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Molgramostim

Molgradex

Extended indication Autoimmune (acquired) pulmonary alveolar proteinosis (PAP).
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Molgramostim
Domain Lung diseases
Reason of inclusion Indication extension
Extended indication Autoimmune (acquired) pulmonary alveolar proteinosis (PAP).
Proprietary name Molgradex
Manufacturer Savara
Mechanism of action Growth factor
Route of administration Inhalation
Therapeutical formulation Nebulizing fluid
Budgetting framework Intermural (MSZ)
Additional remarks Recombinant human granulocyte macrophage colony stimulating factor (GM-CSF).

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date September 2024
Expected Registration October 2025
Orphan drug No
Registration phase Clinical trials
Additional remarks NCT04544293

Therapeutic value

Current treatment options Longspoeling met GM-CSF
Therapeutic value No estimate possible yet
Substantiation De IMPALA studie heeft zijn primaire eindpunten niet gehaald
Duration of treatment continuous
Frequency of administration 1 times a day
Dosage per administration 300 µg
References NCT02702180

Expected patient volume per year

Patient volume

< 100

Market share is generally not included unless otherwise stated.
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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension Yes
Indication extensions Nontuberculous Mycobacterium Infections
References Clinicaltrials.gov; Savara pipeline

Other information

There is currently no futher information available.