Respreeza

What is Respreeza and what is it used for?

Respreeza is a medicine used in adults with human alpha1-proteinase inhibitor deficiency, an inherited disorder that can cause lung problems including increased breathing difficulties and can also affect the liver. Respreeza is used to slow down lung damage in patients with severe deficits.

Respreeza contains the active inhibitor of human alpha1-proteinase.

How is Respreeza used?

Respreeza is available as a powder and solvent for the preparation of a solution for infusion (drip into a vein). The first infusion should be administered under the supervision of a healthcare professional experienced in the treatment of human alpha1-proteinase inhibitor deficiency. Subsequent infusions can be administered by the patient or by the patient himself.

The recommended dose of Respreeza is 60 mg / kg of body weight, given once a week. The infusion should last about 15 minutes.

The medicine can only be obtained with a prescription. For more information, see the package leaflet.

How does Respreeza work?

The active substance in Respreeza, the alpha1-human proteinase inhibitor, is a natural component of the blood whose function is to protect the lung tissue. It is obtained from human blood and acts by replacing the protein lacking in patients with human alpha1-proteinase inhibitor deficiency.

What benefit has Respreeza shown during the studies?

Respreeza has been shown to slow lung damage in a main study involving 180 patients with lung injury due to human alpha1-proteinase inhibitor deficiency. In this study, Respreeza was compared with placebo (a dummy treatment) and the main measure of effectiveness was the decrease in lung density. Pulmonary density is an indicator of the extent of lung damage: the greater the decrease in lung density, the greater the injury to the lungs. The reduction in lung density after 24 months was about 2.6 g / l in patients treated with Respreeza compared to about 4.2 g / l in subjects treated with placebo.

What is the risk associated with Respreeza?

The most common side effects with Respreeza (which may affect up to 1 in 10 people) are dizziness and headache. Allergic reactions were observed during treatment, some of which were serious.

In view of the risk of serious allergic reactions, Respreeza should not be used in patients with a protein called IgA deficiency and who have developed antibodies against it, as these individuals are more prone to manifest allergic reactions. For the full list of limitations and side effects with Respreeza, see the package leaflet.

Why has Respreeza been approved?

The main study conducted with Respreeza showed that the medicine is effective in slowing lung damage in patients with human alpha1-proteinase inhibitor deficiency and this effect was considered significant in those with severe deficits. Regarding safety, the main fear related to the use of Respreeza is allergic reactions; however, information on how to manage this risk was provided in the product information. No other important safety concerns emerged regarding the medicine.

Therefore, the Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Respreeza's benefits are greater than its risks and recommended that it be approved for use in the EU.

What measures are being taken to ensure the safe and effective use of Respreeza?

A risk management plan has been developed to ensure that Respreeza is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Respreeza, including the appropriate precautions to be followed by healthcare professionals and patients.

In addition, the company that markets Respreeza will carry out a new study to assess whether a higher dose (120 mg / kg body weight) may offer greater benefits than the currently recommended dose.