gene Regulation, Human Genetics and Disease, Cell Biology, and Screening and Computation) to identify novel drug targets and develop breakthrough medicines. The early focus of Fulcrum’s product engine is on two severe genetic diseases: Fragile X syndrome (FXS) and a form of muscular dystrophy called facioscapulohumeral muscular dystrophy (FSHD). Each disease arises from a single gene mutation that creates an error in gene regulation. In the case of Fragile X, the silencing of the FMR1 gene eliminates the cell’s ability to make a protein needed for brain function. In FSHD, the precipitating event is the activation of a gene called DUX4 that should be silent in adulthood. This activation leads to muscle wasting as cells die. It received funding of USD 115 million from EcoR1, Highliner Capital, Boxer Capital, and 14 Other Investors.
• Founded in 2002, Alnalym is a US-based developer of RNA interference-based therapeutics for multiple disorders. The company's pipeline includes drugs that are used to treat hereditary ATTR Amyloidosis, acute hepatic porphyria, and in treating hypercholesterolemia. Further, it develops drugs for cardiometabolic diseases, infectious diseases, central nervous system, and ocular diseases. It received funding of USD 17 million from Blackstone, Genzyme, Polaris Partners, and 11 Other Investors.

Market Competition

Key players in the Isovaleric Acidemia Treatment Market are Amgen, Replimune Group Inc., Transgene SA, Oncolys BioPharma, Targovax, Lokon Pharma, Vyriad, TILT Biotherapeutics, VCNBiosciences, DNAtrix.

Some Recent Developments in this industry are:

• In 2020, Nutricia established a sustainable plant in the Netherlands. The Nutricia Cuijk plant was a massive investment that increased the volume of specialized infant formula and the market's revenue generation.
• In June 2022, Mead Johnson & Company, LLC announced that it had been completely merged with Reckitt Benckiser Group plc (RB). With Reckitt Benckiser plc groups' good innovation and marketing processes, it was expected that the Mead Johnson brand and business would continue to boom.
• On February 17, 2022, Maze Therapeutics, a company translating genetic insights into new precision medicines, announced the initiation of dosing in the company’s Phase 1 clinical trial of MZE001 in healthy volunteers. MZE001, an oral glycogen synthase (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup, is being evaluated for the potential oral treatment of patients with late-onset Pompe disease.
• On May 15, 2020, Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, announced positive initial data from the confirmatory third cohort and longer-term data from the first two cohorts of the ongoing Phase 1/2 study of DTX401, an adeno-associated virus (AAV) based gene therapy for the treatment of glycogen storage disease type Ia (GSDIa).

Report Scope

Key Segments Profiled in the Isovaleric Acidemia Treatment Market Industry Survey

By Treatment:

• L-Carnitine Supplements
• Glycine Supplements
• Others

By Region:

• North America
• Latin America
• Europe
• Asia Pacific
• Middle East & Africa (MEA)