Investigational Gene Therapy for Hereditary Angioedema Gets Orphan Drug Designation

According to a story from, the gene therapy company Adverum Biotechnologies recently announced that the US Food and Drug Administration (FDA) has granted them Orphan Drug designation for their experimental product ADVM-053. This drug is in development for the treatment of hereditary angioedema. Adverum focuses on the development of treatments for serious, rare diseases that do not have approved treatments.

About Hereditary Angioedema

Hereditary angioedema is a rare genetic disorder which is characterized by episodes of severe swelling. Without treatment, such episodes can occur every two weeks or so and can last up to several days. These attacks are potentially fatal if the airway becomes blocked. It is caused by a heritable genetic mutation. Symptoms of hereditary angioedema include swelling of the limbs, face, genitalia, lips, airway, and intestinal tract. For many patients, symptoms often first appear around the onset of puberty. When the intestinal tract is involved, abdominal pain and vomiting can occur. Thankfully, there are a number of treatments available that can help prevent attacks from appearing and can also bring down swelling. With sufficient access to treatment, outcomes for the condition are generally good. To learn more about hereditary angioedema, click here.

A Potential Game Changer

ADVM-053 is a gene therapy that is intended to be used only once per patient and could potentially provide long term relief from symptoms of the condition.

About Orphan Drug Designation

Orphan Drug designation from the FDA is typically reserved for experimental drugs that display certain capabilities. In order to qualify in most circumstances, the therapy must be intended to treat a disease that is considered rare, which is defined as any disease that affects less than 200,000 people in the US. In addition, the drug must also demonstrate advantages in safety or effectiveness over currently available treatments, or have the potential to address a currently unmet medical need. The designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a seven year period of market exclusivity if the drug gains approval.

Adverum plans to submit an Investigational New Drug (IND) application to the FDA before the end of the year.

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