Molgramostim Medicine 12 June 2019 Extended indication Treatment of pulmonary alveolar proteinosis in adults. Therapeutic value No estimate possible yet Total cost € 30,000,000.00 Registration phase Registration application pending Product Active substance Molgramostim Domain Lung diseases Reason of inclusion New medicine (specialité) Main indication Extended indication Treatment of pulmonary alveolar proteinosis in adults. 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 Intermural (MSZ) 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 ATMP No Submission date March 2026 Expected Registration April 2027 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Longspoeling met GM-CSF Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation De IMPALA studie heeft zijn primaire eindpunten niet gehaald. Duration of treatment Frequency of administration 1 times a day Dosage per administration 300 µg References NCT04544293 (IMPALA-2) 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 tot 8 patiënten in Nederland). De prevalentie op 3,7-6,2 per miljoen inwoners (63 tot 105 patiënten in Nederland). Maximaal zullen hier dus ongeveer 100 patiënten voor in aanmerking kunnen komen. Expected cost per patient per year Cost € 100,000.00 - 500,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References Op basis van dosering en vergelijkbare GM‑CSF‑biologicals is een verwachte prijs in de orde van €100.000–€500.000 per patiënt per jaar Additional remarks Potential total cost per year Total cost € 30,000,000.00 Total cost for sluice Additional remarks Off label use Off label use 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