Now Approved:
Same Evrysdi, now in tablet form
For illustrative purposes only.
Results in infants as young as 2 months with Type 1 SMA
Results in infants as young as 2 months with Type 1 SMA
Results in infants as young as 2 months with Type 1 SMA
FIREFISH is a 2-part, open-label study of Evrysdi in 62 infants aged 2 to 7 months with Type 1 SMA. Part 1 explored the dose and safety of Evrysdi in 21 infants. Part 2 measured the effectiveness and safety of Evrysdi in 41 infants. Fifty-eight infants aged 2 to 7 months who received the recommended dose of Evrysdi in Parts 1 and 2 were included in a pooled analysis.
Changing the course of SMA
Infants with Type 1 SMA exceeded expectations for development compared with infants who did not receive treatment
Of the infants taking the recommended dose of Evrysdi
Of the infants taking the recommended dose of Evrysdi
FIREFISH PART 2 (MAIN MEASUREMENT)
AFTER 1 YEAR
of infants (12/41) were able to sit without support for at least 5 seconds, as measured by BSID-III
FIREFISH PART 2 (MAIN MEASUREMENT)
AFTER 1 YEAR
of infants (12/41) were able to sit without support for at least 5 seconds, as measured by BSID-III
FIREFISH PARTS 1 AND 2
AFTER 1 YEAR
of infants (19/58) were able to sit without support for at least 5 seconds, as measured by BSID-III
FIREFISH PARTS 1 AND 2
AFTER 1 YEAR
of infants (19/58) were able to sit without support for at least 5 seconds, as measured by BSID-III
AFTER 2 YEARS
of infants (35/58) were able to sit without support for at least 5 seconds, as measured by BSID-III
AFTER 2 YEARS
of infants (35/58) were able to sit without support for at least 5 seconds, as measured by BSID-III
Infants with Type 1 SMA are typically not able to sit on their own without treatment
Infants with Type 1 SMA are typically not able to sit on their own without treatment
BSID-III stands for Bayley Scales of Infant and Toddler Develoment-Third Edition.
Remarkable achievement of key milestones not typically seen in infants without treatment with Type 1 SMA
Of the infants taking the recommended dose of Evrysdi
AFTER 2 YEARS
of infants (23/58) were able to sit without support for at least 30 seconds, as measured by BSID-III
AFTER 2 YEARS
of infants (23/58) were able to sit without support for at least 30 seconds, as measured by BSID-III
AFTER 2 YEARS
of infants (16/58) were able to stand, as measured by HINE-2
- 9/58 could stand supporting weight
- 7/58 could stand with support
AFTER 2 YEARS
of infants (16/58) were able to stand, as measured by HINE-2
- 9/58 could stand supporting weight
- 7/58 could stand with support
BSID-III stands for Bayley Scales of Infant and Toddler Development–Third Edition. HINE-2 stands for Hammersmith Infant Neurological Examination–Module 2.
The Bayley Scales of Infant and Toddler Development–Third Edition (BSID-III) is a gross motor scale that assesses a range of physical abilities such as sitting, rolling, and crawling.
The Hammersmith Infant Neurological Examination–Module 2 (HINE-2) assesses 8 developmental milestones for infants, including head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking.
“When we first started this journey, we were unsure, and the unknown scared us. But now, thanks to Evrysdi, it excites us.”
Amber, mom to Payton who is living with Type 1 SMA
Slowing the progression of SMA
Of the infants taking Evrysdi (all dose strengths)
Of the infants taking Evrysdi (all dose strengths)
AFTER 1 YEAR
of infants (54/62) were alive and able to breathe without permanent support*
AFTER 1 YEAR
of infants (54/62) were alive and able to breathe without permanent support*
AFTER 2 YEARS
of infants (52/62) were alive and able to breathe without permanent support*
AFTER 2 YEARS
of infants (52/62) were alive and able to breathe without permanent support*
Without treatment, no more than 25% of infants with Type 1 SMA are expected
to survive beyond 14 months without permanent breathing support
Without treatment, no more than 25% of infants with Type 1 SMA are expected to survive beyond 14 months without permanent breathing support
*Permanent support was defined as having a tracheostomy (a surgery where a tube is inserted in the front of the throat into the windpipe) or more than 21 days of either noninvasive ventilation support (16 or more hours a day) or being intubated (a procedure where a breathing tube is inserted down the throat and into the windpipe) to help with breathing, in the absence of an acute reversible event.
EXPLORATORY OBSERVATIONS SUGGEST
Infants taking Evrysdi had better feeding and swallowing abilities compared with infants without treatment*
Of the infants taking the recommended dose of Evrysdi, after 2 years:
of infants (48/52) were able to feed orally
of infants (50/52) were able to swallow
Without treatment, 87% of infants with Type 1 SMA typically require feeding
support via a feeding tube by 18 months old
Without treatment, 87% of infants with Type 1 SMA typically require feeding support via a feeding tube by 18 months old
This information is considered exploratory, which means the clinical trial was not specifically designed to show a treatment effect on feeding and swallowing. Data should be interpreted with caution.
*Includes infants who could eat by mouth or in combination with a feeding tube.
Hear from community members
Celebrating new joys
Get to know Amber, mom to Payton, and watch some of the daily moments that make her excited for the future.
Good morning! Are we gonna have a good day? Yeah. Yeah!
My name is Amber. I've been married to my husband, John, for just over five years. We've been together a little over eight. And we have two amazing kiddos. So we have our son, Michael, who is four years old, and then we have our sweet baby girl, Payton. She is two. Payton has Type 1 SMA.
When we first started this journey we were unsure and the unknown scared us. But now, thanks to Evrysdi, it excites us. We're excited to see what the next chapter of Payton's life looks like. I don't know that there's a day that goes by that I don't feel so much pride in everything that she does, knowing that she has been through so many obstacles, she has overcome so many challenges, and she shows up every day.
The past two years have been scary, overwhelming, heartbreaking, exciting, joyful. They have been the hardest two years for our family, but they have been the most amazing two years for our family. When we first received Payton's diagnosis, I dove right in. I wanted to learn everything about SMA. I wanted to learn what treatment options were available, how I could get her the best possible care. That meant looking at treatments, it meant looking at medical equipment, it meant having conversations and really becoming an expert in SMA. I am constantly researching, constantly trying to learn more, and I came across Evrysdi. When considering Evrysdi I spoke with Payton's medical team, and we really discussed first and foremost if this treatment was right for her. And then we discussed the safety as well as any potential side effects. After we spoke with them and we were able to evaluate the side effects and the potential positive impact it would have in her daily life, we decided to move forward with Evrysdi. I think that our family's been so fortunate. We're fortunate that she's here today, and that's an amazing milestone that so many have not gotten and we're so thankful for that.
I love watching Payton, the intent behind her action. She's so curious. She doesn't know what limitations she has, so she just tries to beat them every day. I think for Payton, her curiosity drives her. And I think that's part of why she's so strong today, because she doesn't know how to measure a limitation. She just knows to keep trying. And if it doesn't work the first time, maybe we'll try a different way. And I think that her curiosity has really been her building block. It's allowed her to develop new skills and to learn new things. Payton loves spending time with her cousins and her friends. She loves swinging in her swing. She loves doing puzzles. It's no secret, Payton loves all things water. I remember from the very beginning, those bath times, you could just see her face kind of light up. She'd get so excited, and playing on the swing set with her brother, hearing them giggle together, that's the stuff that melts my heart.
Payton has this contagious spirit about her. I think I've watched so many people meet her and they can't help but smile and just love her. She is just a breath of fresh air. Anytime a parent thinks about goals, I think it always is that you want them to be the best possible version of themself. When we first started the journey of SMA, we didn't know what Payton's future held, but we knew that we didn't want those three letters to define her. We didn't want her to be SMA. We wanted her to be Payton. So in all of our daily activities and all of the things we do as a family, we make sure that that is not our core focus. We hope that through those efforts, we're instilling that same mantra in her, and that she'll continue to not allow SMA to be the focus of who she is, but really come into her own and be the kindhearted, curious, adventurous little girl that we know she is.
The most important part of Payton's daily routine is that Evrysdi. So every morning she wakes up, she gets breakfast, and then we give her Evrysdi by mouth and we're ready to start the day. Evrysdi fits into our family's routine so nicely. I'm able to give it first thing in the morning and then we're on the go for the rest of the day.
Our family is constantly traveling, so having a medication that we could take with us was very important. Making sure that it could be on the go just like our family. So, we oftentimes have our Evrysdi cooler bag packed and we're ready to go.
For those that are out there still looking for treatment, I would just encourage you to do your research, to know the options that are out there, but most importantly to get early treatment. Speak with your doctor, take their guidance, allow them to help you, because your child's future is bright.
I think every parent goes into parenthood with all these milestones that your children are gonna achieve. And we learned early on that those milestones would be hard for Payton. But she is persistent, and she is a very determined little girl. So every day she shows up. She is the happiest little girl. She always has a smile on her face.
At the end of the day, Payton is just a little girl who wants to play with her toys. She's not a disease, she's not three letters, she's just a two-year-old girl who loves princesses.
So pretty!
This is Evrysdi in action.
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Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.
Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.
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