[WEBINAR HOST]
So for people with hemophilia A (severe), they’re really not able to be spontaneous, right? They generally have to think about what they’re doing and when they need to infuse their prophylaxis if what they’re going to do is have a bleed. So, it really just is thinking about ROCTAVIAN as an option and how that could change your outlook on what your activity looks like.
So of course, we would be remiss without doing a lot of safety information and some overviews throughout this 5-year data, but ROCTAVIAN is a one-time gene therapy. It’s used for the treatment of adults with severe hemophilia A. As I mentioned, they do use a modified virus called a vector and that’s an adeno-associated virus, the AAV5, to deliver a working copy of the Factor VIII gene.
So, this isn’t changing your DNA or mutation. You do still have that hemophilia mutation. But what we hope happens is that that copy of the working gene will allow your body to produce clotting factor on its own.
Reasons not to take ROCTAVIAN: We did talk about the infections already, but then notably, because this is going to your liver, we would want to make sure that your liver is in good health and that you are also not allergic to mannitol, which is an inactive ingredient in ROCTAVIAN.
So, you can see Andrew S. here. You know that since his ROCTAVIAN infusion that he’s not had to do his prophylaxis, and we’ll get into that as well. But ROCTAVIAN in and of itself—one-time infusion that was backed by the longest and largest Phase 3 gene therapy clinical trial in severe hemophilia A patients.
So as part of that clinical trial, there were 134 individuals with severe hemophilia A who did receive ROCTAVIAN as a one-time infusion. So, looking at that clinical trial data, we can see that after 5 years of having ROCTAVIAN infused, that the majority of patients, their Factor VIII levels are still at about 30 percent, which you know is mild hemophilia range.
You can see that levels do go up initially and then do seem to decline quickly. However, what the 5-year data shows us, which is wonderful, is that those levels really stay kind of steady state. So, you know, there’s always those questions about how long will it last and we’re seeing that that efficacy is lasting quite a while.
So, like I said, majority of patients with severe hemophilia did maintain those mild ranges. So, 84 percent of patients were able to stay in that mild range, meaning their own body is making factor vs having to infuse prophylactically.
[WEBINAR HOST]
So, this is one of the other Andrews that you’ll hear from tonight, Andrew W. So, he and Andrew S. are actually 2 patients in the clinical trial. They received their dose several years ago. And you know, I won’t read all this because he’ll tell you his story. But really just again, thinking about what’s that impact to a patient, to you as a patient, if you could make your own Factor VIII.
One thing to cover, of course, is that ROCTAVIAN doesn’t work for everybody. It’s as with all treatments in this space, right? We see that some people respond better to other things and we’re not always sure as to why that is. So just to note, if this is something you’re interested in, having that discussion with your healthcare provider.
Now additionally, beyond not having to do prophylaxis, we want to know were patients having bleeds, right? And what we did see is that before patients were infused with ROCTAVIAN, that annual bleed rate or ABR was about 3.
And remember these were patients that were doing prophylaxis prior to, and then the 5- year data does show us that there was an 88 percent reduction in that ABR number to less than 1 bleeds with patients after they received ROCTAVIAN.
So really what that looks like is patients, if you think about someone with severe hemophilia A and of course this study was, or clinical trial, was started before patients were on maybe a non-factor therapy.
Many of them had to do at least 3 times weekly infusions, right? So, what that looks like over a year and I’m sure that any of these gentlemen can speak to that the number of syringes or infusions, right, in a year is 136—thinking about that impact to your life and how much that really… all the time spent on that.
And that’s only prophylaxis, right, that didn’t even take into account bleeds. So, looking at after ROCTAVIAN, 95 percent reduction again with the patients in the clinical trial, average infusions declined to about 7 per year.
And this is taking into consideration patients that maybe didn’t have as high of a response, or their response was lost a little more quickly.
So still, that average level is pretty incredible when you see that picture. In addition to that, right, we always talk about with our patients what their quality of life looks like. And especially with hemophilia, while I don’t have hemophilia or a bleeding disorder myself, I see it in patients, I see it in my stepson and that anxiety that it causes patients to have. And then of course, what the consequences of having a bleed on their joints or other bleeds, right? We’ve had patients with really severe bleeds, and just what that does to them and how they’re able to have to live with that and be a part of their family and their society, and work, and want to do things with their kids and everything like that.
[WEBINAR HOST]
So, part of the study was, the hemophilia quality of life questionnaire was administered to patients, and then looked at after 5 years, what did that change for them? And we can see that before ROCTAVIAN, their quality of life was certainly a lower score than what it was 5 years after ROCTAVIAN.
And we can see like even from getting infused at that 1 year to the 5 years, despite knowing that levels may have declined a little bit, that quality of life is still at a higher score, around 82 percent. So I think that really speaks volumes to what patients have experienced.
And of course, we can’t have every single one of them on the webinar tonight, right, to tell you, but I think it really will go in line with what you hear from Dave and both the Andrews tonight.
So again, Andrew, W., who you’re who you will hear from. Just one of the reasons he volunteered for the trial was to stop his continuous prophylaxis, right? Not having to worry about getting up and doing his infusion before he got his day started.
So of course, some safety data that we’ll discuss: 134 people enrolled in the clinical trial. We’ve got 5 years of data and of course long-term safety will continue to be studied. This data will be collected for years and years to come. But right now, we can just speak to what we know from the 5-year data.
So, some things that your provider should be talking to you about, your healthcare team before you get ROCTAVIAN, it’s just really what are what are things that we have to monitor for afterwards?
What are things that could happen during your infusion? And some maybe changes that you’ll need to make. So first of all, you know we do have to make sure that your liver is healthy. And this is done with some lab work and some imaging, such as an ultrasound.
So, it’s depending on what your liver looks like if you’re eligible. Of course, we still want to make sure that afterwards your liver continues to be healthy. So, there are blood tests that are obtained once a week or more depending on your healthcare provider and the discussion you have with them.
But based on your liver health and if your liver enzymes do start to go up, you may start a temporary dose of steroids. Again, something you’ll talk to your doctor about.
[WEBINAR HOST]
Now during the infusion itself, this should be administered where there are medical professionals that understand not only ROCTAVIAN, but what to do in the event of an infusion-related reaction. So, I know these are common with any medication that we infuse via IV and something that, you know, what we prepare for as a team, whether we’re doing any infusions or gene therapy.
But some symptoms that could occur, you know, maybe itching, rashes, sneezing, feeling like you’re short of breath, back pain, nausea, low blood pressure, those types of things. So of course, if during your infusion you were to experience this, you would want to tell your team that’s there immediately.
Otherwise, some common reported symptoms really kind of flu-like illness have been reported in the days following the administration, and again something your team can help you with, if you were to experience them.
Now something that we talk about is, hypothetically there is that risk of liver cancer because of the fact that ROCTAVIAN can insert itself into human body cells. Now we have not had any reported cases of cancer by any of the patients that went through the clinical trial, or even after that, right? So, just something that we want to make sure all patients are aware of in that your doctor might have you do regular liver ultrasounds or different blood tests, on a yearly basis, a couple times a year just to make sure that there’s no development of that.
So, kind of something I already talked about, most common side effects, nausea, vomiting, fatigue, headache, your liver enzymes that could go up. About 83 percent of the patients did experience some ALT elevations. And again, that’s just something that each individual patient and their healthcare team will decide if that does happen, what the next step will be, if it’s a steroid taper, or further labs, or anything like that.
So, in the clinical trial studies, there have been zero people that developed inhibitors to Factor VIII. Patients that have active Factor VIII inhibitors are not able to get ROCTAVIAN, but there are patients that have had a history of inhibitors and certainly we have not seen any development to inhibitors.
Zero people developed blood clots. Again, kind of hypothetically, we do know that patients that have a Factor VIII level over 150 percent over 200 percent could be at a theoretical risk of clotting, which is something really different, right, for our patients. But again, some patients did experience high levels of Factor VIII. However, even with those high levels, we did not see any blood clots and then zero people experience treatment-related deaths.
[WEBINAR HOST]
So again, even though there are patients that did experience, you know, some mild- infusion related symptoms, nobody had a severe reaction or any other complications following ROCTAVIAN.
So if ROCTAVIAN is something that you’re thinking of wanting to do, or you have patients that are interested in it, there are eligibility testing steps that need to be taken, such as looking for preexisting antibodies to that AAV virus because as I mentioned, the copy of the working gene gets packed in that viral shell. The replicating virus gets taken out, right? So, it doesn’t cause a virus in you, but because you’re getting that capsid, your body, if you have antibodies already, could fight against it and then ROCTAVIAN wouldn’t be able to work for you. So that is something that healthcare providers will generally have as the first step. But again, if you’re interested, connect with your healthcare team, just start that process.
So, this is Dave. He’ll talk about his one infusion of ROCTAVIAN really kept him from having to do continuous prophylaxis for, well over a year. So again, I’m not going to steal his thunder, but just again, to really reiterate that it is truly possible to be a patient with severe hemophilia A and not have to do prophylaxis after getting ROCTAVIAN.
Something that BioMarin does offer is a program called RareConnections™. It is a team of experts that can help both patients and the healthcare staff to navigate the ROCTAVIAN journey.
A lot of people do ask about the expense of ROCTAVIAN. Is it going to be covered? Will my insurance pay for it? Is there an out-of-pocket cost, co-pays, things like that? The RareConnections™ team can truly help with all of those questions and help look at, you know, whether it’s benefits investigation, what your insurance policies are, things like that. So, there are, you know, a lot of insurance plans, including Medicaid and Medicare plans that do cover the cost of ROCTAVIAN gene therapy. So as a patient, don’t think that there’s no way your insurance will pay for it, it’s not possible. There are a lot of people who are out there to help get patients ROCTAVIAN if that is something that they feel is right for them and would like to pursue that.
So again, a few safety things; we’ve really already covered these. We want to make sure patients don’t have preexisting antibodies to the virus because again, it would cause ROCTAVIAN not to work for you.
Anyone with inhibitors, active infections, especially to the liver or otherwise, again would not be candidates because we need a healthy liver so that the ROCTAVIAN copies can, or ROCTAVIAN can be delivered to your liver and start making Factor VIII copies.
[WEBINAR HOST]
Hypothetically it could insert itself into DNA, human body cells, so you may have extra testing to undergo to watch for any theoretical risk of cancer.
Your provider will talk to you about needing to come back for routine lab work and how frequently that will occur, and for how long, and then what they will do if you do have elevated liver enzymes.
Again, you just want to talk to your doctor about any other preexisting conditions that you might have that may not be hemophilia-related, but your provider may not know about.
Of course, again, because we want your liver to be healthy and we want your liver to stay healthy, you will likely be asked to make some lifestyle changes, potentially including not using alcohol for at least a year after getting ROCTAVIAN. Simply again, because we know alcohol can be hard on your liver, especially if you have more than a few drinks in a sitting. So that’s something you will be asked to do.
And then we would also ask that any new medications that you start after receiving ROCTAVIAN that you talk to your provider about because many supplements, medications can be hard on your liver as well. So that’s just a discussion to always have with them.
Of course, if you are with a female partner and you want to become pregnant, we would ask that you wait for at least 6 months. So that’s something they will also talk to you about. And then of course, making sure that if you have any side effects after getting ROCTAVIAN that you report those as well.
Again, ROCTAVIAN’s a one-time treatment. There are no other treatments at this point for hemophilia A gene therapy. But it does not mean if others come out in the future, you’re not eligible for those. And really that just comes down to the virus that is delivered because you will create antibodies after receiving ROCTAVIAN to the AAV virus, right? So that is the reason for that.
[DAVE N.]
A big part of getting to the day where we all received ROCTAVIAN was kind of what led us there. And by that, I kind of mean the treatments that we were all on that led us to the point where we all thought that ROCTAVIAN was going to be something that we were all interested in. So, I’d be super curious to hear from the Andrews, but my experience growing up with hemophilia: I was born in the late ’80s, got hemophilia at birth, genetic mutation. And as a kid I developed an inhibitor to Factor VIII. So, I was on a lot of Factor VII medications my entire life, which didn’t always work the best. Eventually later in life, as I matured, I decided to get rid of my inhibitor and started going on prophylaxis, 3-day-a-week treatments, probably similar to you guys as well. And it was just—we do it, right? Getting stuck with needles is just something that becomes a part of life. But even from a really young age, I wanted more. I wanted something better.
So, after the prophylaxis, I went to monthly stomach injections and that was good for the first couple years. And then I was like, well, I don’t like sticking myself in the stomach that much. I want something more. And luckily around that time, my doctor talked to me about ROCTAVIAN, which was the next kind of step in the progression of hemophilia treatments. And that’s what led me to taking it. So, I think it’s important to know about the journeys and what we’ve each been through that led us to this decision. And that’s my story.
Andrew W., I’d be interested to hear where you started and what led you to ROCTAVIAN.
[ANDREW W.]
Yeah, absolutely. So, I got kind of lucky early on. I guess it’s a little more backstory than we really need, but my caretender nurse, you know, who took care of me as a little kid is now my person at my hemophilia treatment center. So that’s such a connection there.
Even from the ’90s, you would hear gene therapy and that was always a hope. And she called me personally and said, there’s a clinical trial. Would you be interested? As soon as I heard gene therapy, I was like, I’ll be right down there. Let’s talk about it. And, you know, it was because immediately I called my dad too.
I mean, at that point I kind of joked about it because—huge sports family and again, spontaneous genetic mutation. So, they knew what to do with me, but they didn’t. Baseball, football, I don’t know, but I was, it’s almost like, baseball? And he’s like, well, you’re 30, you know, 27, little league’s a couple years past.
But it was definitely—I got right down there to talk about it. And then, once I got to talk with the doctors and the family, it just seemed right for me. Andrew Schultz. What was what it like in that?
[ANDREW S.]
Yeah, I’ll add, for me, I think I experienced hemophilia as a ceiling, sort of a boundary for my activity. And how I pack for a trip and so many logistic things revolved around this disorder. And so, I know. But I will say I was stable. I had a treatment regimen. You know, when I did travel, I had a letter I would wave, you know, saying it’s OK for me to travel with these needles and strange vials of liquid. And so, I had it all under control.
And yet when I heard about gene therapy and we started doing research, we’d be remiss not to say, but these are our decisions. Any decision that you’re going to make should be a decision you make with like me, my nurse and my doctor, and my brother, and my family, and you know, anybody—Google, anybody I could find.
But when I started to learn about gene therapy, I wondered, “Man, I wonder if there’s, something different.” I was stable and I had a plan, a path. And yet this was presented to me and, and it felt like, man, maybe this could be different from the treatments I did that added 12 hours to the half-life or you know what? It, whatever it was that came beforehand.
Then, you know, spoiler alert, since I said yes, I found that that yes, it was, it met that expectation quite a bit.
[DAVE N.]
I think that it’s interesting to hear because, we’ve all kind of had the same mindset, I think when it came to when we heard about ROCTAVIAN.
Obviously, you’ve got to talk to the people that are important to you in your life, your doctors, your nurses. But I think we all kind of had the same reaction. You know, we’re like, yeah. And this is, we all started somewhere. We were either treating on-demand, prophylaxis, monthly stomach injections. This was just the next natural step in our in our treatment regimen, so to speak.
So, I just find it very interesting that we all kind of were like, yeah, well, let’s do it. You know, even Andrew W., you said, you know, your nurse called you up and you’re like, I’m on my way down there right now. I mean, I had, I kind of had the same reaction too. You know, my doctor told me about it, and I was like, yeah, let’s, let’s do it, you know.
So, I think that’s really cool that we all kind of shared that same experience. And I think a lot of people listening to whether you’re a hemophiliac or you know, somebody who has hemophilia, I think if you really think about it, that’s what we all want. That’s what we’re all looking for. And that is, you know, what ROCTAVIAN has provided us so far on our journey.
Ǫuestions and concerns that you guys might have had, you know, when it came to ROCTAVIAN? I mean, I know we heard about all the, you know, it’s, it’s new. We don’t know what the future brings. We’re all kind of, we’re taking a chance at a lifestyle change, which has obviously worked out pretty good for the three of us so far. But what other questions and concerns did you guys have? Because I was, you know, kind of open to doing it.
[ANDREW S.]
Yeah, I will say for me, you know, the trade-offs that Andrew shared. No alcohol for a year, don’t have a kid for 6 months. Visiting… visits to the doctor felt daunting at first when I first heard them. Man, you know, what’s it, what’s it going to be like? What’s my life going to be like with these additional hurdles? And, and I think the concern for me was, will it be worth it? Kind of like I said, I, I felt like, well, I have a stable thing, you know, can it actually be better? Can it actually be different?
And so, as I talked to my doctor that was, that was the question for me is, you know, is the trade-off. Hey, was this, you know, you know, is, are these sacrifices that I will make going to be worth the potential upside? And, and, and I think to, to address those questions, it really was, you know, primarily for me, my nurse Julie, who just… we texted a lot… we had a lot of conversations and, and ultimately, as I understood more about gene therapy and what it is and what it can do and, and then encountered more and more people who had positive things to say, shared the research and, and things like that. Those were the folks that that gave me confidence to say yes.
[WEBINAR HOST]
So kind of along those lines guys, as you’re talking about all of this and you know, just everything that went into your decision making, I we did have saw a question come in that, you know, this seemed like a good time to answer and it is the question is liver function tested weekly for how many weeks post infusion? So, I can answer that. Per the ROCTAVIAN package insert label, it is advised to do weekly Factor VIII and liver enzyme testing and… for 26 weeks. So, for the first 6 months and then after that it is advised to check labs one or every 1 to 2 weeks for the next 6 months. Of course, like I mentioned before, everyone’s healthcare provider will be a little bit different, right? They may ask for maybe twice a week or if something is doesn’t look right, come back. They may be fine with doing every two weeks, but that’s something you’ll talk to them about. But that’s just kind of what it looks like.
I think one thing I will say that I’ve noticed and it generally… you can correct me if I’m wrong, but patients really want to know their Factor VIII level, right? They want to know is it working and is it going up. So certainly, I think it probably looked a little bit different for you, Dave, than it did for the Andrews because clinical trials are a little bit more rigorous and that they’re more structured, right? You’re not able to just come in on your lunch break, get your labs drawn and you’re good, right? But you know, I guess was that a huge deciding factor for you to do ROCTAVIAN when it came to like knowing you’d have to come every week and every other week for that long… and it we only have a couple minutes.
[DAVE N.]
Yeah, I can answer that kind of quick. For me, no. You know, if you’re telling me before this all started that for a certain amount of time I would have to get stuck weekly. I mean, come on, we’re hemophiliacs here. You know, we get stuck all the time. What are you… What are you, what are you talking about? So, for me, it was an easy decision. You know, that was probably the easiest part of it was, was the draws and stuff like that.
But it, you know, for me being after the test group and kind of knowing what some of the numbers were at that point, it was just exciting for me to see what my factor levels were going to be, you know, because I was comparing it to you guys in your first year. And I was like, oh man, I can’t wait to, to, to see what’s going on. And obviously I had great results and, and I was very happy. But I mean, that was a big motivating factor to keep going with all that stuff because I just wanted to see where I was at. I knew everybody at my center also was pretty excited to see where I was at as well.
[WEBINAR HOST]
Yes, we were. And how about for you guys real quick, I mean part of the clinical trial, right, you’re going into it knowing exactly the protocol you’ve got to follow in it and have to travel and things maybe, but obviously not a deterring factor. But did that weigh into your decision a lot?
[ANDREW W.]
Like [Norman cheers] at my hemophilia treatment center, like near the parking garage guy, the door guy, like, you know, he’s doing it twice a week. But you know, you know, it’s something you think about. But, you know, after they would, you know, all these tubes of blood. And I was like, I want my results are the Factor VIII. I’m sure everything else is important. That’s your all’s numbers and a little tedious, but I’m like, what’s the Factor VIII at this week? And next in the family group chat. And then you got the nurses and the doctors coming in and we’re looking at it. And once it goes from, you know, less than one to something you can measure, I’m like you’re ready to you’re ready to get in there and get those results. So, you know, it can be tedious, but you will you want to get that number. So that, that definitely helps seeing that, that Factor VIII, hopefully, you know, going up.
[ANDREW S.]
Even as, as trial participants, we like you said, Andrea, we had a little bit more rigorous, we couldn’t do any visits at home for the first several years. And, and so I think that’s an option. Not every one of these visits has to be these weekly visits at the hospital.
And so, I drove an hour and a half once a week for, you know, every week for forever. And yet I never had an ounce of dread heading there because like you, like you said, like when you, when you hear your level and, and even just to, to get time with your, your doc, with your, your team and like celebrate that stuff together and dig in and figure out, okay, now you know, my level being 2 percent more, what does that mean for my life? I think yeah, it was. It was the highlight of my week.
[ANDREW W.]
I got through the, you know, all the stuff we needed to test for. They confirmed that I had a beard. I think that could be a requirement too.
[DAVE N.]
Totally. Beard and glasses.
[ANDREW W.]
No. But they were like, you know, you’re good. We’re going to get you an infusion date. And I just, they kind of kept. I was, I was waiting every day and thinking it’d be some notice. And they called me like, actually, we could get this for you tomorrow in Michigan. I was like, oh, let’s, let’s go.
And you know, I’m really, it had, you know, a couple weeks to kind of even hype this up in my mind even more and you know, I’m ready for a head up there. Flew instead of drove. It’s like a 5-hour drive… and delayed. It ended up being like a 7-hour flight. So that was kind of a funny story there. But, you know, other than the length of time, you know, I was almost waiting for some prophetic thing to happen that I’d hyped up in my mind, you know, it was very similar to the infusions I’ve been giving myself, you know, two to three times a week for for years. You know, hung around for several hours just to be monitored for any, you know, adverse infusion-related side effects. But, you know, thankfully for me, nothing to nothing to report.
And then, you know, headed back home to my hemophilia treatment center for those regular follow-ups that that lessen a little over time. Thankfully I didn’t have a 90-minute commute, but yeah, just headed back home to be monitored at the HTC closest to me.
And yeah, what was that? A big moment kind of at the time was like, you know, this is it. And it was a little anticlimactic for me. And that could be different for everybody, but hopefully it’s a similar experience to that. What about you guys?
[ANDREW S.]
Yeah, I’ll say I had, I had a similar experience the “Oh easy, you know, it’s done.” You know, received an infusion. It was a longer infusion. I think it took an hour or so and and then they, they monitored me and, and we went. For me afterward, I had some flu-like symptoms for a couple of weeks and that was the only the only side effect after.
[ANDREW S.]
And so, you know, I remember beforehand, yes, similarly feeling just all this anticipation and then and then afterward it was kind of a nonevent, you know a thing, I checked it off the list. Had had a few flu-like symptoms that went away and and then was the fun part, you know, watching my factor levels rise.
[DAVE N.]
Yeah. So, I think I could definitely relate to the feelings that you had as far as side effects I think I just had a headache for a couple days. But you know, I feel like I was super, super fortunate when it came to my treatment. (A), again, having you guys who came before me and (B), my hemophilia center. And Andrea alluded to this, she was there, you know, I mean, so I had somebody who in my corner who knows their stuff, who I’ve felt super confident with my entire time at my hemophilia center.
So, I got there, I think everybody at my center was there, whether they were supposed to be working or not. I had a bunch of people in the room. I sat in a nice big comfy chair. I talked to everybody for like 8 hours and I was out of there too.
I mean, I can kind of relate to you guys to where all of a sudden it was over and I was like, “Oh, okay, well,” and you know, you don’t know what to expect when it comes to something like this, but it’s nice that it was simple, simple for us as patients. Obviously, I’m sure everybody behind the scenes was, was stressing out and stuff like that. But yeah, a very, very easy process I think for us to go through.
I think the most stressful part about it was passing all the tests that we had to, to go through beforehand, you know, you know, not having the, the vector or immunities to the vector and stuff like that. And I, I was kind of sweating that because I know that could be a common thing. But luckily, you know, I got rid of my inhibitor at a younger age. So, I was cleared of all that and it, it went super smooth. I mean, I it’s, it’s hard to say more than that.
[WEBINAR HOST]
Yeah, that’s awesome listening to your stories. And and yes, every single time we’ve done multiple gene therapy ROCTAVIAN infusions and also for hemophilia B. And so, we always say it’s like literally the most exciting, most anticlimatic day ever, but there’s still like so much excitement, right? So anyway, thank you for sharing.
[ANDREW S.]
Yeah, I, I will, I will begin by sharing, you know, like I said, if I can take you back to that moment 7 years ago, October, I think it was this month. I, oh, it’s 2 days from now. That’s or no, tomorrow is my my [hemiversary], we call it, but my infusion was October 23rd… I, I came home the next day and got, you know, started these regular weekly visits and, and for the first couple months, I think about a month and a half, we just were mainly watching, you know, okay, we got a little, little bit of a factor level going up, making sure the liver is okay. And then at about a month and a half, I remember my doctor saying, “Hey, we can try going off prophylaxis.” And, and that was such a significant moment because for me, prophylaxis since I was 10, 21 years then had been my constant companion. And, and so life after ROCTAVIAN then, you know, kind of started in that moment.
We went off prophylaxis. We monitor, you know, I, I just like texted with my, my nurse probably every day that first week because I wasn’t sure if it was going to work. You know, I was… my level was getting pretty high and we were feeling confident. We’d really talked a lot. But the, you know, the, the proof in the pudding of not having factor it just, it just felt profound. But after the first couple weeks, I remember I, I didn’t get any shots, and I didn’t get any bleeds. And in fact, for me in 7 years, I’ve not received prophylactic shot. So, you know that that experience then starting, you know, a month and a half in has just carried forward. And so, you know, I love the graphic of that pile of needles. You know, that was that was my life, this pile of needles was my life. And then this one shot in North Carolina 7 years ago then changed that. And and suddenly you like I said, kind of hemophilia was the ceiling on me growing up. Suddenly this, this ceiling felt redefined.
No, no longer am I kind of a slave to monitoring my, my factor level and making all these plans for when I’m going out of town or, or you know what, all the logistics of, of administering factor and the worry of wondering what my levels at today. I mean, should I get a shot early? We’re going to go play kickball and I don’t know. And so, I think then, you know, for me, if I, if I had a couple words this year, it’d be, it’d be freedom from prophy. Life after ROCTAVIAN has meant freedom from prophylaxis and security knowing my factor levels are steady.
That that graph we saw of the factor level kind of peaking and then and then troughing knowing that where that level is at and kind of knowing my number has allowed me to to just yeah, have peace of mind, security. And then and then convenience, you know, such, such convenience to travel without shots to not have to spend all the time I did in worrying about how am I going to treat, you know, breakthrough bleeds or, or treat my hemophilia.
I’m curious. Okay, I talked a long time, Andrew and and Dave, I’m curious for you, life after ROCTAVIAN, do you have a word or maybe even an image that you think kind of exemplifies what life has been like?
[DAVE N.]
You know, I don’t want to cop out of an answer or something like that, but, I mean, I could have said what you said word for word.
[ANDREW S.]
Well, you can’t. You have to say something different.
[DAVE N.]
Yeah. Well, I just, I I find that so interesting because, you know, it’s not often, not for me at least. Growing up, I moved around a lot, but I didn’t have, I never had, you know, I never talked to a lot of other hemophiliacs. So, my journey and my experiences were always my own. And hearing you guys kind of saying say the same thing or similar things there’s it is pretty incredible to hear. But yeah, I mean, I completely agree with everything you were saying. You know, a year and a half ago is when I took my treatment and I also have had no shots since then.
Now, that’s not to say that I haven’t had or felt some bleeds coming on because, you know, we’re still hemophiliacs. We still, you know, it can get bleeds or get bleeds. But my aha moment, I think of this entire experience after was when I felt the telltale signs of an elbow bleed coming on. And I think everybody or that’s a hemophiliac kind of knows that feeling. And I was like, okay, I’m going to I iced it and I elevated it when I slept that night. And I woke up the next morning, and it was completely gone. And my elbow was back to normal. And I was like, okay, this is this is what I signed up for. And I think it was in that moment that, you know, it really became real for me or my my life after ROCTAVIAN like came to life because I knew in situations like that ice and elevation is enough now. You know, what’s that like? But that was just an aha moment for me afterwards. But again, the freedom from prophylaxis, not having to worry about what you’re doing. I’m going on a trip; I need to pack medicine. I’m going on an airplane; I need to, Andrew, you were saying this before I got to hand this letter to somebody. It, it, it really is not on my mind anymore like it was before. You know, I don’t think about I’m doing this, also hemophilia. I’m going here, also hemophilia. It’s I’m going to go do this, I’m going to go over there. And it it’s just a a really good feeling that it’s not front and center in my mind anymore.
[ANDREW S.]
Yeah, that’s great. How about you, Andrew?
[ANDREW W.]
You know, count a lot of similar stuff there, but you know, it was, it was weird. I held my emergency factor very closely. You know, 2 weeks out. It’s like really need to infuse and I’m like, that’s different. And I started that, that kind of ceiling that you talked about, I didn’t really realize it was there until after I don’t think. Like I, I got to the point where like, you know, my nephew would be like, you want to go throw a ball? And I’m like, probably not today. And I kind of didn’t realize how much I’d kind of lowered my activity level through the years to kind of make it easier managing my hemophilia and just the, the spontaneous activities. You know, we, we took a bunch of trips.
My my aha moment was a couple weeks, you know, of no infusions, I cut myself really bad kind of line of the beard up, running late. I’m heading to get factored, it clots before I get to the other room. And I was like that that’s never happened.
[DAVE N.]
Awesome.
[ANDREW W.]
And, you know, I kept that close. But really just that, you know, my nephew was living with me at the time and just loves throwing football. And the biggest thing for me was just if he gets off the bus, you know, I could throw football. Like when did I infuse last? And, and little things like that really, really were profound for me.
[DAVE N.]
No, that’s awesome. I think I like your word usage of the spontaneity. And then when you’re talking about that medicine and stuff like that, that actually reminded me that, you know, obviously we still have medicine and stuff like that that we keep. But I remember that the medicine that I had after I took ROCTAVIAN actually went out of date in my fridge. I had to turn it in, and I was like, oh, is that a thing? I didn’t realize that could happen.
[ANDREW S.]
I will say just one more thing I had not seen in this presentation yet until Andrea showed it. The, the 7 percent bump for quality of life. And I was just thinking, it’s so funny. It’s just a slide. Look at this quality of life bumped up 7 percent. But I experienced that and, and you, you know, talking about spontaneity, like who, who would have ever thought a hemophiliac would describe themselves as spontaneous? But agreed. I, I feel like that 7 percent bump for me, which I think I went, I went from like 70s to 90, but that 20 percent bump for me was, was spontaneity, was peace of mind. You know, when I’m, I just coached my son’s football team. Awesome! And, and the freedom to choose that was, was, I mean, at least in in part, brought on by just the freedom from prophylaxis, the freedom from constantly wondering where I’m at; I’m a hemophiliac, where are my levels at; when did, when did I infuse last? And so, man, yeah, what a profound 7 percent that I think we all experience.
[ANDREW S.]
And hopefully, if you’re here, if you’re watching, if you’re here you can experience that from us. We’re just guys who have, I think, made a decision that we value enough to come to a webinar like this and share about.
[NARRATOR]
Indication and Important Safety Information What is ROCTAVIAN?
ROCTAVIAN is a one-time gene therapy used for the treatment of adults with severe hemophilia A who do not have antibodies to the virus, AAV5 which is determined by a blood test. ROCTAVIAN uses a modified virus, called a vector, to deliver a working copy of the Factor VIII gene to liver cells to enable your body to produce clotting factor on its own, which helps the blood to clot and prevents or reduces the occurrence of bleeding. The modified virus does not contain viral DNA and does not cause disease in humans.
Do not take ROCTAVIAN if you:
What is the most important information I should know about ROCTAVIAN?
ROCTAVIAN may cause serious side effects during the infusion and afterward:
What should I tell my doctor before I get ROCTAVIAN?
Talk to your doctor about the following:
What should I avoid after taking ROCTAVIAN?
What are the possible side effects of ROCTAVIAN?
What other information should I know before getting ROCTAVIAN?
Talk to your doctor about the potential risks and benefits of ROCTAVIAN. Whether a patient experiences a benefit or not, the risks discussed here and with your doctor still apply.
These are not all the possible side effects of ROCTAVIAN. Talk to your doctor for medical advice about side effects. You may report side effects to BioMarin Pharmaceutical Inc. at 1-866-906-6100 or FDA at 1-800-FDA-1088.
Please see additional safety information in the Prescribing Information and Patient Information at ROCTAVIAN.com.
*Webinar transcript contains voice-over only and does not reflect onscreen text content.
ROCTAVIAN® is a registered trademark of BioMarin Pharmaceutical Inc. © 2025 BioMarin Pharmaceutical Inc.
All rights reserved. COM-RC-0127 12/25
Do not take ROCTAVIAN if you:
What is the most important information I should know about ROCTAVIAN?
ROCTAVIAN may cause serious side effects during the infusion and afterward:
What should I tell my doctor before I get ROCTAVIAN?
Talk to your doctor about the following:
What should I avoid after taking ROCTAVIAN?
What are the possible side effects of ROCTAVIAN?
What other information should I know before getting ROCTAVIAN?
Talk to your doctor about the potential risks and benefits of ROCTAVIAN. Whether a patient experiences a benefit or not, the risks discussed here and with your doctor still apply.
These are not all the possible side effects of ROCTAVIAN. Talk to your doctor for medical advice about side effects. You may report side effects to BioMarin Pharmaceutical Inc. at 1-866-906-6100 or FDA at 1-800-FDA-1088.
Please see additional safety information in the Prescribing Information and Patient Information.
What is ROCTAVIAN?
ROCTAVIAN is a one-time gene therapy used for the treatment of adults with severe hemophilia A who do not have antibodies to the virus, AAV5 which is determined by a blood test. ROCTAVIAN uses a modified virus, called a vector, to deliver a working copy of the Factor VIII gene to liver cells to enable your body to produce clotting factor on its own, which helps the blood to clot and prevents or reduces the occurrence of bleeding. The modified virus does not contain viral DNA and does not cause disease in humans.