FDA Grants Orphan Drug Designation for DCR-A1AT for Treatment of Alpha-1 Antitrypsin Deficiency

Dicerna Pharmaceuticals works in ribonucleic interference (RNAi) therapies as treatments of rare diseases. They have recently been granted the orphan drug designation by the European Commission for their drug, DCR-A1AT. This therapy is meant to treat alpha-1 antitrypsin deficiency, which can lead to lung and liver disease. The orphan drug designation is meant for therapies that aid in the treatment, diagnosis, or prevention of rare diseases that have no satisfactory treatment available. With this designation Dicerna will receive incentives and funding to ensure that DCR-A1AT will be able to help people with alpha-1 antitrypsin deficiency.

About Alpha-1 Antitrypsin Deficiency (A1AD)

Alpha-1 antitrypsin deficiency (A1AD) is a rare disease that is characterized by low levels of the Alpha-1 Antitrysin protein. This deficiency leads to lung and liver disease, along with skin problems. It effects one of every 1,500 to 3,000 people of European ancestry, and it is much less likely to affect those of African or Asian ancestry.

A mutation in the SERPINA 1 gene causes this disease, as it responsible for the production of the Alpha-1 Antitrysin protein. This protein protects the body from an enzyme called neutrophil elastase, which is meant to fight infections. If this enzyme is unregulated then it will attack healthy body tissue. Abnormal proteins can get stuck in the liver, which leads to liver damage.

Severity of symptoms can vary by the affected area and the individual. The main symptoms of this deficiency are lung and liver disease, which bring their own symptoms. For lung disease people may experience shortness of breath, wheezing, activity intolerance, fatigue, unintentional weight loss, recurring respiratory infections, a rapid heart rate when standing, and advanced lung disease, which is known as Emphysema. If one develops liver disease their symptoms may include a swollen and distended abdomen, jaundice, elevated liver enzymes, dark urine, swollen hands and feet, chronic itching, cirrhosis, and an increased risk of liver cancer. A minority of people with A1AD experience skin symptoms, which are skin lesions and hard, painful lumps under the skin.

As of now, there is no cure for A1AD. Treatment is aimed towards the lung disease, as it is the most common symptom of this deficiency. Options include inhaled bronchodilators, steroids, vaccines to prevent infections, early diagnosis and treatment of lung infections, supplemental oxygen, lung rehabilitation, and a lung transplant. A specialist is recommended for those who develop liver disease or skin symptoms.

About DCR-A1AT

DCR-A1AT is a ribonucleic acid interference (RNAi) therapeutic that is spontaneously administered. Dicerna is developing it for the treatment of the liver disease that comes with A1AD, as A1AT is a protein that primarily found in the liver. This drug targets the SERPINA 1 gene that causes this deficiency, and it reduces the production of the abnormal protein in the liver. A clinical trial of this therapy is currently being conducted. If approved, this drug will be the first to treat liver disease associated with A1AD.

DCR-A1AT is a part of Dicerna’s RNAi technology platform, called GalXC™. The aim of this technology is to silence genes in the liver and other tissues that cause diseases. RNAi therapies bind to receptors in the liver cells, which then gives access to the RNAi machinery in the cells. The GalXC technology’s goal is to make this RNAi therapy as potent as it can be. Dicerna uses this technology in diseases associated with the liver and other organs.

Find the original article here.


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