ACHIEVING NEW MOTOR MILESTONES

Infants taking SPINRAZA showed improvements in milestones that
are rarely, if ever, achieved in untreated infants

Sofia
age 2.5

Infantile-onset (Type 1) SMA
treated with SPINRAZA

Type 1 Spinraza (nusinersen) patient motor milestones

ENDEAR was a well-controlled, phase 3 clinical study with 2:1 randomization, meaning two-thirds of individuals received SPINRAZA and one-third was untreated.

The study assessed the proportion of “responders,” or individuals with an improvement in motor milestones, as measured by Section 2 of the Hammersmith Infant Neurological Examination (HINE).

HINE Section 2 motor milestone responders

Hammersmith Infant Neurological Examination Section 2 motor milestone responders in SPINRAZA® (nusinersen) ENDEAR clinical trial

Clinically meaningful benefits were seen in the planned interim analysis of infants who completed at least 6 months (183 days) of treatment. The final analysis showed even greater milestone improvements in a larger group of infants who had completed at least 9 months of treatment.

The HINE Section 2 uses a point system to measure progress in various categories of motor milestone development for infants between 2 and 24 months of age.

What happened to the other infants who were not considered “responders”?


While not all infants met the specific “responder” criteria, all infants who were alive either maintained or improved their HINE Section 2 score after at least 6 months of treatment in the interim analysis.

Mother holding Type 1 child with SMA

One mom’s story

The doctor told me that she tested positive for SMA, and typically children with Type 1 SMA only live 12 to 24 months. And he pretty much said, ‘Enjoy the time that you have with her, because she’s not going to be here for very long.’ I didn’t want to take that as a final answer, and I’m so glad I didn’t. When I found out that there was a potential treatment, I had hope, and we enrolled her in the phase 3 clinical trial. While I still worry about Sofia’s SMA, that feeling of hope has not gone away. SPINRAZA has changed the way we see SMA as parents.

—Sofia’s mother

Watch Sofia’s story
SPINRAZA stories:
Unlocking Sofia’s inner star

IMPORTANT SAFETY INFORMATION

Increased risk of bleeding complications has been observed after administration of similar medicines. Your healthcare provider should perform blood tests before you start treatment with SPINRAZA and before each dose to monitor for signs of these risks. Seek medical attention if unexpected bleeding occurs.

Increased risk of kidney damage, including potentially fatal acute inflammation of the kidney, has been observed after administration of similar medicines. Your healthcare provider should perform urine testing before you start treatment with SPINRAZA and before each dose to monitor for signs of this risk.

The most common side effects of SPINRAZA include lower respiratory infection, fever, constipation, headache, vomiting, back pain, and post-lumbar puncture syndrome.

These are not all of the possible side effects of SPINRAZA. Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Before taking SPINRAZA, tell your healthcare provider if you are pregnant or plan to become pregnant.

Please see full Prescribing Information.

As a courtesy, our full Prescribing Information is also available en Español. For prescribing decisions, please refer to official approved labeling.

This information is not intended to replace discussions with your healthcare provider.


INDICATION

SPINRAZA is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients.