Rare Disease Report

FDA Approves At-home Treatment for AAT Deficiency

JUNE 14, 2016
James Radke, PhD
The FDA has approved an expanded label for Glassia [Alpha-1 Proteinase Inhibitor (Human)], so that adult patients with emphysema due to severe alpha-1 antitrypsin (AAT) deficiency can now self-infuse the orphan drug at home.

What is AAT Deficiency

Patients with AAT deficiency have low or undetectable levels of alpha-1 antitrypsin or AAT, a protein that helps protect lung tissue. 
AAT normally works to inactivate another protein released from white blood cells to fight infection. When not tightly controlled by AAT, this protein can damage normal tissues, especially those in the lungs. That is why AAT deficiency predisposes people to lung disease. The risk is even higher in people who smoke.

There are an estimated 100,000 people in the United States who have AAT deficiency but is is likely under diagnosed with estimates that 90% of AAT deficiency patients do not know they have the condition. As we wrote earlier this week, patients with COPD, emphysema, or bronchiectasis should be tested for AAT deficiency to help in detecting this rare condition.

At-home Therapy Will Save time and Money

Treatment with Glassia replaces the missing or deficient AAT protein in the blood and lungs.
Glassia is administered intravenously once a week. In the past, patients only had the option to receive treatment by a medical professional. Now, they can administer the drug at home, thereby saving time and money.
In a press release, Amir London, Kamada's Chief Executive Officer said, "By avoiding the need for reconstitution, our product allows for an overall reduced treatment preparation time. With this new approval, patients have the convenience of self-infusion at home, in addition to potentially reducing costs previously associated with infusion services for administering augmentation therapy in the hospital.”
In the United States, Canada, Australia, and New Zealand, Glassia is manufactured and distributed by Baxalta (Shire). Outside of those countries, Glassia is manufactured and distributed by Israeli-based Kamada.

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