Biogen has provided the following community statement on Spinraza.

Dear members of the SMA community,

Following a busy spring of data presentations at medical congresses and the annual Cure SMA Conference, we are pleased to provide updates on research highlighting SPINRAZA® (nusinersen) data and real-world experience for individuals with spinal muscular atrophy (SMA). Recent updates from Biogen’s clinical development program include results from the NURTURE and SHINE studies as well as the CS2/CS12 integrated analysis. The data provide additional insight on SPINRAZA’s longer-term efficacy, safety and durability profile for infants, children and adults with SMA.

Globally, over 8,400* individuals with SMA have been treated with SPINRAZA, which has been approved in more than 40 countries. In addition, more than 1,000 of the 2,900+ people who have been treated with SPINRAZA across all 50 U.S. states are adults, and 95 percent of individuals who have begun treatment with SPINRAZA remain on therapy today. This real-world experience helps us understand the diverse and growing population of people who have received SPINRAZA treatment to date.

We also enjoyed spending time with the community at the recent annual Cure SMA Conference in Anaheim, CA, where, in addition to sharing the latest clinical data, we were grateful to be able to support opportunities for people of all ages to connect and share experiences. This included a lounge and reception for adult patients, a luncheon symposium for patients and caregivers and at the community-friendly poster session!

A Broad Range of SMA Patients Experience Continuing Benefits With SPINRAZA Treatment: Results from the SHINE study

The SHINE open-label extension study includes patients with infantile-onset (most likely to develop SMA Type 1 or 2) and later-onset SMA (most likely to develop SMA Type 2 or 3) with patients ages 2 to 15 when they received their first dose of SPINRAZA. SHINE patients had participated in previous SPINRAZA clinical trials and are now enrolled in this long-term observational study. Safety was consistent with previous trials.

The study results illustrate SPINRAZA’s longer-term benefits for patients treated for up to nearly four years, as explained in our data presentations at recent conferences such as the American Academy of Neurology (AAN) Annual Meeting, including:

  • Improved Motor Milestones and Survival for Infants: Infantile-onset patients treated with SPINRAZA and followed for up to four years achieved additional or new motor function improvements, such as the ability to sit without support (≤5.42 months, n=30, 60 percent achieved; ≥5.42-≤7.96 months, n=21, 38 percent achieved). These infants also experienced improved event-free survival rates as compared to SMA natural history, as detailed in ENDEAR-SHINE data.3
  • Advanced Scores on Motor Skill Assessment for Infants: In the same study, infants treated with SPINRAZA in both ENDEAR and SHINE increased their average CHOP INTEND scores by 16.8 points after nearly three years of treatment.3
  • Increased Motor Function for Children: Children between two and nine years old with later-onset SMA who initiated SPINRAZA treatment earlier, at an average age of 4.1, experienced greater improvements in motor function compared to those who began treatment later, at an average age of 4.9. While no participants were able to walk at baseline, four were walking independently at nearly two years of follow-up, according to CHERISH-SHINE data.3

Individuals With Later-onset SMA Regained Motor Function With Continued SPINRAZA Treatment: Results From the CS2/CS12 Integrated Analysis

An analysis reinforcing SPINRAZA’s long-term benefits for individuals with later-onset SMA was published in Neurology®, the medical journal of AAN. The data illustrate that continued SPINRAZA treatment may help some older individuals with SMA regain motor function that they had previously lost and stabilize disease activity, allowing them to improve important activities such as walking.

Using data from the CS2/CS12 open-label study, the integrated analysis found that motor function improved for participants treated with SPINRAZA – ages five to 19 at the end of the analysis – notably:

  • Regaining the Ability to Walk: In a first-of-its-kind development, two patients with SMA Type 3 regained the ability to walk independently. One patient with SMA Type 2 who could not walk gained the ability to walk independently, something that has never been observed in untreated individuals with SMA Type 2.4
  • Increasing Distance Walked: Patients with SMA Type 3 increased their ability to walk by an average of 92 meters at approximately three years as measured by the Six Minute Walk Test (6MWT). The average decline in patients with SMA Type 3 who are not treated is 1.5 meters per year.4,5

Biogen is committed to advancing the science of SMA so that we can continue to help all affected by this disease. Additionally, we strive to make SPINRAZA accessible to all through our SMA 360°™ support services program. Please follow this link to explore possible options and connect with one of our Lead Case Managers (LCMs), who are Biogen employees that can help navigate logistics like the insurance landscape.

SMA Awareness Month is on the horizon and we continue to deepen our commitment to supporting individuals with SMA of all ages and across all types. We look forward to sharing additional updates with the SMA community later this year.

Best regards,

The Biogen SMA Team

INDICATION

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 and upper respiratory infections, constipation, headache, 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.

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

References
1. Data on file. Biogen, Cambridge, MA.
2. Biogen. Biogen Announces New Data Further Establishing SPINRAZA® (nusinersen) As a Foundation of Care in Spinal Muscular Atrophy for a Broad Range of Patients [Press release]. 1 Jul. 2019.
3. Biogen. Biogen Presents Data at 2019 AAN Annual Meeting Affirming Longer-Term Safety and Durability of Treatment With SPINRAZA® (nusinersen) [Press release]. 6 May 2019.
4. Darras, Basil T. et al. Nusinersen in later-onset spinal muscular atrophy: long-term results from the phase 1/2 studies. Neurology. 2019; 92:e1-e15. 24 Apr. 2019.
5. Mazzone E, Bianco F, Main M, et al. Six-minute walk test in type III spinal muscular atrophy: a 12-month longitudinal study. Neuromuscular Disord. 2013;23(8):624-628.

* Based on commercial patients, early access patients and clinical trial participants as of July 2019.
† Based on commercial patients in the U.S. (including Puerto Rico) as of July 2019.