BEQVEZTM (fidanacogene elaparvovec-dzkt) is
NOW FDA APPROVED
A one-time gene therapy infusion for eligible adults with hemophilia B*
Tim, who received the BEQVEZ infusion in 2021.†
Individual results and duration of effect may vary. Monitoring is required following this one-time treatment.
*BEQVEZ is a one-time gene therapy used for the treatment of adults with moderate to severe hemophilia B who are receiving routine prophylaxis, have a current life-threatening bleed or a history of life-threatening bleeds, or have repeated serious spontaneous bleeds. Before treatment with BEQVEZ, your healthcare professional will conduct a blood test to check for antibodies to the AAVRh74var virus. The results of this testing will help determine if you may receive BEQVEZ.
†Tim participated in the BEQVEZ clinical study. He was compensated for sharing his story.
Meet Tim and Bill, actual BEQVEZ clinical trial participants.
Check out Tim’s story
transcript
Tim
This is the last time that I had to infuse myself with factor before I got BEQVEZ. Ooh. This is actually me getting BEQVEZ. You can see even behind the mask. I am very excited.
00:23
Tim
Okay.
00:24
Tim
Hey, I’m Tim. I got BEQVEZ back in 2021. Let me tell you all about it. Oh, there it is. There’s me.
00:35
Tim
BEQVEZ is a one-time gene therapy for certain adults with moderate to severe hemophilia B. Your doctor will perform certain tests and review your medical condition and history to determine if BEQVEZ is right for you.
00:47
Tim
A lot of my childhood was just in and out of the hospitals. Eventually, I started on a prophylactic schedule. But then there’s all the logistical planning that comes along with having hemophilia. So much of my time was dedicated to getting the treatment, ordering the factor, making sure that I had it on hand, and it's stored properly, kept at the right temperature. It was just the biggest headache.
01:07
Brit
Hey Bub, wanna dye your hair?
01:09
Tim
Yeah.
01:09
Brit
Alright, let’s go. You gotta take this off... you're gonna get dye on it.
01:12
Tim
Yes, yes, yes.
01:12
Tim
Yes. Having hemophilia, it’s more than just about you. It’s about your family...
01:18
Brit
Woooo. Alright! Got a towel here for you.
01:21
Tim
…which I have right here.
01:21
Tim
My beautiful wife, Brittany.
01:23
Brit
Hey. A little before picture…
01:30
Tim
Alright, let’s do it.
01:31
Brit
Let’s do it.
01:32
Tim
Through my twenties and even early thirties, I just was constantly neglecting my hemophilia against my doctor’s advice. When my band became a little bit more serious, it involved a lot of travel. In my head, I'm band first, music first. If I can’t treat today, I’ll treat tomorrow. We were traveling in a van all across the country. There was many times where I was in the back of the van infusing myself right before a gig.
02:02
Brit
Let me see ya.
02:03
Tim
I was missing a lot of treatments because I was just too busy. And then I would always pay the price. And I would have breakthrough bleeds in my hip caused by my mismanagement of my hemophilia.
02:13
Brit
Here. Come over here. What’s dad doing?
02:20
Tim
There’s always been that little thing in the back of my head. One day, maybe science will be far enough advanced that I can just do this one treatment, and I can just live my life. As my doctor informed me about the possible results from the gene therapy, he said, this is not a cure. It’s not a cure for hemophilia. It’s gonna work differently for everyone. And it’s a one-time thing.
02:47
Tim
He also told me he’d let me know when I could stop factor IX prophylaxis or resume it, and whether I’d need to take FIX for surgeries, injuries, or bleeds. My doctor advised about NAb testing and liver health tests. We also talked about the importance of not donating sperm and being abstinent, or using a condom for the first six months after the infusion, and limiting alcohol during the first year.
03:12
Tim
I said, let’s do it. I’m ready to change my lifestyle. I was just…just kind of daydreaming about my future. So if you have any hesitation with getting BEQVEZ, I would recommend talking to…
03:35
Tim
…your doctor to help determine if BEQVEZ is right for you.
03:40
Tim
The care team was super supportive and they planned the entire thing out so well for me, and they were so…
03:46
Tim
…hospitable to our schedules. They just gave me a timeline of when my blood draws were gonna be. Once or twice a week at first, and over time, less and less.
03:57
Tim
My doctors were super prepared and knew of the possible side effects. I personally didn't have any side effects, but some people who took BEQVEZ needed corticosteroids to help manage elevated liver enzymes. My doctors always told me I still have to have my factor on hand, I still have hemophilia. Just for me personally, it was a lot more mild hemophilia.
04:16
Tim
It’s not possible to predict who will respond to BEQVEZ and how long it will last.
04:22
Tim
Since BEQVEZ, I’ve had zero bleeds and zero regrets. My factor levels have been mild. Oh, that’s when we were exploring the temple in Tokyo. So I had my first a-ha moment about a year after getting BEQVEZ. I was on tour with my band in the UK, and of course something happened where I, like, stubbed my toe or hurt myself. So immediately my brain goes, great. Now I have to deal with factor and treating myself so I’m not, you know, bleeding in a foreign country. But then I had this moment where I’m like, I don’t have to treat myself.
04:55
Tim
I didn’t realize how much of a time commitment it really was until I just didn’t have to deal with all this stuff anymore.
05:06
Tim
It’s like I took hemophilia from ten and just turned it down on the volume notch. What was that, Nacho? Oh, Nacho says, talk to your doctor and see if BEQVEZ is right for you. Alright, from the top. Tippity top! This one’s for all the people out there living with hemophilia. To just take all that, just everything related to hemophilia and just make it just this thing that’s in the back of your mind.
05:41
Tim
For me, that means the world.
05:48
Tim
This is me living with hemophilia differently.
05:52
Tim
Keep watching for Important Safety Information and see the full Prescribing Information on BEQVEZ.com.
05:57
Voiceover
Indication and Important Safety Information for BEQVEZ (fidanacogene elaparvovec-dzkt).
What is BEQVEZ? BEQVEZ is a one-time gene therapy used for the treatment of adults with moderate to severe hemophilia B who are receiving routine prophylaxis, or have a current life-threatening bleed or a history of life-threatening bleeds, or have repeated serious spontaneous bleeds. Before treatment with BEQVEZ, your healthcare professional will conduct a blood test to check for neutralizing antibodies to the AAVRh74var virus. The results of this testing will help determine if you may receive BEQVEZ. Important Safety Information. Before receiving BEQVEZ, tell your healthcare professional about all your medical conditions, including if you have kidney or liver problems, including hepatitis, have factor IX inhibitors or a history of factor IX inhibitors, have an active infection. BEQVEZ may cause serious side effects, including increased liver enzymes.
06:51
Voiceover
Most patients treated with BEQVEZ developed elevated liver enzyme levels and most did not experience any symptoms. Your healthcare professional will monitor liver enzymes and factor IX activity levels before administration of BEQVEZ and frequently following the administration to detect and identify possible elevations in liver enzymes and to monitor your response to BEQVEZ. Your doctor may prescribe a corticosteroid for the treatment of elevated liver enzymes. Limit alcohol consumption during the first year following BEQVEZ infusion, as alcohol may reduce the effect of BEQVEZ and may increase liver enzyme levels. Infusion reactions, including hypersensitivity and severe allergic reactions (anaphylaxis) may occur. Alert your healthcare professional right away if you get any symptoms of hypersensitivity, which may include, but are not limited to low blood pressure, fever, heart palpitation, nausea, vomiting, chills, or headache. BEQVEZ can insert itself into the DNA of cells in the human body.
07:45
Voiceover
The effect that insertion may have on those cells is unknown but may contribute to a theoretical risk of cancer. There have been no reported cases of cancer caused by treatment with BEQVEZ. The most common side effect of BEQVEZ is increased liver enzymes. These are not all the possible side effects of BEQVEZ. For more information, ask your healthcare professional. Talk to your healthcare professional before receiving any vaccinations if you are taking a corticosteroid. Talk to your doctor about any medications you plan to take including over-the-counter medications, herbal supplements, and vitamins as certain substances can affect the liver and may reduce the effectiveness of BEQVEZ. Your healthcare professional will test your factor IX activity levels and for factor IX inhibitors. After receiving BEQVEZ, your doctor will discuss whether and when you are able to stop prophylaxis,
08:28
Voiceover
if you need to resume prophylaxis, and actions you may need to take for surgeries, procedures, injuries, and bleeding events. Do not donate blood, organs, tissues, or cells for transplantation following administration of BEQVEZ. BEQVEZ is not intended for administration to women. Males should not donate sperm and should use a male condom or not have sexual intercourse for up to six months after receiving BEQVEZ. Patients and caregivers should ensure proper handling of any materials that have come into contact with a patient’s urine, feces, saliva, mucus, or semen in the first six months after BEQVEZ infusion. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1800 FDA 1088. Please see full Prescribing Information at www.BEQVEZ.com.
09:17
Tim
Talk to your doctor about BEQVEZ.
Check out Bill's story
transcript
00:13
Bill
Being born in 1969, I've lived through the entire gamut in hemophilia care. Hi, I'm Bill, and I'm considered a legend in my own mind. I received BEQVEZ in 2016. I was one of the first people to receive it. BEQVEZ is a one-time gene therapy for certain adults with moderate to severe hemophilia. B. Your doctor will perform certain tests and review your medical condition and history to determine if BEQVEZ is right for you. So this is probably my all-time favorite vehicle. Right now, it needs a little bit of engine work, but I'm hoping to get this thing back together and back on the car show circuit. Living with hemophilia with early treatments was, you know, pretty difficult. In the beginning, it was literally just pack me in ice. There was no such thing as prophylaxis. It was a treat on demand.
01:16
Bill
When factor came around, they were able to create a home treatment program. Eventually, products came out that again made things easier and easier. Being in antique and classic cars, one of my big things is I'll go to car shows. One thing I definitely don't miss after a big car show, I'd have to infuse the night before, do my walking during the day, be feeling pretty rough when I got home, and potentially have to treat again. That was just the price that I had to pay for making sure that I was involved in activities. Initially, I was getting factor prophylaxis once every ten days, and sometimes, additionally, for breakthrough bleeds. As all hemophilia patients know, infusing yourself is not fun.
02:11
Bill
So I'd been doing research on gene therapy casually for quite a while and happened to see a news article about a study that was about potentially receiving BEQVEZ. So one of the first steps of the process was to meet with the team lead. They explained in depth what they were going to do, how it was going to happen, potential risk versus rewards, obviously discussed it with my family. My doctor explained that both the individual results and how long those results last may vary. However, they also reassured me that they'd let me know when I could stop FIX prophylaxis or resume it, and whether I need to take FIX for surgeries, injuries, or bleeds.
02:50
Bill
So part of the process for checking my eligibility for BEQVEZ was to look for neutralizing antibodies, known as NAbs, against the vector delivering the modified factor IX gene, and also assessing my liver health. We also talked about the importance of not donating sperm and being abstinent or using a condom for the first six months after the infusion, and limiting alcohol during the first year. And then, my medical team considered it to be a good fit for me, which it worked out in my favor. So my infusion was very seamless. The people doing the process were extremely nice, helpful, answered any questions that I had. And they monitored me for any possible infusion reactions on the day of my infusion. I didn't have any adverse effects from BEQVEZ, but talk to your doctor about any risks or benefits.
03:44
Bill
The most common side effect seen in the BEQVEZ studies was elevated liver enzymes. They also gave me a timeline of when my blood draws were going to be, starting with once or twice a week at first and over time, less and less. So my whole motivation for looking into gene therapy was to try and reduce the number of bleeds. And then I went to my record of two and a half years without getting infusions. After receiving BEQVEZ, my number of infusions went way down. At the age of 50, I started taking taekwondo and recently received my black belt. Martial arts has definitely been a confidence booster for me. Not only understanding and learning about my body, becoming more physically fit, also being able to spend more time with my son, who is also doing martial arts.
04:38
Bill
It's been fantastic watching my son progress. When he got his black belt, that was certainly quite the proud papa moment.
04:51
Bill
Woah! There it goes!
04:53
Noah
What are you doing?
04:55
Bill
I don't know. It is a one-shot on goal. There's no redos. Unfortunately, if it doesn't work, there's no fix to do an update or tune up on it. So just make sure you talk with your doctors and make sure that the product is a good fit. Hard left and hit it. You'll start doing donuts.
05:15
Noah
Hard left?
05:17
Bill
Hard left. Yup. I don't miss having to remember to infuse. It got to the point where the last time I had to get factor infusion for an injury, I kind of forgot how to put it together. It took me a little while. This is me living with hemophilia differently. Keep watching for Important Safety Information and see the full Prescribing Information on BEQVEZ.com.
05:46
Voiceover
Indication and Important Safety Information for BEQVEZ (fidanacogene elaparvovec-dzkt).
What is BEQVEZ? BEQVEZ is a one-time gene therapy used for the treatment of adults with moderate to severe hemophilia B who are receiving routine prophylaxis, or have a current life-threatening bleed or a history of life-threatening bleeds, or have repeated serious spontaneous bleeds.
Before treatment with BEQVEZ, your healthcare professional will conduct a blood test to check for neutralizing antibodies to the AAVRh74var virus. The results of this testing will help determine if you may receive BEQVEZ.
Important Safety Information
Before receiving BEQVEZ, tell your healthcare professional about all your medical conditions, including if you: have kidney or liver problems, including hepatitis; have factor IX inhibitors or a history of factor IX inhibitors; have an active infection.
BEQVEZ may cause serious side effects, including: increased liver enzymes.
06:41
Voiceover
Most patients treated with BEQVEZ developed elevated liver enzyme levels and most did not experience any symptoms. Your healthcare professional will monitor liver enzymes and factor IX activity levels before administration of BEQVEZ and frequently following the administration to detect and identify possible elevations in liver enzymes and to monitor your response to BEQVEZ. Your doctor may prescribe a corticosteroid for the treatment of elevated liver enzymes. Limit alcohol consumption during the first year following BEQVEZ infusion, as alcohol may reduce the effect of BEQVEZ and may increase liver enzyme levels. Infusion reactions, including hypersensitivity and severe allergic reactions (anaphylaxis) may occur. Alert your healthcare professional right away if you get any symptoms of hypersensitivity, which may include but are not limited to low blood pressure, fever, heart palpitation, nausea, vomiting, chills, or headache. BEQVEZ can insert itself into the DNA of cells in the human body.
07:35
Voiceover
The effect that insertion may have on those cells is unknown but may contribute to a theoretical risk of cancer. There have been no reported cases of cancer caused by treatment with BEQVEZ. The most common side effect of BEQVEZ is increased liver enzymes. These are not all the possible side effects of BEQVEZ. For more information, ask your healthcare professional. Talk to your healthcare professional before receiving any vaccinations if you are taking a corticosteroid. Talk to your doctor about any medications you plan to take including over the counter medications, herbal supplements, and vitamins as certain substances can affect the liver and may reduce the effectiveness of BEQVEZ. Your healthcare professional will test your factor IX activity levels and for factor IX inhibitors. After receiving BEQVEZ, your doctor will discuss whether and when you are able to stop prophylaxis,
08:18
Voiceover
If you need to resume prophylaxis, and actions you may need to take for surgeries, procedures, injuries, and bleeding events. Do not donate blood, organs, tissues, or cells for transplantation following administration of BEQVEZ. BEQVEZ is not intended for administration to women. Males should not donate sperm and should use a male condom or not have sexual intercourse for up to six months after receiving BEQVEZ. Patients and caregivers should ensure proper handling of any materials that have come into contact with a patient’s urine, feces, saliva, mucus, or semen in the first six months after BEQVEZ infusion. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. Please see full Prescribing Information at www.BEQVEZ.com.
09:05
Noah
Hold up, hold up.
09:09
Bill
What is that look on your face, dude?
09:10
Noah
You, like, surprised me. You just, stop! I gotta, like.
09:14
Bill
I'm still recording!
09:15
Noah
Oh, I gotta, like, warm my hand. Fine.
09:17
Bill
This is my story.
Talk to your doctor about BEQVEZ.
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