Shire plc and Kamada Ltd., a plasma-derived protein therapeutics company focused on orphan indications, announced that the United States Food and Drug Administration (FDA) has approved an expanded label for Glassia [Alpha-1 Proteinase Inhibitor (Human)], marking the first treatment for adult patients with emphysema due to severe Alpha-1 Antitrypsin (AAT) Deficiency that can be self-infused at home after appropriate training.

Patients with AAT Deficiency have low or undetectable levels of a protein called alpha-1 antitrypsin or AAT, which helps protect lung tissue from damaging enzymes that are released by white blood cells. AAT deficiency can result in early onset emphysema. Treatment with Glassia replaces the missing or deficient AAT protein in the blood and lungs.

There are an estimated 100,000 people in the United States who have the disorder, though under-diagnosis remains an issue, as fewer than 10 percent of those living with AAT deficiency have been properly diagnosed. The World Health Organization (WHO), the American Thoracic Society (ATS), the European Respiratory Society (ERS), and the Alpha-1 Foundation’s Medical and Scientific Advisory Committee (MASAC) recommend that all patients with chronic obstructive pulmonary disease (COPD) be tested for the disorder.

“Patients with Alpha-1 Antitrypsin Deficiency are managing a challenging disorder that may require regular regimented care. For these patients, our company strives to find ways to provide them with more choices and flexibility in their treatment regimen,” said Blaine Forshage, head of the US Immunology Franchise for Shire. “With this new label for Glassia, we’re now able to offer the only AAT augmentation treatment that is approved for patients to self-administer at home, helping to deliver on our goals to support the Alpha-1 community.”

Approved in 2010, Glassia is the first and only liquid ready-to-use augmentation product approved for treatment of clinically evident emphysema due to severe AAT Deficiency. Kamada and Baxalta (formerly Baxter International Inc’s BioScience business and now part of Shire) entered into an exclusive strategic cooperation agreement for the distribution and license of Glassia in 2010. Under the terms of the agreement, Baxalta is the exclusive distributor of Glassia in the US, Canada, Australia and New Zealand, and is licensed to produce Glassia using Kamada’s technology at a Baxalta facility for sales in those countries.

“Self-infusion, after proper training, can be a convenient way for Alphas to receive their augmentation therapy,” said Henry Moehring, Alpha-1 Foundation president and CEO. “We at the Foundation are always gratified to see expanded treatment options for Alphas, and we applaud the FDA’s approval of this new labeling.”

“We are excited that the FDA has now permitted patients to self-infuse Glassia at home,” said Amir London, Kamada’s chief executive officer. “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. We are very pleased with the continued increase in the number of US patients treated with Glassia, and believe that self-infusion will contribute significantly to future growth. Importantly, due to the company’s state-of-the-art production facility, Kamada has the capacity to support the increasing demand for Glassia. Finally, we look forward to a strong partnership with Shire that is beneficial to the patients we serve, and both of our companies.”

Glassia is an Alpha1-Proteinase Inhibitor (Human) (Alpha1-PI) indicated for chronic augmentation and maintenance therapy in adults with clinically evident emphysema due to severe hereditary deficiency of Alpha1-PI (alpha1 antitrypsin deficiency). Glassia increases antigenic and functional (anti-neutrophil elastase capacity, ANEC) serum levels and antigenic lung epithelial lining fluid levels of Alpha1-PI.

The effect of augmentation therapy with any Alpha1-PI, including Glassia, on pulmonary exacerbations and on the progression of emphysema in Alpha-1 antitrypsin deficiency has not been conclusively demonstrated in randomized, controlled clinical trials.

Clinical data demonstrating the long-term effects of chronic augmentation and maintenance therapy of individuals with Glassia are not available.

Glassia is not indicated as therapy for lung disease in patients in whom severe Alpha1-PI deficiency has not been established.

The serious adverse reaction observed during clinical trials was exacerbation of chronic obstructive pulmonary disease (COPD).

The most common adverse reactions occurring in >0.5% of infusions in clinical trials were headache and upper respiratory infection.