The pompe
disease market is growing at a significant rate mainly due to special
provisions for orphan drugs. Regulatory assistance in emerging nations is one
of the major trends seen in the pompe disease market.
Pompe
disease is a type of lysergic acid diethylamide (LSD). LSD refers to a group of
diseases arising from the increase of specific substrates and is inherited
autosomal recessive disorders. Lysergic acid diethylamide is progressive and
having uneven age of onset and clinical symptoms. Pompe disease is a hereditary
and repeatedly incurable disorder caused by lack of acid alpha glucosidase, an
enzyme desired to split down glycogen in particular structures of our body.
Patients
having pompe disease have little amount of or no acid alpha glucosidase enzyme
activity, so break down of glycogen is not possible. The additional glycogen
builds up in the patient and is stored in skeletal muscle, heart and other
tissues, which causes the progressive indications of pompe disease. Increase of
glycogen in certain organs and tissues restraints normal functions of patient’s
organs. Pompe disease is also known by other names such as glycogen storage
disease type II, acid maltase deficiency, lysosomal alpha-glucosidase
deficiency, acid alpha-glucosidase deficiency and glycogenosis type II.
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Pompe disease is categorized into two broad categories as adult-onset pompe disease
and infantile onset pompe disease. Both the parents pass on one strangely
altered copy of the gene to their child. A parent with an altered copy of the
gene is known as a carrier and the disease can affect both men and women
irrespective of the gender. The disease is acquired by the individuals at the
time of birth only, but the onset of indications differs significantly. The
researchers have identified more than 300 genetic mutations that can cause
pompe disease. Enzyme replacement therapy and diet therapy are useful in
dropping the clinical demonstrations of the disease.
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Government
regulations, such as orphan drug exclusivity and limited patient population are
the factors restraining the growth of the pompe disease market.
Some of the
competitors in the market are Genzyme Corporation, EpiVax, Inc., Amicus
Therapeutics, Audentes Therapeutics, BioMarin Pharmaceutical Inc., Valerion
Therapeutics, LLC, Oxyrane, and Sangamo BioSciences Inc.
