Bigoen has provided the below community statement on Spinraza.
Dear Members of the SMA community,
As we approach the two-year anniversary of the U.S. Food and Drug Administration’s (FDA) approval of SPINRAZA® (nusinersen), we want to thank you for your ongoing support. We share your commitment to advancing research to improve the lives of those with SMA, their caregivers and communities.
We are pleased to announce that nearly 6,0001 individuals with SMA have been treated with SPINRAZA worldwide – more than 2,4002 of whom come from the U.S., where all 50 states have at least one treatment center.3 To help the community find the closest treatment center, the following link is available to help. We are encouraged by the range of patients, from infants to adults, who have benefited from SPINRAZA. Additionally, for those that choose SPINRAZA therapy, greater than 95 percent remain on treatment.2 We have also continued to make progress in supporting the establishment of adult treatment sites as adults represent roughly one-third2 of patients in the U.S. treated with SPINRAZA.
We recognize that those living with SMA have many questions about ongoing disease and treatment education. Our Support Treatment Education Program (STEP) offers an opportunity for caregivers and patients to hear firsthand from experienced healthcare providers about SMA and SPINRAZA and connect with others in the community. For more information please click here.
We continue to advance our SMA research through our SPINRAZA clinical development program, which in total contains more than six years of data4– to work toward our goal of benefiting those with SMA. The past months have included key developments in our program with new clinical data and the acquisition of a muscle-strengthening product development program, which are detailed below.
Expanding Scientific Understanding of SMA with New Research
At the 23rd International Annual Congress of the World Muscle Society (WMS) in Mendoza, Argentina, we presented research on the natural history of SMA and data from our ongoing clinical trials. Interim results from NURTURE, the ongoing, open-label, single arm, safety and efficacy study of SPINRAZA among presymptomatic infants with SMA, showed that all participants treated with SPINRAZA (n=25) were alive, did not require permanent ventilation and were able to sit without support, as well as 22 out of 25 were able to walk with assistance and 17 out of 25 were able to walk independently.
These motor milestones, achieved after a median 27.1 months on study, demonstrate that SPINRAZA can positively impact the course of the disease in children who initiate treatment during a presymptomatic phase.
We continue to see progress in the evaluation of alternative methods of injection of SPINRAZA for individuals with complex spines. Techniques such as imaging-guided injection or the use of intrathecal catheters have resulted in promising results.
We continue to investigate neurofilament as a potential biomarker in SMA and will continue to evaluate how it can inform further research and deepen scientific knowledge of SMA.
Broadening Our Development Portfolio
Earlier this year, we were pleased to announce the acquisition of a muscle-strengthening program from AliveGen, Inc., which included BIIB110, a protein that targets the myostatin pathway. We plan to study this approach in multiple neuromuscular diseases, including SMA, and are encouraged by the program’s potential to provide additional benefits for patients.
As previously shared during the Biogen Q2 earnings call, the investigational new drug (IND) application for our SMA gene therapy program is currently on clinical hold in the U.S. We are currently assessing options to determine the extent to which we have a viable path forward.
We are grateful for your support and participation in clinical studies to further expand scientific understanding of SMA and the benefits that SPINRAZA can provide for individuals of all ages. As the community of people treated with SPINRAZA grows, we will continue to advance our SMA research and SPINRAZA clinical development program to identify potential new ways to help patients. We look forward to providing additional updates in the months ahead.
The Biogen SMA Team
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 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.
1 Based on commercial patients, early access patients, and clinical trial participants as of September 2018.
2 Biogen data on file. Based on commercial patients as of September 2018.
3 Biogen data on file. Based on commercial patients as of September 2018. Includes Puerto Rico.
4 Biogen data on file.