PROLASTIN

Rare disease - First assessment

Opinions on drugs - Posted on Jul 24 2020

Reason for request

Inclusion

First assessment.

Favourable opinion for reimbursement in the long-term adjuvant treatment of patients with alpha-1 antitrypsin (AAT) deficiency [phenotypes PiZZ, PiZ(null), Pi(null, null), and PiSZ], with moderate to severe airway obstruction only (FEV₁ 35–60%) and on the basis of assessment of clinical condition (disability).

What therapeutic improvement?

No clinical added value in the current therapeutic strategy for patients with AAT deficiency.

Role in the care pathway?

The medicinal treatment of patients with AAT deficiency-related emphysema is the same as that for all patients with chronic obstructive pulmonary disease. It consists primarily of bronchodilators and inhaled corticosteroids as a fixed-dose combination with long-acting β2-adrenergic agonists in patients at risk of exacerbations.

Alpha-1 antitrypsin replacement is the only specific treatment for AAT deficiency.

Role of the medicinal product in the care pathway

PROLASTIN (human alpha-1 antitrypsin) is a first-line replacement therapy in patients with alpha-1 antitrypsin (AAT) deficiency [phenotypes PiZZ, PiZ(null), Pi(null, null), and PiSZ] with moderate to severe airway obstruction only (FEV₁ 35–60%) and on the basis of assessment of clinical condition (disability).

Clinical Benefit

Low	The clinical benefit of PROLASTIN (human alpha-1 antitrypsin) is low in the MA indication.

Clinical Added Value

no clinical added value

Considering:

the available clinical data and, in particular, a randomised, double-blind, placebo-controlled study already assessed by the Committee in the context of the evaluation of the other proprietary medicinal products containing human alpha-1 antitrypsin,
the lack of definition of a clinical relevance threshold enabling estimation of the clinical relevance of the effect observed on slowing the decline of pulmonary parenchymal density with alpha-1 antitrypsin compared to placebo,
the medical need currently partially met by two proprietary medicinal products based on human alpha-1 antitrypsin, but the need for additional alternatives to compensate the situations of supply pressure and shortage previously reported with the two medicinal products already available,

the Committee considers that PROLASTIN (human alpha-1 antitrypsin) provides no clinical added value (CAV V) in the current management of patients with alpha-1 antitrypsin deficiency.