I think it was approved because it met the same type of criteria as Fabryzyme on surrogate biomarkers in the amenable mutation patients. What that really means for kidney function will be deduced with the confirmatory trial the FDA is asking of them.
Agreed that Amicus's product won't make them profitable, given their current leaner cash burn of 172 million in Q3, and their expected 2018 total revenue from Galafold of 80 million.
The EMA approved the drug 1.5 years ago, and so getting about 50 million from EMA after that much time doesn't sound like it's taking off like wild-fire.
Japan approved it 9 months ago, and Amicus mentioned they are in 'now' double digit prescriptions there, meaning probably in the region of about 10-15 prescriptions, which doesn't sound too amazing to me.
And how many patients does that mean though? 1 prescription is 1 patient or in 9 months 1 patient could have had repeat prescriptions? I mean the product comes in 123mg capsules that need to be taken every 2 days. So for 9 months, would the patient be prescribed directly 135 capsules (about 10 boxes) to cover that time period? Given that one box of Galafold contains 14 capsules, it would seem they would need a new prescription every month.
US approved it 4 months ago and there there have over 100 'individual prescriptions', so again what does the individual prescription really mean, and would that mean effectively about 25-50 patients?
So it seems a bit of an ask to reach the CEO's expectation of 1 billion total company sales by 2023, in 4 years. I can only assume he would expect this to all come from Galafold as the other products in the pipeline seem much further away than 4 years to marketing approval in any territory. I somehow doubt they will increase Galafold revenues by over 10 times in 4 years given its present uptake, but one never knows.
I think Amicus's M/C is based on the sensation from analysts that it is a revenue making company, and that it has quite a few preclinical and discovery gene-therapy compounds that it bought in, and gene-therapy is a huge buzz-term for analysts. Just look at bluebirdbio R&D expenses circa 100 million per quarter, and while they have their 1st product (gene-therapy) being reviewed by the EMA under accelerated approval based on mainly Phase II data, they were almost double their present market cap over a year before the submission of data to the EMA, putting their M/C at the time into the 10 billion region. Credit to them is their product seems to be working in beta-thalassemia patients. While most seem to be reducing dependence on blood transfusion, they seem to have a few that became transfusion independent but for how long that will remain needs to be seen. While great work 10 billion valuation pre-approval is remarkably high and again attributed to analyst buzz-terms. Meanwhile as you say PLX would be in line for a billion dollar product per year with great data going out 24 months and we are at 60 million M/C. Seems almost laughable.
I really can't wait to have the data from the 100 patients thus far delivered to the FDA for Accelerated Approval, and with the recent positive trend eGFR Bridge data. With the clinical data to date I don't see what would stop that happening. Once that does or whether or not it happens before Balance trial data, again I feel from the data and duration of efficacy that it would be a much clearer switch-over of patients from Fabryzyme and Replagal to 102, and then we're flying, hopefully anyway :)
