Peroxisomes are the membrane-bound organelles present in the eukaryotic cells except erythrocytes with highest concentration in the liver and kidney. They are involved in catalyzing various catabolic and anabolic functions in cellular metabolism. Peroxisome disorders are autosomal recessive disorders that occurs due to malfunctioning in peroxisome biosynthesis, assembly, and associated biochemical functions. It is estimated that 1 in 20,000 individuals in North America are affected by peroxisomal disorders. X-ALD is the most common peroxisomal disorder occurring in 1 out of 20,000 males in the U.S. Next to this, is the Zellweger syndrome occurring in early infancy which has an incidence rate of 1 out of 50,000 in the U.S. This disorder occurs in all countries, among all races and ethnic groups.
There is not any specific treatment available for the cure of peroxisomal disorders. Rather, several symptomatic treatments are available such as FDA approved cholic acid medication which helps in with trteating liver disease, weight gain, and vitamin absorption. Other than this; daily multivitamin, steroid or anti-seizure medication may be given as part of the treatment. Peroxisomal disorders treatment depends on the disorder type, age of onset, and the progression rate. There are several risks and benefits associated with the available therapies. Generally, multiple therapies and full care is given to the child for its survival. Along with this, several palliative care is also provided for survival assistance to these patients.
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Peroxisomal Disorders Treatment market: Drivers & Restraints
As specific peroxisomal disorders treatment is not available for curing the disease, this drives the market of several medications for symptomatic treatment. Clinical trials run be various research institutes and companies for the treatment of rare genetic disorders also supports the market growth. Unavailability of specific treatment for this particular rare disease is the biggest restraint of peroxisomal disorders treatment market. Furthermore, the survival rate of children with this disorder is very less which also restricts the market for its treatment. Very few research activities are going on in this direction which further slowdown the market progress for peroxisomal disorders treatment.
Peroxisomal Disorders Treatment market: Segmentation
By Treatment Type:
- Cholic acid treatment
- Other symptomatic treatment
By Disease Type:
- X-linked adrenoleukodystrophy (X-ALD)
- Zellweger syndrome (ZS)
- Others
By Age Group:
- New-Born Babies
- Older Children
By End Users:
- Hospitals
- Clinics
Companies involved in the peroxisomal disorders treatment market are involved in the development and approval of new products such as Retrophin, Inc.’s Cholbam recently got approved by FDA in 2015. Significant opportunities are present with the market players and research institutes to develop specialized medications in order to cure the disease. Betaine is another drug, currently in clinical trials for the treatment of peroxisomal disorder treatment. Prenatal counseling should be provided by government and non-government organizations to families having an affected child or are known carriers of such genetic disorders.
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Geographically, the market for Peroxisomal Disorders Treatment can be segmented into five regions, North America, Latin America, Europe, Asia Pacific and Middle East & Africa. North America dominates the global Peroxisomal Disorders Treatment market owing to awareness about the disease and available government support. This is followed by the European region due to the availability of healthcare facilities for the treatment of rare diseases as well as focus on research activities for finding treatment of rare and genetic disorders. Asia Pacific market is not developed in this specific treatment type but shows a significant growth rate over the forecast period. Latin America followed by Middle East & Africa represents the leaqst market share as well as market growth in the peroxisomal disorders treatment market owing to the less developed healthcare facilities as wella s less awareness about the disease in these regions.
The key player with FDA approved drug Cholbam for Peroxisomal Disorders Treatment include is Retrophin, Inc. Other drugs such as steroids and multivitamins are provided by other generic pharmaceutical manufacturing companies.