Replacement therapy (also called substitution or augmentation therapy) is a general term for replacing the missing alpha-1 antitrypsin in the bodies of lung or panniculitis affected Alphas.
Currently, all the alpha-1 antitrypsin used is extracted from donated blood plasma. Large amounts of plasma are need to produce sufficient antitrypsin to be used therapeutically. For this reason the treatment is very expensive.
Attempts have been made to produce alpha-1 antitrypsin in other ways but to date these have not been successful.
The method of introducing the antitrypsin into the body is by intravenous infusion. Only 10% to 15% of the drug reaches the lower repiratory tract where it protects the alveoli in the same way as naturally produced alpha1 antitrypsin. Clinical trials are currently underway to test the effectiveness of inhaled antitrypsin. This procedure is simpler than an infusion and possibly more effective (25% to 45% reaches the repiratory tract).
AAT is produced by the following pharmaceutical companies:
|Baxter||Aralast & Aralast NP|
|CSL Behring||Zemaira (see note 3)||USA, Brazil, New Zealand|
|Grifols||Trypsone (see note 1)||Spain, Mexico, Brazil|
|Grifols||Prolastin (Prolastina, Prolastin C & Pulmolast)||USA and others. See note 2|
Note 1. Production of Trypsone ceased in 2014.
Note 2. In the EEA this product is licensed as Prolastin in Austria, Ireland, Italy, Germany, Greece, Poland and Portugal. It is licensed under the name Prolastina in Denmark, Finland, Norway, Spain and Sweden. In Belgium the licensed product is called Pulmolast.
The existence of a marketing licence does not imply that the product is reimbursed or made generally available to patients who may benefit from it.
Note 3. In December 2013 CSL Behring submitted a Marketing Authorisation Application to the European Marketing Agency. The application was based on the results of the RAPID Clinical Trial. The name of the product was changed from Zemaira to Respreeza.
The EMA Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorisation for Respreeza®, a highly purified alpha-1 protein derived from human plasma, indicated to treat patients with alpha-1 antitrypsin deficiency (AATD). The final decision will be made by the European Council.