I read that Savara has been granted “Breakthrough Therapy Designation“ for development and review of Molgradex for apap. Does anyone have more info on this?
Daryn Wynne
Jun 27, 2019
I have a quick question in regards to inhaled GM-CFS. What is the best solution to use when mixing the GM-CFS. The document that comes with the medication does list a few different options. If I use distiled water the shelf life in the documention mentions that it only good for a max of 6 hours. I will be splitting the bottle into 2 (every 12 hours).. So disstilled water wouldn’t be an option for the second dose. Other option is bacteriostatic water which give a shelf life of 20 hours or day (sorry don’t have the paper work handy). With the bacteriostatic i google it in regards to using it in a nebulizer and there are issue with it causing bronchitis. I anyone could help fill me with some information and the best way to do this it would be great as i can not find a lot of information in regards to this. Thank you, Daryn
I don't have any more info on other than what I have read on the internet. The "Breakthrough Therapy Designation" will definitely help fast track the process to getting Molgradex FDA approved.
Oh my thank you for the information. I heard there may be a shortage of Leukine due to the sale of the company, Sanofi. They sold the formula for making Leukine to a company in the United States. They should be up and running soon, hopefully the new medicine Molgradex will be less expensive. It is wonderful to here we all may have another option. Happy New Year to all and may we have a great Lung Year
Although Savara did receive Breakthrough Designation they will most likely have to run another PH III before potential approval, which will take years. They missed the primary endpoint on their recent PH III due to 4 patient outliers. Excluding those patients in the study that received supplemental oxygen, the study would have succeeded and most likely Molgradex will be on the market later this year,early next. There was clear signs of efficacy for aPAP patients, however the FDA seems to be steadfast on P-Values and not patient outcomes.Overall a tough break for aPAP patients.