1530 - Purified human alpha1-proteinase inhibitor (A1-PI) for the treatment of alpha1-proteinase inhibitor deficiency, leading to chronic obstructive pulmonary disease (COPD)

Page last updated: 23 February 2018

Application Detail

Status

Open

Description of Medical Service

Purified human A1-PI for therapy in adults with congenital deficiency of alpha-1 antitrypsin and with clinically significant lung disease, in addition to current BSC for COPD.

Description of Medical Condition

Alpha1-proteinase inhibitor (A1-PI) deficiency, also known as alpha1-antitrypsin deficiency, is a rare hereditary disorder characterised by low blood serum concentration of A1-PI, predisposing patients to chronic obstructive pulmonary disease (COPD) characterised by panacinar emphysema.
A1-PI deficiency results when the liver produces an abnormally low and insufficient level of serum A1-PI. Severe deficiency results in the inability to maintain an appropriate anti-proteinase defence, subjecting the individual to life-long and progressive loss of lung tissue.
Extra-pulmonary manifestations include different types of liver disease (including cirrhosis, hepatitis, and hepatoma), necrotizing panniculitis, systemic vasculitis, and other inflammatory and neoplastic diseases.
The progression of lung disease is typically slow with symptoms often appearing within the fourth or fifth decade of life with a delay of 7 to 10 years before appropriate diagnosis. Natural history studies have indicated that severe A1-PI leads to considerably reduced life expectancy, and that emphysema and liver disease are the most common causes of death.

Reason for Application

Health technology assessment

Medical Service Type

Therapeutic

Previous Application Number

Not Applicable

Associated Documentation

Application Form

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Consultation Survey

Consultation Survey (Word 508 KB)
Consultation Survey (PDF 307 KB)

PICO Confirmation

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Assessment Report

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Public Summary Document

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Meetings for this Application

PASC

12-13 April 2018

ESC

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MSAC

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