Sara Pellegrino – Senior Director, Investor Relations John Crowley – Chairman & Chief Executive Officer Bradley Campbell – President & Chief Operating Officer Chip Baird – Chief Financial Officer.

Ritu Baral – Cowen & Company Joseph Schwartz – Leerink Partners Roy Buchanan – Janney Montgomery.

Good day, ladies and gentlemen and welcome to the Amicus Therapeutics Third Quarter 2016 Financial Results Conference Call and Webcast. At this time, all participants are in a listen-only mode. Later, we will a conduct a question-and-answer session and instructions will follow at that time.

[Operator Instructions] I would now like to turn the conference call over to Sara Pellegrino, Senior Director, Investor Relations. Please go ahead..

Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics third quarter 2016 financial results, corporate highlights and program updates. Speaking on today's call, we have John Crowley, Chairman and Chief Executive Officer; Bradley Campbell, President and Chief Operating Officer; and Chip Baird, Chief Financial Officer. Dr.

Jay Barth, Chief Medical Officer; Dr. Hung Do, our Chief Science Officer; and Dipal Doshi, our Chief Business Officer and General Manager of Scioderm are also here and available to participate in the Q&A session.

On this call, we may make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business as wells as our plans and prospects. Our forward-looking statements should not be regarded as representation by us that any of our plans will be achieved.

Any or all of the forward-looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward-looking statement, which speak only as of the date hereof.

All forward-looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof.

For a full discussion of such forward-looking statements and the risks and uncertainties that may impact them, we refer you to the forward-looking statement on Slide 2 in this presentation of our third quarter results slide deck, the forward-looking statements and risk factor sections of our Annual Report on Form 10-K for the year ended December 31, 2015, and our Quarterly Report on Form 1O-Q we filed later today with the Securities and Exchange Commission.

At this time, it is my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics..

Great, thank you, Sara, and good morning, everybody.

I'm pleased to welcome everybody to this call and we'll begin with an update on the significant progress that we've made during the third quarter across our five key strategic priorities and for this I'll go ahead and ask you to please reference Slide number 3 in our deck as you're following along in the web.

So first, let me begin with the Galafold international launch which as you will see both very early in the product launch is going exceptionally well.

Today is a milestone for Amicus in our evolution to a commercial stage company, as we are here for the first time reporting full quarter launch detail, as well as our first ever quarter with revenue from product sales.

Brad will go into much more details but I will -- I just want to say upfront that I really am extremely pleased with the commercial progress in bringing this new medicine to Fabry patients who have not had a new treatment option in more than 13 years and where we know there is still such unmet needs.

We're pleased to report that we have now 50 patients with amenable mutations on reimbursed Galafold as of the end of October which is more than double the number we've reported on our last call as of July 31. The vast majority of these patients are in Germany and most are switching from the approved ERT products.

Germany is the only EU country currently where Galafold is commercially available which of course is typical to enroll the product launch as Germany is most often the first launch country for new medicines approved by the European Medicines Agency or EMA.

In Germany [indiscernible] as we know it has been an important indicator for subsequent launch countries. This is a very important country for Amicus and for our first product launch.

So recently I had the chance to visit a number of Fabry treatment centers in Germany and I can tell you from meeting at those centers knowing that we have a great leadership team at Amicus in Germany and I believe they are very effectively communicating the strength of the label and our data package to positions and that they are doing so in a manner consistent with our high business ethics here at Amicus.

There is tremendous enthusiasm about this precision medicine approach and the KOLs initial experience in treating Fabry patients with amenable mutations Galafold seems to be very positive.

Although that Germany is also very important because it is the third largest Fabry market in the world; and I think it's also particularly important as well as better weather for the Galafold launch.

In this prior to the EMA approval that we have in late May of this year, we had no clinical sites nor any patients in Germany at approval at the end of May. So the German launch really was from ground zero. So early days were very encouraging and again, Brad will provide more detail shortly.

Our second priority is to pursue additional regulatory submission for Migalastat. Here we continue to work with regulators in key geographies, we remain on-track to provide a U.S. regulatory update in the fourth quarter and we are targeting a Japanese new drug application in the first half of 2017.

Our third priority is to successfully execute our important clinical studies in Pompe disease and [indiscernible]. Here we are eagerly anticipating initial interim data from our Pompe clinical study by year-end 2016. We are also targeting top line data from our Phase 3 ED study in the first half of 2017.

We also have strengthened the balance sheet, our fourth key strategic priority in the third quarter and since the beginning of the year since support both, our commercial launch as well as the advancement of our pipeline. And finally our fifth strategic priority is to build out our biologics capabilities and capacity in our overall pipeline.

As part of this strategy, we acquired a new preclinical program for CDKL5 deficiency, a devastating neurologic rare disease earlier this year, and we expect to focus on our current programs for the remainder of 2016.

So with the commercial launch underway, two programs in the clinic and a biologics platform for future growth, we are executing our vision to become a leading global biotechnology company focused on making a significant impact in the lives of patients living with rare devastating diseases.

So with that introduction, let me go ahead and turn the call now over to Brad who will take us through more details on Galafold. Brad joins us from our international headquarters in London. Brad, go ahead please..

Thanks very much, John, and good morning everybody. I'll begin on Slide 5 with the same launch metrics that we provided last quarter and all of these are as of October 31. First as John mentioned, we did have over 50 patients on reimbursed Galafold again at the end of October. These include both naïve and ERTs switch patients.

The majority are being treated on a commercial basis in Germany with the remainder treated throughout expanded access programs or EAPs. And as John mentioned, Germany is a very important benchmark for the rest of the launch and we're seeing some very early indicators of success there.

To begin, the vast majority are switch patients, and we now estimate that 20% of the eligible switch patients in Germany are now on Galafold, so great early uptake in that market. And as a reminder, all these patients and their physicians are newly experienced because we did not have any clinical sites in Germany during the development program.

And although still early days, our data show that none of these patients have discontinued or switched back the enzyme replacement therapy after starting on Galafold.

Again, while we think reporting as indicators in Germany provides the sense of our initial launch success, I would note that we do not expect to provide this country-level granularity going forward.

Turning to the rest of the international rollout, we have launched in total of three countries at a commercial basis and through EAPs and we've recently secured reimbursement in two additional countries where launch is forthcoming.

And finally, we now have 15 countries with active pricing and reimbursement discussions underway; so great progress across the European and international markets.

Turning to Slide 6, I'll comment a little bit further on the EU market which of course is an important region that represents 34% of the global Fabry market in 2015, a significant portion of which comes from EU-3 with Germany as we said before, it's the third largest Fabry market in the world with approximately 1,000 diagnosed patients, roughly half of whom are not currently treated, so significant growth opportunity there.

In France, patients continue to access reimbursed Galafold through our cohort ATU while we navigate the countries pricing and reimbursement process. And importantly, in the UK we are in midst of pricing and reimbursement discussions under the highly specialized technology or HST process.

And in October NICE issued their draft recommendation to commission or approved Galafold for patients 16 years and older with the diagnosis for Fabry disease who have an amenable mutation.

We're very pleased with this draft recommendation within the evaluation and consultation document and we continue to collaborate with NICE towards the publication of the final evaluation determination that will support reimbursement and launch in England and Wales.

Turning now to our global regulatory strategy for Migalastat which is on Slide 7; I'll begin with an update on approvals and submissions throughout these territories.

The EU approval of course opens our ability to access more than 70% of the global regulatory market in the EU member states as well as countries that allow EAPs or accepts submissions on the basis of EU approval.

One important update from the CHMP in Europe -- they've now reached at 44 new amenable mutations for the EU label which is now being updated to include a total of 313 amenable mutations, and we think this is an important validation for our precision medicine approach, as well as our broad dynamic European label where we have formal mechanism to request updates as new mutations are discovered.

In Switzerland, the therapeutic product authority, Swissmedic, has now approved Galafold with the same indication as our broad EU labels covering all amenable mutations referenced in the EU label. We've now successfully completed six additional regulatory submissions with more to come.

And over the remainder of the year we would expect additional EAPs to come online in both Europe, as well as other international territories. And finally, we're on-track for targeting the first half of 2017 for submission in Japan. And as John mentioned in his introduction, we do remain on-track as well to provide a U.S.

regulatory update by the end of this year.

So in summary, we believe the momentum for the Galafold launch, our regulatory progress with strength of our data package or European label, the unmet need remain in the Fabry base; and importantly, the quality of our international organization the relationship they are building in the field represents tremendous momentum in the launch.

Of course we remain committed to the broader Fabry community and we have a big vision to help all of the Fabry patients.

Not only will we continue our endeavor to seek regulatory approvals for Galafold, for Fabry patients with amenable mutations but for those patients with non-amenable mutations, we are designing a novel proprietary Fabry ERT that incorporates an intravenous form of Migalastat in the formulation.

We're actively working behind the scenes in this pre-clinical program so that years ahead we can offer solution to all Fabry patients driven entirely by their genotype. And with that, I'll now turn the call back over to John to provide an update on our clinical pipeline.

John?.

Great. Thank you, Brad for all those updates. Turning now to Slide 9, let me talk a little bit about three major channels again in Pompe and what we're trying to achieve here. So the first challenge is the activity and stability.

The second relates to tolerability and immunogenicity, and here we are looking to address both of these challenges with our novel treatment paradigm consisting of our proprietary enzyme replacement therapy designated in ATB200 along with the addition of our chaperons, AT2221, a specific small molecule designed to bind to and stabilize ERT, ATB200.

The third major challenge with the current ERT related to uptake and targeting and to solve for this what we've done is, our ERT ATB200 has been designed with optimized levels of mannose-6-phosphate and what we believe to be very important levels of glycosylation and the right sugar structures in the karyotyping [ph].

So today I will describe more details about our clinical study and what we are expecting to see shortly with our initial data readout by the end of this year.

So Slide 10 describes the Pompe data cascade; so here I'll highlight our ongoing clinical study designated at ATB200-02, this is an 18-week open label safety and PK study with an option of long-term extension study that is designed to establish important proof-of-concept and to differentiate our proprietary ERT product ATB200 co-administered in a fixed dose regiments with our chaperons AT2221.

All patients in cohort 1 are ambulatory ERT switch patients. During the third quarter, the data safety monitoring word reviewed the safety data from cohort 1 and cleared up to begin enrollment in cohort 2 consisting of non-ambulatory Pompe switch patients.

As well as our cohort 3 with Pompe patients who have never before received ERT or ERT naïve patients. So I'm pleased to report that we are currently treating patients in all three cohorts of this study. So what is the data sequence along these cohorts? Let me just walk you through that.

In the fourth quarter, we are on-track to report interim data in the first four ambulatory ERT switch patients in cohort 1. And then in the first half of 2017, we expect to include additional important readouts. These included additional 18-week data and extension data from cohort 1.

These data will also include 18-week data and extension data in cohorts 2 and 3. Beyond that we'll continue to generate long-term data and functional measures in the ongoing extension study.

We believe that given the complexity of this disease that the totality of the data from this study will be available by mid-next year to inform both, the dose collection and the design of our next clinical study.

So Slide 11, we talk a little bit about what we call our score card here, so never I've reviewed the study design in the major inflexion point in this study. Let's turn to Slide 11 where we've outlined the multiple ways which we think we may achieve success here.

So first very importantly in Pompe's safety; our initial question is, can we safely switch patients immediately from Lumizyme onto our drug. We are very encouraged by the safety profile thus far, especially following our positive safety monitoring board review this summer.

The next question is, can these patients tolerate our two [ph] regimen with limited infusion-associated reactions? This would be a very positive indicator that our product is distinct because more than 25% of patients on Lumizyme have been reported to have infusion-associated reactions; so something will be monitoring with our drug regimen very closely.

With respect to PK, we want to answer the question, can we demonstrate a differentiated PK profile with in and optimal range. This is also extremely important because in our pre-clinical studies, even at lower exposures, ATB200 demonstrated significantly greater glycogen reduction than equivalent doses of Lumizyme.

Also looking at the immune profile of our drug will be very important. As a reminder, 100% of patients on Lumizyme develop antibodies as has been reported and antibody titers can impact treatment outcome.

In the switch patients who already have antibodies to Lumizyme, we want to answer the question, do the antibodies increase or remain the same in the book [ph] on ATB200 and AT2221.

And later on in cohort 3 with the naïve patients, we want to be able to answer the question to know what is the onset and level of antibodies formation in patients who have never been exposed to VOT [ph]. And finally, we're also looking at a series of exploratory biomarkers as an indirect read-through and targeted uptake as well as immunogenicity.

We expect to have all data from the study by mid-2017 and we're also continuing to do preclinical studies in Pompe and will also have more to share in the coming year on some very important findings from this preclinical work.

We are actively working to get into a pivotal study as soon as possible; and in the meantime, we're scaling up manufacturing right now to the 1,000 liter scale so that we can run our next study with a commercial scale of ATB200 plus chaperon which we believe would be very important.

On Slide 13 let me discuss briefly our EB program before I hand the call over to Chip to take us through our financial results. So let me review here the Phase 3 studies which we designated SD-101 for EB and again, this is on Slide 13.

We've seen significant momentum in the enrollment in the study and the data is on-track to report in the first half of next year 2017. During the third quarter of this year we submitted our Statistical Analysis Plan or SAP to the FDA for finalization.

This SAP is based on written feedback from the dermatology division at FDA in which the agency agreed to elevate the important end point of time to wound closure as a co-primary endpoint together with our previously specified primary end point of proportion of patients with target wound closure.

So in terms of progress with this study there continues to be a high level of interest from the EB community. We are seeing a very strong uptick in enrollment, now with a total of 28 sites active in the United States, Europe and Australia. We continue to see 100% conversion of patients from the primary treatment period to the extension study.

We believe also that our strict entry criteria, as well as the overall study design now increases our likelihood success for this study as we look forward to advancing to completion into topline data. So with that, Chip, let me go ahead and turn it over to you for the financial overview..

Great. Thanks, John, and good morning, everyone. I'll start today's discussion with our financial results on Slide number 15 beginning with our third quarter revenue and this is a notable milestone as the first time we are reporting product sales in the company's history and reflective of our transition to a commercial stage company.

Total product revenue in the third quarter of 2015 was $2.1 million and as of September 30, we have made the transition to recognizing revenue on an accrual basis and expect to continue as additional countries come on.

Moving down P&L, total R&D expenses for the third quarter of 2016 increased to $32.5 million, as compared to $21 million for the third quarter of 2015.

The increase here is primarily due to one-time expense associated with the MiaMed asset purchase as well as investment in our Phase 3 EB clinical development which was not part of Amicus at this time last year.

Total selling, general, and administrative expenses for the third quarter of 2016 was $17.5 million as compared to $15.4 million into the third quarter of last year. The increase was primarily due to investment in pre-commercialization and commercialization activities related to the Galafold launch.

Net loss was $46.7 million or $0.33 per share in the third quarter of 2016 compared to the net loss $37.8 million or $0.32 per share for the third quarter of 2015. The wider loss is attributed to an increase in total operating expenses, and as of September 30, 2016, we have 142.3 million shares outstanding.

Moving onto Slide 15, few comments on our current cash position and our overall 2016 financial guidance; cash, cash equivalents and marketable securities totaled $212.4 million at September 30, 2016. As previously disclosed, we've raised an additional $39.3 million in net proceeds through the ATM equity finance facility during the third quarter.

As a reminder, we've raised the full $100 million registered under the ATM facility.

Through continued careful management of our expenses, we expect to remain on an original 2015 net cash, net guidance between $135 million and $155 million, and our current cash position including proceeds from the ATM and additional debt earlier in the year is projected to fund operations into late 2017 through several important inflexion points that John outlined previously in the call.

This summarizes our key financials for the third quarter of 2016, and additional details will be found on our Form 10-Q which will be filed later today. I'm also happy to address any questions during the Q&A but for now, I'll turn it back to John..

Great, thanks Chip. And I'll just go ahead and conclude before we turn it over to Q&A.

On Slide 18, again with just a brief snapshot of our key value drivers and upcoming milestones related to our three lead programs, plus those momentum as you've heard here, obviously very excited with the launch of Galafold in the very early days particularly in Germany, the addition of these 44 new mutations to our label, the positive draft recommendation from NICE on first review, which is quite unusual.

So in many ways, I think it was a very, very strong quarter for us. And again, all three of our lead programs in Fabry, EB, and Pompe are representing commercial opportunity of between $500 million and $1 billion in sale.

As a reminder, we fully own all of the rights to all of our products and technology platform and while there has been significant amount of interest in all of our programs from the partnering and licensing perspective either for full program and to our certain territories and while this quick provides us source of financing, we will do so in the future only if any partnerships would provide a better, faster, and more cost effective way to advance our medicine and to deliver them to patients.

I continue to believe that we have one of the strongest portfolios in the field of rare, devastating diseases and that we are on-track to achieve a very bold vision to become a leading global biotechnology company and to provide medicines that have the potential to positively impact people's lives.

So operator, with that we're happy to take questions..

[Operator Instructions] Our first question comes from Ritu Baral with Cowen & Company. Your line is now open..

Hi guys, thanks for taking the question. I did want to ask about the U.S. discussions; is the structure of discussions as you previously guided with the type E meeting around now or has it moved to an iterative sort of structure and discussions? And I have one follow-up..

Ritu, we're not going to comment on ongoing discussions -- regulatory discussions with Migalastat here in the United States; we'll only confirm that we will provide an update by the end of this quarter..

Got it, which is why I have my follow-up. Okay, so after real question about Europe then.

Can you go into the profile of the patients that you're seeing in Germany -- the profile that switch patient versus the profile of the 90s patients that you've seen so far, not well -- maybe not just in Germany? And also what sort of approximate Pan-European price we're seeing at this point?.

Yes, so two questions; one, kind of the general nature or profile of the patients -- these first 50 commercial patients that we're seeing; and second, you asked a question tailbox [ph] there at the end about pricing?.

Yes, the Pan-European price you're seeing right now?.

Brad, if I can, I'll ask you to comment on both of those..

Sure, thanks John. Ritu, I think the first point for Germany in particular is really what we made on the call previously which is that, these are all patients and physicians who have had no previous experience with Galafold as we didn't have any clinical sites there.

So clearly I think that reflects an interest in the part of physicians and patients to explore Galafold as a new treatment option.

From a profile perspective, other than being in line with our label of course, I'll say that I think it's in line with where we thought these patients would which is the mix of patients who perhaps have been dissatisfied on -- current ERT patients who have been looking for treatment options but for whatever reasons weren't willing to start an enzyme replacement therapy for the naïve patients; it's males and females.

So I think it's reflective really of the broad label that we received from EMA and we would expect that to continue in other markets as well.

On your second question around pricing, I'll say that it is -- our pricing strategy remains in line with our previously stated strategy which is that this is the first precision medicine of proof of Fabry, the first new therapy in over a decade and we have chosen our strategy to price [indiscernible] with enzyme replacement therapy on a country by country basis; and we think that reflects the value that Galafold can bring to patients and also can give some savings back to the healthcare system by avoiding the infusion-associate costs and so far that pricing strategy I think has been quite successful.

And just as a reminder, the global average price -- list price for ERT is roughly between $200,000 and $300,000 although of course that varies country by country..

On the profile are you seeing any trends in classic versus non-classic mutations?.

No. Again, I think it's reflective of the patient's -- remember, we studied both of those population in our clinical studies and they are reflected in the label and really physicians and patients are asking, do I have amenable mutation? And if so -- and if they are interested in trying Galafold then they do.

So it's not -- it hasn't been skewed so far to one part of the population or another..

Great, thanks..

Great.

Ritu, I'll just add to that -- having been in field last month for a couple of days in Germany and visiting a number of these key treatment centers, you know there were a number of skeptics prior to launch and we were quite conservative with our internal metric in this very competitive market in Germany, in terms of patients switching, in fact people told us you'll never get people off of ERT to come on this product.

And to see in the first full quarter that we already had 20% market share in Germany of amenable patients, treated amenable patients is pretty extraordinary.

And I think again, its goes to the data and the quality of our leadership team and I could tell you the divisions in Germany are extremely experienced and quite sophisticated and quite rigorous in their review of data and the care of their patients.

So again, a very important market for us to see in the early days such significant uptake I think hopefully bodes well for the product..

Our next question comes from Anupam Rama with JP Morgan. Your line is now open..

Hi this is Ucco [ph] on the call for Anupam, thanks for taking our questions.

Also 15 countries where you have reimbursement negotiations ongoing, how many do you expect will conclude in 2017? And also what additional metrics from a launch trajectory do you think you will need given the country by country rollout before you're able to provide revenue guidance?.

Yes, Brad maybe do you want to comment on what -- to the extent we can and what we may see by the end of '16? And then maybe Chip, if you want to answer the question on what metrics do we need to see before we start providing revenue guidance..

Sure.

So in terms of when specifically we expect countries to come onboard or how many we refrain from giving that level of specificity; but again I'll draw you to the metric we showed on the call which is, we do -- we have recently secured two additional reimbursements until we would expect to have to be launching them in those markets here relatively soon, and we would expect to continue to see countries coming onboard through the end of this year and through next year as well.

So we can't give more specifics than that but again, I think you'll see that role in launch continue as we continue to secure reimbursements in those markets or secure extended access where those vehicles exist..

Yes, this is Chip.

Just to comment on the guidance going forward and the kind of metrics that we're providing, we feel in these early days of launch that the patient metrics in terms of number of patients on at the end of the given month near the earnings call is the most meaningful metric in these early days of launch as we move through that rolling launch period.

As we have greater insights and clarity around when these additional countries will come on, we will incline to provide revenue guidance, we've not set a specific date for that guidance.

But as we move to revenue guidance and as revenue becomes the more meaningful and telling measure of factoring [ph] a launch, we will migrate from more patient-focused metrics to metric focused on the revenue..

Our next question comes from Joseph Schwartz with Leerink Partners. Your line is now open..

Thanks very much. So I was just wondering if -- since you've mentioned that most of the patients who are going on to Galafold in the EU are switching from the current ERT products; I was wondering if you can talk about whether you have any initiatives to penetrate the proportion of the market which is not currently treated.

And then do you have any insights on how many of those patients are actually accessible in terms of them being identified and seen by physicians and otherwise active in the community?.

Yes, Joseph I'll comment on the net spreads, we'll add a little more color, absolutely the drug as you know is labeled for patient 16 and older, both ERT naïve and ERT treated.

This is absolutely in line with our expectations although the magnitude is perhaps greater of the patient switching because these are the people by nature who are coming in very frequently to the physician site.

The naïve patients tend to come in to the key treatment center a couple of times a year; so we knew that that would be a longer cycle to begin to penetrate those 500 or so patients in Germany who are diagnosed with Fabry but not treated.

So absolutely the discussions with our commercial team and our medical team are both with respect to ERT treated and naïve; and we expect overtime to see more and more of those naïve patients.

Brad, maybe you can comment on within that pool of naïve patients, who do we believe that characteristics of patients who we believe would be suitable for treatment today..

Sure. Joe there is a natural pent-up demand in some cases where those naïve patients have one ongoing treatment but for one reason or another have not and that could be accessed or could be the burden of caring for multiple family members who are on treatment themselves as this is a typically familial disease.

It could be that they are earlier in their disease progression and haven't yet progressed to a point where they may meet the treatment criteria in their particular market or could be just as John said, that they are in a longer cycle in terms of seeing their thought leader or their physician who would make the ultimate treatment decision.

So there are a number of dynamics going on but as John said, we fully expected that and in the short-term, the low hanging fruit is -- are the patients who are on ERT, who are in the system much more actively; and in the longer term or say, perhaps the medium-term and then the longer term, it's those patients who are diagnosed and untreated.

And the only other thing I guess that would remind you of is the extended access programs that are existing today are really second-line therapy; so really, Germany is the only place where we have an ability to treat both naïve and switch patients and so we would expect that as more countries come onboard, more fully -- as we penetrate into the ERT market we will also penetrate into the diagnosed untreated market as well..

Our next question comes from Taveen Ahmad [ph] with Bank of America. Your line is now open..

Hi, good morning, thanks for taking my questions, maybe a couple for Brad.

We were happy to see that NICE as you mentioned, included your drug and draft guidance but how does it work in conversion from draft till sign-off; how long does that take? And in that period, would you be able to have any advantage in getting additional traction in the UK? And in that process do you also have to have any meetings or submit additional data for that draft to become final? And then secondly, with regards to your addition of new amenable mutations, how does that process work? Was that information that you had in-hand when you had originally filed for your approval in Europe? And if not, how do you go about submitting the request for additional mutations and is that a quick turnaround from the time that you requested to the time that's agreed upon? Thanks..

Thank you, Taveen. Brad, if you've got both of those I'll let you handle both, beginning with Taveen's question on NICE and the conversion from draft to final..

Yes, two great questions. For the NICE process, it is ongoing, so we don't want to comment too much on that process other than to say that we're pleased with how it's going so far and we're collaborating fully with that process.

What we can share is that the next step in the process is the public hearing which is scheduled for November 22, and typically they would issue their final evaluation document shortly thereafter. And so we would expect that process to conclude sometime at the end of this year or very early next year.

So we are on-track there and I'm pleased with that process so far.

As it relates to the process of mutation -- it's a great question and I think again it reflects how supportive and forward thinking the Europeans have been in terms of the way that they have embraced the notion of precision merits and remember, we did I think provide some color that the initial label which included 269 mutations -- that actually included a number of mutations that were characterized between the study and then getting the final approval.

And so we had already had an example of where they were essentially supporting the notion that it would be assay that determined amenability and once amenability was determined it could be entered into the label.

Now we've seen 44 mutations who were not part of the discussion during the negotiations for the label that have been characterized in the meantime, since we've gotten our approval.

And so the process there will be, you go through the typical regulatory submission to extend the label on Europe; once they agree to that which they have now recently done, then we update the website and it's the very seamless process but now in a position goes to look up one of these new mutations, it will be listed as amenable and on-label and then therefore they can prescribe.

On a go-forward basis, that's generally how we'll handle this.

So if we characterize the new mutation and historically it's been something like 5% or so -- new mutations are characterized each year, either the physician identifies one or we identify one ourselves, we'll characterize the assay, determine amenability and then submit it probably in some sort of batch form to the Europeans, they'll approve it and it shows up on the website.

So actually it's a pretty easy process and one that we think is really reflective of this whole concept of precision medicine..

Taveen, did that answer your question, or both of your questions?.

Our next question comes from Roy Buchanan with Janney. Your line is now open..

Hi, great, thanks for taking the question.

I just had one on John's comment towards the end of the call to make sure I'm clear about the partnering; is it -- I guess kind of planned only finance in the future through partnering?.

No, we'll look at a range of finance options again with whatever we think is in shareholder's interest to minimize any future dilution.

I just wanted to make the point that obviously with a commercial product now with significant potential in the rare disease and a very robust pipeline, we've had a number of authorities reach out to us with ideas and proposals for potentials for partnering either whole geographies or entire programs.

We are nowhere near doing a deal necessarily but I just wanted people to know going forward, potentially we would consider that. So the bar is going to be very, very high..

Our next question comes from Mike Yeolf [ph] with Robert W. Baird. Your line is now open..

Thanks for taking the question. Just -- on the Pompe program, you're expecting sort of proof of principal data by mid-next year.

Is the current plan there to go forward in a larger Phase 2 or is it to go directly into a Phase 3? And then secondly, was filing in Japan targeted for the first half of next year? Can you maybe comment on the next steps there to meet that goal? Thanks..

Sure. So to remind everybody, with Pompe we will see the initial data here in the next month or so. By the end of this year, again -- those are the first four patients in cohort 1 and that will contain data that we think will help establish very important early proof of principle.

But the proof of principle -- we've always believed in this complex disease, it's going to come across all three of these cohorts and across the series of data releases from Q4 of this year culminating in about mid-2017. So that's what to expect on a range of outcomes.

With respect to the next study, we've not yet meet with regulators to discuss what the next study is. Our hope is that the data may provide the strength to allow us to come to a pivotal study that's yet to be determined.

With respect to Japan, I'll just comment that we are right now -- officially [ph] -- our regulatory teams and consultants procuring the Japanese NDA and that remains on-track to be filed in the first half of this year.

Again, if you remember the middle of this year, the Japanese regulatory authorities indicated that we did not have to do any further clinical studies in Japan or anywhere and that we've made move forward now with the filing of the Japanese NDA which again accelerated our timeline by about three years to approval in Japan..

[Operator Instructions] Our next question comes from Salvin Richard with Goldman Sachs. Your line is now open..

Hi, this is actually Tom on for Salvin. Thanks for taking the question. I just had a follow-up on the Pompe program; so in the interim readout in the next month or so as you mentioned.

Could you just go into little more detail on what's from the biomarkers MITR? And then since this is -- these first four patients are Lumizyme switch, would we see a comparison to baseline on Lumizyme in those patients? Thanks..

Yes, with respect to the biomarkers, there are a number of traditional biomarkers that we're going to be looking at here. Again, very early in terms of the number of patients, you would be looking at things like Hex4 in the year end, they are PK/PTK measures in muscle, surrogates for potential improvements in muscle strength.

Again in very early studies in a handful of patients, these are exploratory, we're not sure what could be -- I'll remind everybody that we have not seen any of this data other than the safety data that we've reported through the DSMB although see it here very, very shortly.

So I think that's important to note with respect to the biomarkers and what to see. And Tom, I'm sorry, remind me the second part of your question..

And I'm showing no further questions. I will now like to turn the call back over to John..

No operator, I just -- the gentlemen from Goldman Sachs, the analyst Tom, I just want to make sure I covered his questions..

And Tom, do you mind us queuing back up again? All right Tom, your line is now open..

Yes, sorry about that.

Yes, so the question was since these are patient switching's from Lumizyme to your drug, would we see a comparison of their baseline measurements in these biomarkers to their biomarkers on your drug?.

Yes, we will..

Okay, great. Thanks for the questions..

I'm showing no further questions. I will now like to turn the call back over to John Crowley for any further remarks..

Great, thank you operator and everybody, it was a very strong third quarter for us. And again, we begun to deliver on some of the important data, particularly with respect to successful launch of Galafold in the very early days in Europe. And more data to come including the U.S.

regulatory update, the Pompe data, and as we close the finalization event, important statistical analysis planned revision in our Phase 3 EB program. So more news ahead of this as we're building into a very strong successful 2017. So thanks everybody for listening. Have a great day..

Ladies and gentlemen, thank you for participating in today's conference. You may all disconnect. Everyone have a great day..