WHY SPINRAZA/PRESYMPTOMATIC STUDY
Improvements are possible with SPINRAZA
When started earlier, SPINRAZA may help children progress on WHO motor milestones consistent with normal development.
Supportive study: NURTURE
Who: 25 infants aged 6 weeks and younger who had not yet shown symptoms of SMA
Study time: This supportive study is ongoing
Primary outcomes: Time to death or respiratory intervention†
Secondary outcomes: The effect SPINRAZA has on reaching WHO motor milestones
Limitations: Small number of participants. The study is open-label. The study has no control group so the data may not be as reliable as a controlled clinical trial.
Safety: Consistent with the SPINRAZA prescribing information. After a follow-up of over 14 months to 5.7 years, 25/25 infants experienced any AE (mild, moderate, or severe), and no new safety concerns were identified
After at least 14 months of treatment:
At the initial interim analysis performed after all infants had received SPINRAZA for at least 14 months (median, 25 months; range, 14 to 34 months), 100% (25/25) of infants were alive without the need for permanent ventilation,‡ 100% (25/25) were sitting without support, 88% (22/25) were walking with assistance, and 77% (17/22) of infants were walking independently.
After nearly 5 years of follow-up*:
100% (25/25) of presymptomatic infants were alive without permanent ventilation after a median follow-up of 4.9 years in the study. 84% (21/25) never required respiratory intervention
†Respiratory intervention is defined as ventilation for ≥6 hours/day continuously for ≥7 days, or tracheostomy.
‡Permanent ventilation is defined as ventilation for ≥16 hours/day for >21 days in absence of an acute reversible event, or tracheostomy.
The majority of presymptomatic infants achieved the following WHO motor milestones:
*At least half of patients were followed for at least 4.9 years.
Watch these stories of hope
Parents of children living with SMA share their experiences with early diagnosis and the power of early treatment.
Transcript
Super:
MORE STORIES OF HOPE
Parents of children living with SMA reflect on the significance of early diagnosis and power of early treatment
JENNIFER (KIRRA’S MOTHER): You deliver this beautiful baby and you hold your baby and the first phone call is not mom, dad, brother, sister. Your first phone call is insurance and your doctors. And so it’s hard sometimes, but that’s what’s at the top of your list because you know that that’s going to get the ball rolling.
PEARL ANN (MAVRIK’S MOTHER): It’s very important to diagnose SMA early. The faster you know, the faster you can react.
ALYSSA (JUDE’S MOTHER): We have five children, and two of them have spinal muscular atrophy. Because his older sister has SMA, Jude was diagnosed in utero via amniocentesis.
JENNIFER (KIRRA’S MOTHER): We had lost a child, and we were living with Nathan, who was experiencing SMA daily. With Kirra’s diagnosis, we were heartbroken. You spend a few days crying. You spend a few days not knowing what to do next, and then you just know it. Mom takes over, and you just know that whatever it looks like, I’m going to do whatever I can for my baby, and I think that that’s any parent.
FEMALE VOICEOVER: In 2016, SPINRAZA® (nusinersen) was approved as a prescription medicine for infants, children, and adults living with SMA. A health care provider will monitor for signs of bleeding complications or kidney damage while taking SPINRAZA. These are not all the possible side effects.
Super:
In 2016, SPINRAZA® (nusinersen) was approved as a prescription medicine for infants, children and adults living with SMA
[Logo and ISI appear here]
These are stories of 3 families living with SMA. This is their experience. Others may have different experiences.
Super:
These are stories of 3 families living with SMA. This is their experience; others may have different experiences.
JUDE // AGE 5
KIRRA //AGE 5
MAVRIK // AGE 6
JENNIFER (KIRRA’S MOTHER): Kirra is my five-, almost six-year-old daughter. She is spunky and feisty, a sweet little personality, my little sidekick. Kirra likes to roller-skate, to play outside, to ride her bike, to go for walks. She loves to snuggle and read a book.
Super: JENNIFER
Daughter Kirra (age 5) started treatment with SPINRAZA at 11 days old before SMA symptoms occurred
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
PEARL ANN (MAVRIK’S MOTHER): Mavrik is quite the character in his own little way, and he really loves dinosaurs and playing with his siblings.
Super: PEARL ANN
Son Mavrik (age 6) started treatment with SPINRAZA at 7 weeks old before SMA symptoms occurred
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
ALYSSA (JUDE’S MOTHER): Jude likes to play outside and play with trains…play with his siblings. We read books. He likes to shoot rockets and throw balls. He’s just a quiet-natured kid. Knowing that SPINRAZA was available gave us a lot of hope.
Super: ALYSSA
Son Jude (age 5) started treatment with SPINRAZA at 9 days old before SMA symptoms occurred
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
JENNIFER (KIRRA’S MOTHER): I don’t even know if I can put into words how excited we were that our baby was going to be treated. And so, Kirra was dosed at 11 days old.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
ALYSSA (JUDE’S MOTHER): We learned about SPINRAZA as a treatment because Annabel’s physicians told us that was an option. And so when Jude was born, we already knew that that was a treatment option for him. Jude started treatment before SMA symptoms started showing up.
PEARL ANN (MAVRIK’S MOTHER): The neurologist did recommend SPINRAZA and he received his first dose of SPINRAZA at seven weeks old. This is what Mavrik needs. This is what he gets.
A few of the milestones I’ve tracked in the past was rolling over, sitting, supported, unsupported, and learning to crawl.
Super: In an ongoing supportive study, 25 infants who had not yet shown symptoms of SMA were treated with SPINRAZA for nearly 5 years.
The study had a small number of participants and no untreated group to compare results, so the data may not be as reliable as other clinical trial data.
Those were very tear-jerking milestones for us.
Super: IN THE STUDY: 100%
25 out of 25 were sitting without support after at least 14 months of taking SPINRAZA, and continued to do so after nearly 5 years.
Milestones we’re currently working on is walking without assistance.
Super: IN THE STUDY: 88%
22 out of 25 were walking with assistance after at least 14 months taking SPINRAZA.
Super: IN THE STUDY: 96%
24 out of 25 were walking with assistance after nearly 5 years.
JENNIFER (KIRRA’S MOTHER): Kirra’s doing a great job meeting her milestones. She’s sitting, walking. She is a joy to watch.
Super: IN THE STUDY: 77%
17 out of 22 were walking independently after at least 14 months taking SPINRAZA.
ALYSSA (JUDE’S MOTHER): Jude did achieve motor milestones, just in a delayed pattern. So he was able to sit unassisted, but it was after the six-month mark. And he walked, but it was after he was a year old. He hasn’t started running yet, but I have hope that he will.
Super: IN THE STUDY: 92%
23 out of 25 were walking independently after nearly 5 years.
Super: IN THE STUDY: 100%
25 out of 25 were alive without permanent ventilation after at least 14 months of taking SPINRAZA, and continued to do so after nearly 5 years.
JENNIFER (KIRRA’S MOTHER): We didn’t know what to expect. So when Kirra Faith started cruising in our living room and holding onto our couch, and when you could hold her hands and walk across the room, there are no words.
SPINRAZA is given as an injection into the lower back, a procedure called an intrathecal injection, delivering medication directly into the central nervous system.
Super: SPINRAZA is given as an injection into the lower back, a procedure called intrathecal injection.
Treatment with SPINRAZA starts with four injections in the first two months as initial doses, and after that, SPINRAZA is given every four months.
Super: SPINRAZA starts with four injections in the first two months as initial doses, and after that SPINRAZA is given every four months.
Some lab tests are run when first starting SPINRAZA, and as recommended by a medical professional, they are run again before each dose to monitor their blood and kidneys.
Super: SPINRAZA may increase the risk of bleeding and kidney damage. These are not all the possible side effects.
Talk to your doctor about risks and benefits before starting SPINRAZA.
PEARL ANN (MAVRIK’S MOTHER): I want other parents to know they’re not alone.
Super: Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
ALYSSA (JUDE’S MOTHER): I think there’s a lot of hope.
JENNIFER (KIRRA’S MOTHER): You will get through this and you don’t believe that you can, but you will.
ALYSSA (JUDE’S MOTHER): SPINRAZA gave us hope and we want other families to know that it can give their children hope as well.
JENNIFER (KIRRA’S MOTHER): We talked to her doctor about SPINRAZA. We don’t see any reason to change. She’s doing amazing. She’s living with SMA and she’s living with SPINRAZA, and we have found a happy medium. She’s doing great, and we’re so grateful for what she is doing.
[ISI present in video and available below.]
Super: ©2023 Biogen. All rights reserved. 11/23 SPZ-US-5527 225 Binney Street, Cambridge, MA 02142
FEMALE VOICEOVER: If you are considering treatment, talk to your child’s doctor about SPINRAZA.
Super: If you are considering treatment, talk to your child’s doctor about SPINRAZA.
SPINRAZA.com
Additional Data
After ~3 years of follow-up§
88%22 out of 25
able to suck/
swallow well
based on maximal HINE-1 score at the
day 778 visit
After ~4 years of follow-up
84%21 out of 25
never required
tube feeds
92%23 out of 25 never needed
to
suction excess saliva or
drool
based on caregiver assessment of swallowing difficulty during mealtime over a period of 7 days
§At least half of patients were followed for at least 3.8 years.
“For Camden to do what he is able
to
do, we are just so thankful.”