Molgramostim

327112008

New Medicines

Molgradex · Autoimmune (acquired) pulmonary alveolar proteinosis

Information

Molgradex
New formulation
Savara
Savara

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
None
Yes

Category

An inhaled formulation of molgramostim, a recombinant human granulocyte macrophage colony stimulating factor (GM-CSF).
PAP is a rare lung disease characterised by the accumulation of lipoproteinaceous material in the alveoli. There are three main forms of PAP, congenital, secondary and acquired. Acquired PAP is the most common type, accounting for about 90% of all cases. Symptoms may include shortness of breath and cough; in severe cases, respiratory failure may occur. The prevalence of PAP in the EU is estimated at 1-9 per 1,000,000 population [1].
Autoimmune (acquired) pulmonary alveolar proteinosis
Inhalation

Trial or other data

Jun 19: results of PIII IMPALA trial announced, the study did not meet its primary endpoint of alveolar-arterial oxygen gradient (A-aDO2), with an estimated 4.6 mmHg treatment difference (p=0.17) [4]


Feb 16: PIII IMPALA (NCT02702180) study starts. This study evaluates inhaled molgramostim in the treatment of 51 autoimmune PAP adult patients. A third of the patients will receive inhaled molgramostim daily for 24 weeks, a third will receive inhaled molgramostim every other day for 24 weeks and a third will receive inhaled matching placebo for 24 weeks. Study sites include Denmark, France, Germany, Greece, Israel, Italy, Japan, Netherlands, Russia, Switzerland & United Kingdom. Primary outcome is absolute change from baseline to 24 weeks of Alveolar - arterial oxygen concentration; collection of these data is expected to complete Aug 17 [2].

Evidence based evaluations