High Research & Development Initiatives on the Development of Stem Cell Based Therapeutics are Expected to Fuel the Growth of the Alpha 1-Antitrypsin Deficiency Treatment Market

Alpha 1-antitrypsin deficiency is an inherited genetic disorder that causes insufficient production of the protein alpha 1-antitrypsin (A1AT) in individuals. A1AT is a protease inhibitor that has the capability to protect body tissues from enzymes of various inflammatory cells such as neurophil elastase. Insufficient production of this protein leads to pulmonary medical complications chronic obstructive pulmonary disease (COPD), cirrhosis and emphysema. At present, the alpha 1-antitrypsin deficiency treatment market is a highly promising and attractive market venture due to increased government support and high adoption rate for novel therapeutics. Some of the symptoms of A1AT deficiency include:

-Tiredness
-Vision problems
-Shortness of wheezing and breath
-Repeated pulmonary infections
-Weight loss

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Some of the available therapeutics used for the treatment of alpha 1-antitrypsin deficiency are as follows:

-Prolastin
-Zemaira
-Aralast

Promising drugs for treating alpha 1-antitrypsin deficiency that are still in the pipeline are as follows:

rAAV1-CB-hAAT – a recombinant adeno-associated virus vector that helps in expressing alpha-1 antitrypsin gene in individuals affected with the condition

POL-6014 – a highly potent, novel, reversible and selective inhibitor of human neutrophil elastase that might be helpful in treating alpha 1-antitrypsin deficiency

CT-2009 – a drug molecule ready for IND that can selectively disrupts RANTES and Platelet Factor 4 for treating alpha 1-antitrypsin deficiency

Geographically, Europe and North America are projected to be the leading markets for alpha 1-antitrypsin deficiency treatment due to the high prevalence and incidence rate of alpha 1-antitrypsin deficiency in these regions. It is observed that this genetic disorder is most common among North Americans of European descent and Europeans. Racial groups other than white people are expected to be affected less frequently. In the U.S., one individual per 3000 - 5000 individuals is affected by alpha 1-antitrypsin deficiency and about 25 million individuals carry one deficient gene. Both men and women are equally affected in numbers.

Major factors propelling the growth of the alpha 1-antitrypsin deficiency treatment market are increasing incidence rate of genetic disorders and high adoption rate for novel therapeutics. Moreover, increasing government support for developing novel therapies and personalized medicines, along with high research and development initiatives on the development of stem cell based therapeutics are expected to fuel the growth of the alpha 1-antitrypsin deficiency treatment market. Furthermore, high demand for safe and effective pharmacological therapies along with increased life expectancy is expected to drive the growth of this market.

In addition, technological breakthrough such as point-of-care drug delivery systems and increased focus on retaining superior quality of life are some of the factors that might contribute in the growth of this market. However, rise in overall healthcare expenditure and stringent regulatory approvals are some of the market-restraining factors that might hinder the growth of the alpha 1-antitrypsin deficiency treatment market in future.

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At present, the treatment market for alpha 1-antitrypsin deficiency is highly consolidated due to the involvement of few players in the industry. However, it is expected that emerging biotechnological companies focused on the development of personalized medicines for genetic inherited disorders will try to achieve a significant amount of market share in the near future. Some of the companies involved in the development of therapies for treating alpha 1-antitrypsin deficiency are Baxter International Inc., CSL Behring LLC, Grifols Therapeutics, Inc., Halozyme Therapeutics, Inc., iBio, Inc., rEVO Biologics, amongst others.

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