Global Krabbe Disease Treatment Market - 2022-2029

Global Krabbe Disease Treatment Market - 2022-2029

Market Overview

The global krabbe disease treatment market size was valued 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).

Krabbe disease is a life-threatening neurological disorder commonly known as globoid cell leukodystrophy. It is one of a series of conditions known as leukodystrophies, caused by the breakdown of myelin in the nervous system (demyelination). Myelin is the protective layer that surrounds nerve cells and ensures that nerve signals are transmitted quickly. Krabbe illness is also characterized by aberrant brain cells known as globoid cells, huge cells with more than one nucleus. Krabbe disease is identified in children as young as six years old, and there is presently no cure; nevertheless, the condition is controlled by supportive care.

Market Dynamics

Advancement in the therapies for the treatment of Krabbe Disease is expected to drive market growth.

Other experimental medicines have demonstrated varying degrees of effectiveness in preclinical testing, with some of the best results from combining medications. For instance, HSCT has been used with SRT and gene therapy. The combinations consistently yield better outcomes than the matched individual therapies. Other approaches such as ERT, chaperone therapy, and anti-inflammatory therapy also have the potential to contribute as part of a combination or standalone therapies. Moreover, various clinical studies have been carried out. For instance, D-Cycloserine is an FDA-approved medicine for tuberculosis tested in numerous clinical studies for various diseases. Preliminary data suggest that it can slow disease progression in twitcher mice; consequently, D-cycloserine should be studied further to see if it can help individuals with Krabbe disease. Thus, the market is expected to drive in the forecast period from the above statements.

Restraint:

The finite patient pool for clinical trials, lack of awareness of the disease and side effects of the drugs are factors the market is expected to hamper in the forecast period.

COVID-19 Impact Analysis

The COVID-19 pandemic has moderately impacted healthcare systems and the market. The risk of severe forms of COVID-19 may be high in the rare metabolic community, where all patients, including children, experience multiorgan impairments due to their chronic condition. MetabERN, the European Reference Network for Hereditary Metabolic Diseases, conducted two surveys: one for patients' organizations (PO) and the other for healthcare providers (HCPs). COVID-19 incidence was lower in the community of uncommon metabolic patients (72.9 per 100,000 vs. 117 per 100,000) than in the general European population. On the other hand, patients were subjected to significant disruptions in their care, with most appointments and treatments being canceled, curtailed, or postponed. Almost all HCPs (90 percent) used telemedicine to replace face-to-face visits, about half of patients undergoing treatment changes switched from hospital to home therapy, and a quarter of patients had difficulty getting their medications.

Additionally, the pandemic interrupts the supply chain and process. Many companies will vary to other geographic regions to ensure that products remain available and protect their supply chain. Thus, from the above statements, the market got affected, and it is expected to gain traction quickly with the resumption of the economic activities.

Segment Analysis

Stem cell transplantation segment is expected to hold the largest market share in krabbe disease treatment market

The stem cell transplantation segment is expected to dominate in 2021. The segment benefits because if treatment begins before the development of symptoms, that is, when a diagnosis is made via a newborn screening test, this therapy may improve outcomes in newborns. According to current evidence, stem cell transplantation is most beneficial when initiated before a newborn reaches the age of two weeks. Moreover, Pre-symptomatic children who have stem cell transplantation had a slower disease progression and a better quality — and duration — of life than children who do not receive stem cell transplantation before symptoms arise, according to studies. Furthermore, This medication may also benefit older children and adults with modest Krabbe disease symptoms. Thus, the market segment is expected to hold the largest market share in the forecast period from the above statements.

Geographical Analysis

North America region holds the largest market share in the global krabbe disease treatment market

In 2021, North America accounted for the highest revenue share. The increasing prevalence of Krabbe Disease, rising geriatric population, Rapid adoption of cutting-edge technologies and novel medications for the treatment of Krabbe illness, well-established infrastructure and increase in clinical development of the treatment for the Krabbe disease, and rise in investment in research & development are some of the factors the market is expected to boost in the forecast period. For instance, the Krabbe disease affects about 1 in 100,000 people in the United States. It is also known as globoid cell leukodystrophy. Males are affected as often as females.

Also, more than two-fifths of all known cases of Krabbe disease is caused by a missense mutation in the GLC enzyme. Therapeutic approaches for other lysosomal storage diseases are ineffective in treating Krabbe disease due to the lack of accumulated primary substrate within the lysosome and the predominant CNS sickness. The molecules are being researched because they have the potential to pass the blood-brain barrier more efficiently than enzymes due to certain properties such as low molecular weight, low toxicity, and high bioavailability. Allosteric chaperones, directed away from the enzyme's active site, have also been found. Pharmacological chaperone therapy, which is expected to be effective, can be used to treat Krabbe disease. Thus, the North American region is expected to hold the largest market share in the forecast period from the above statements.

Competitive Landscape

Major key players in the krabbe disease treatment market are Lannett, Novartis, Lundbeck, Lupin Pharmaceuticals, Inc., Teva Pharmaceuticals USA, Inc., Meda Pharmaceuticals Inc , Mayne Pharma Group Limited, Pfizer Inc., Schwarz Pharma, Inc, Acorda Therapeutics Inc.

Pfizer Inc.:

Overview:

Pfizer Inc. is an American multinational pharmaceutical company that offers medicines, vaccines, medical devices, and consumer healthcare products for oncology, inflammation, cardiovascular, and other therapeutic areas. It has a portfolio of over 150 products across 15 therapeutic areas. The company develops and produces medicines and vaccines for a wide range of medical disciplines, including immunology, oncology, cardiology, endocrinology, and neurology.

­Product Portfolio:

SKELAXIN(metaxalone): Metaxalone is a muscle relaxant. It works by blocking nerve impulses (or pain sensations) in the brain. It is used with rest and physical therapy to treat skeletal muscle conditions such as pain or injury.

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