Experimental Treatment for Alpha-1 Antitrypsin Deficiency Earns Fast Track Designation

According to a story from globenewswire.com, the biopharmaceutical company Mereo BioPharma Group plc has recently announced that the company’s investigational therapeutic alvelestat has earned Fast Track designation from the US Food and Drug Administration (FDA). This designation is for the treatment of alpha-1 antitrypsin deficiency (AATD)-associated lung disease. The company aims to meet with the agency at the end of the year to discuss further options, including Accelerated Approval.

About Alpha-1 Antitrypsin Deficiency

Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder that can result in disease of the liver or lungs. Typically, problems with the lungs can begin at anywhere from 20 to 50 years old. Symptoms include shortness of breath, wheezing, increased risk of lung infections, and COPD. Not all patients experience liver problems, but in some, cirrhosis and liver failure may occur. A genetic mutation is responsible for the deficiency. Smoking can greatly worsen symptoms and outcomes, and even shorten life expectancy in people with the disorder. Most treatments are meant to improve symptoms and lung function; transplantation may be an option in those with severe disease. In some patients, augmentation of the deficient A1AT protein may be viable and can stop the progression of symptoms and lung damage. People with liver complications are unfortunately not suitable candidates for this treatment. However, long term effects of this treatment have not been studied. To learn more about alpha-1 antitrypsin deficiency, click here.

About Alvelestat

Alvelestat is classified as an orally active inhibitor of neutrophil elastase. It has produced positive data in terms of effectiveness and safety in phase 2 clinical trials. This study included patients with severe emphysema associated with AATD. Another ongoing trial is investigating the treatment in a wider array of patients. The drug is also being evaluated as a treatment for the rare disease bronchiolitis obliterans syndrome

About Fast Track Designation

Fast Track designation is given to experimental treatments in development to treat a life-threatening or serious condition with severe unmet medical need and is intended to expedite the review process.  This designation confers benefits such as earlier and more regular correspondence with the FDA as well as rolling review and submission of the approval application. This designation also provides options for Accelerated Approval.

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