Molgramostim Medicine 12 June 2019 Extended indication Autoimmune (acquired) pulmonary alveolar proteinosis (PAP). Therapeutic value No judgement Total cost Registration phase Clinical trials Product Active substance Molgramostim Domain Lung diseases Reason of inclusion New medicine (specialité) Main indication Lung other Extended indication Autoimmune (acquired) pulmonary alveolar proteinosis (PAP). Current proprietary name Already available biosimilars / generics Proprietary name Molgradex Manufacturer Savara Portfolio holder Mechanism of action Growth factor Route of administration Inhalation Therapeutical formulation Nebulizing fluid Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Recombinant human granulocyte macrophage colony stimulating factor (GM-CSF). Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date 2019 Expected Registration 2020 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Geen officiële weesgeneesmiddelstatus bij de EMA, maar zou hier wel voor in aanmerking komen. Therapeutic value Current treatment options Longspoeling met GM-CSF Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation De verwachting is dat molgramostim in de tweede of derde lijn zal worden ingezet. Resultaten van de IMPALA studie worden later in 2019 verwacht. Duration of treatment Frequency of administration 1 times a day Dosage per administration 300 µg References NCT02702180 Additional remarks Expected patient volume per year Patient volume < 100 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Huisman et al. Ned Tijdschr Geneeskd. 2008;152:1450-4 Additional remarks De incidentie wordt geschat op 0,36-0,49 gevallen per miljoen inwoners per jaar (6-8 patiënten in Nederland). De prevalentie op 3,7-6,2 per miljoen inwoners (63-105 patiënten in Nederland). Maximaal zullen hier dus ongeveer 100 patiënten voor in aanmerking kunnen komen. Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Nontuberculous Mycobacterium Infections References Clinicaltrials.gov; Savara pipeline Additional remarks Other information Additional remarks