Dear Members of the SMA Community,
As we celebrate National Family Caregivers Month this November, Biogen acknowledges the vital role caregivers have in our society, including within our spinal muscular atrophy (SMA) community. We also recognize that caregivers need our support more than ever.
Caregivers in the SMA community face an especially unique set of challenges. Since the onset of COVID-19, nearly half of all households affected by SMA have experienced disruptions in how they access essential services, according to a survey from Cure SMA, adding to an already significant physical and emotional burden.1 Biogen has been collaborating with patients, caregivers, and advocacy groups across the SMA community to deepen our understanding of caregivers’ challenges and find new ways to help address them. We are pleased to share updates about some of these programs below.
In addition, we would like to share with you that we continue to gather new data on SPINRAZA, including through several presentations at the recent World Muscle Society (WMS) virtual conference.2,3 Below we also share information about an independent study that added new findings to the growing body of real-world data supporting SPINRAZA for the treatment of adults with SMA.
As the end of the year approaches, we wish everyone in the SMA community a happy holiday season and look forward to continuing to move further together in 2021.
The Biogen SMA Team
SPINRAZA Home Sample Collection
Earlier this summer, Biogen began a phased introduction of a new program that offers an at-home option for SPINRAZA patients to complete the lab monitoring requirements ahead of a dosing day. The goal of this program is to reduce the burden on people with SMA and their caregivers by offering a convenient at-home option for collecting the samples through trained professional phlebotomists. Biogen’s Home Sample Collection program has already been rolled out in 13 major cities and will be available nationwide beginning in January 2021.
Supporting the SMA Community During COVID-19
Biogen remains committed to helping the SMA community—throughout the COVID-19 pandemic and beyond. Since announcing the program this summer, we have already distributed hundreds of Patient Care Packages, including masks, gloves, hand sanitizer, and disinfecting wipes to SPINRAZA patients across the country in response to concerns related to COVID-19.
DEVOTE Clinical Trial Part B Enrollment Begins
Biogen also announced at WMS that we have successfully completed enrollment of Part A of the DEVOTE clinical trial.4 This study is evaluating the safety and potential for greater efficacy of a higher dose of SPINRAZA in people with SMA. Study investigators have begun enrolling participants into Part B. More information is available on the study website at www.devotesmastudy.com.
Second Independent Study Expands on Real-World Experience with SPINRAZA in Adults with Later-Onset SMA
CHERISH Study – A Pivotal Study in Children with Later-Onset SMA5
CHERISH was a 15-month randomized, double-blind, sham-controlled study (meaning participants were randomly placed into a treated or untreated group) in 126 individuals with Types 2 and 3 SMA, ages 2 to 9 years old. The study evaluated the effect of SPINRAZA on motor function, including upper limb function. The dosing schedule in the study was different than the approved SPINRAZA schedule.
CHERISH met its primary endpoint, demonstrating a statistically significant improvement in motor function in SPINRAZA-treated patients compared to those who did not receive treatment.
The primary endpoint of CHERISH was change from baseline in the total Hammersmith Functional Motor Scale Expanded (HFMSE) score at month 15. The trial had six secondary endpoints, including the percentage of children who had an increase from baseline to month 15 in the HFMSE score of at least 3 points, the percentage of children who achieved at least one of six new World Health Organization (WHO) motor milestones, and the change from baseline in the Revised Upper Limb Module (RULM) score.
Journal of Neurology, Neurosurgery and Psychiatry – Independent Study in Adults with Later-Onset SMA6
On September 11, 2020, a large supportive retrospective study (n=116) focusing on adults with SMA Types 2 and 3 was published in the Journal of Neurology, Neurosurgery and Psychiatry. This independent study included the largest cohort of adult patients with SMA Type 3 investigated to date in a real-world setting (n=103).
Results show SMA Type 3 patients achieved statistically significant improvements in motor function at the earliest 6-month assessment that further increased at follow-up. The SMA Type 2 subgroup showed positive trends in motor function that were not statistically significant, potentially due to the small number of patients in the cohort (n=13).
Adverse events reported were generally consistent with the SPINRAZA Prescribing Information with the most frequent being post-procedure headache (37.1% of patients), where most resolved within a few days except for five patients who required hospitalization.
Additional Limitations of Journal of Neurology, Neurosurgery and Psychiatry Study
- Randomized controlled clinical trials are considered the gold standard of clinical research to assess the relationship between a treatment and an outcome. Observational studies, like the one reported on in this recent publication, present real-world data gathered outside the rigorous randomized controlled clinical trial process. This observational study had an open recruitment and was not formally powered for efficacy and, therefore, there can be factors that may introduce bias and affect the outcomes.
- Another limitation observed in this study was that some data, mostly limited to timed and pulmonary function tests, were missing for some patients. This is because data is collected outside of a formal clinical study and analyzed after the outcome already occurred.
SPINRAZA® (nusinersen) is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients.
IMPORTANT SAFETY INFORMATION
Increased risk of bleeding complications has been observed after administration of some 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 possible side effects of SPINRAZA include lower respiratory infection, fever, constipation, headache, vomiting, back pain, and post-lumbar puncture syndrome.
These are not all 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.
This information is not intended to replace discussions with your healthcare provider.
- Currey, M et al. “Spinal muscular atrophy and COVID-19: Guidance and resources for providers.” Neurology Live, August 13, 2020, https://www.neurologylive.com/view/spinal-muscular-atrophy-covid19-guidance-resources-for-healthcare-providers-.
- Finkel R, Kirschner J, Mercuri E, et al. Longer-term effects of nusinersen on motor function outcomes based on age at treatment initiation. Presented at WMS25: Virtual Congress, the 25th International Annual Congress of the World Muscle Society; Sept. 28 – Oct. 2. Poster P.264, Poster Session 3.
- Muntoni F, Sumner C, Crawford T, et al. Baseline plasma phosphorylated neurofilament heavy chain level predicts sitting in nusinersen treated individuals with infantile-onset SMA. Presented at WMS25: Virtual Congress, the 25th International Annual Congress of the World Muscle Society; Sept. 28 – Oct. 2. Poster P.171, Poster Session 2.
- Finkel R, Day J, Ryan M, et al. Escalating dose and randomized, controlled study of high-dose nusinersen in SMA; study design and updated enrollment for the DEVOTE Study. Presented at WMS25: Virtual Congress, the 25th International Annual Congress of the World Muscle Society; Sept. 28 – Oct. 2. Poster P.265, Poster Session 3.
- Maggi L, Bello L, Bonanno S, et al. Nusinersen safety and effects on motor function in adult spinal muscular atrophy type 2 and 3. Neurol Neurosurg Psychiatry. 2020 Sep 11:jnnp-2020-323822. doi: 10.1136/jnnp-2020-323822. Epub ahead of print. PMID: 32917822.