SMA Treatment Spinraza (nusinersen) Shows Positive Results in New Data

SMA Treatment Spinraza (nusinersen) Shows Positive Results in New Data

In infants with spinal muscular atrophy (SMA), treatment with Spinraza (nusinersen), the first FDA-approved drug for SMA, reduces the risk of death or permanent ventilation, according to new results from the Phase 3 ENDEAR study (NCT02193074).

Biogen presented the data at the British Paediatric Neurology Association (BPNA) annual conference Jan. 11-13 in Cambridge, England.

The FDA recently approved Spinraza for SMA in children and adults, and the European Medicines Agency (EMA) recently validated a Marketing Authorization Application.

In August 2016, the company  reported the results of an interim analysis of ENDEAR. The analysis found that infants receiving Spinraza experienced a statistically significant improvement in the achievement of motor milestones compared to those who did not receive treatment, as measured by the Hammersmith Infant Neurological Examination (HINE).

The ENDEAR study investigated Spinraza therapy in 121 infantile-onset SMA patients, including patients with SMA signs and symptoms at younger than six months and who were screened at younger than seven months. The end-of-study (EOS) efficacy analysis presented at the BPMA conference refers to the 121 patients who had their final study assessment after the interim analysis (conducted in 78 patients) and who attended the six-month follow-up.

Based on the positive results of the interim analysis, the ENDEAR study was stopped and participants transitioned into the SHINE (NCT02594124) open-label study, where all patients receive Spinraza.

In its presentation, Biogen provided data from the pre-specified primary endpoint, time to death, or permanent ventilation from the EOS analysis. The EOS results demonstrated that Spinraza-treated patients had a statistically significant 47 percent reduction in the risk of death or permanent ventilation compared with untreated patients.

Specifically, compared to Spinraza-treated infants, a greater percentage of untreated infants died or required permanent ventilation (39 percent vs. 68 percent, respectively).

“Although ENDEAR was stopped early based on positive interim results, the study still demonstrated that a significantly greater number of infants treated with SPINRAZA survived and did not require permanent ventilation. These data further underscore the impact SPINRAZA may have on individuals living with this devastating disease,” Wildon Farwell, MD, MPH, senior medical director of clinical development at Biogen, said in a press release.

“We are very encouraged that individuals with SMA have already started treatment with SPINRAZA this week in the U.S., and we continue to work closely with regulatory agencies to bring this therapy to patients around the world as quickly as possible,” Farwell added.

Spinraza demonstrated an acceptable safety profile in the trial, with respiratory events and constipation being the most commonly reported adverse events. These adverse events are consistent with those expected in infants with SMA.

Biogen will report further EOS effectiveness and safety results from the ENDEAR trial at a future medical conference.

Spinraza is being tested in two Phase 3 clinical trials: CHERISH (NCT02193074) (childhood-onset) and ENDEAR (infant-onset), and two Phase 2 studies called EMBRACE (NCT02462759) and NURTURE (NCT02386553).

Spinraza is an antisense oligonucleotide (ASO) designed to alter splicing of SMN2, a gene nearly identical to SMN1, to increase production of fully functional SMN protein.

The U.S Food and Drug Administration (FDA) and the European Medicines Agency (EMA) had granted Spinraza special status intended to expedite the investigative review process, including FDA Orphan Drug designation and Fast Track designation in the U.S. and Orphan Drug designation in the E.U.

The EMA’s Committee for Medicinal Products for Human Use (CHMP) granted Accelerated Assessment status for Spinraza.


  1. Norma Clark says:

    So glad a medication is getting close my first grand baby was born with SMA they finally figured out . We had never heard of it . He lived 14 days . Such a sad time for us still hard some days this was 4 years ago .

    • Tim Bossie says:

      We are sorry for your loss Norma. Time may help heal wounds, but the memories keep them alive in our hearts. With you, we hope that the time is coming quickly when no one else will lose their lives because of this.

  2. Pam Gray says:

    I am a 68 year old female with SMA 3 diagnosed thirty years ago, although we knew something was wrong LONG before that. I am ecstatic that a treatment has been discovered for ALL of the SMA community, but , also interested in whether or not it would be effective in someone my age. If so, how do I find our where, when, who and how much the copay is? Thanks SO much for all the hard work!!!!

  3. Shannon says:

    I to learn.more about this miracle for all of us the love someone with this horrible disease. My niece is 12 and was diagnosed with SMA II AT 16 MONTHS. she is an absolute angel and inspiration to everyone that knows her. My greatest hope is to stop the progession of this disease before its too late.

    • Tim Bossie says:

      We hope that your niece is able to get this medication and that she can benefit from it. Stay tuned to our website, or our FB page for more info when it is released.

  4. Marve Corchado says:

    I’m happy that their is a medication therapy for SMA patient like my daughter was newly diagnosed with SMA 1 and hoping as soon as possible spread that medication around the world specially Middle East a lot of patient affected to this deadly disease. My daughter is an American citizen and a patient in abroad(Middle East) waiting the medication therapy(spinraza) for international.. please

  5. Atilla Karaca says:

    I live in Turkey, I have SMA type III like my two brothers. It’s miraculous news. I was diagnosed when I was 3 yo. I have been waiting for a cure for forty years now. During this time I have lost many friends, relatives, acquaintances and my brother. I wish we can reach that medicament soon.
    Special thanks to all who are involved in developing such a medication.

    • Tim Bossie says:

      We do hope that you and your brothers will be able to benefit from this new treatment. It is such a major advancement in the battle against this illness.

    • Pam Gray says:

      To Atilla, how wonderful that you are still above ground and functioning!! I am 68 and have always had challenges as I am certain you have. Like you, I am very excited about Spinraza, and the possibilities for everyone’s families that are affected by this disease, particularly those infants !!! Now, THEY have a chance! Is there any information about adults OUR age receiving treatment?

  6. Jason Andreou says:

    My son has sma type 3 , he is 16 and unable to walk now and unable to do many things , I’m willing to do what ever it takes for my son to get the medication , we need more info on we’re and when and cost for this treatment , we have been in the dark too many years now . Please help all that suffer , thank you . Jason

  7. Julissa Ramon says:

    My son has sma type 3 , he is 16 and has a hard time walking and is unable to do many things a teenager is able to , I’m willing to do what ever it takes for my son to get the medication , we need more info on we’re and when and cost for this treatment , we have been in the dark too many years now . Please help all that suffer , thank you .

    • Tim Bossie says:

      We do not have any information about availability to the public outside of trials as of now. The cost is rumored to be substantial, but again, we do not know for sure. Sorry we could not bring more information and shed a little light on this for you and your family. We will continue to monitor this and make sure that we report on specifics when we have them.

  8. Pam Gray says:

    Please contact Biogen for detailed information regarding Spinraza,treatment. I did and am currently awaiting a call frommy neurologist to move forward with completion of paperwork to submit to the company for approval of Spinraza treatment! Look up Biogen on the internet!!!

  9. Ehab Abu Alezz says:

    my Daughter 13 years old with SMA II we are in the ,iddle east and such case is very rare I would like to know when Spinraza will be avilable at our aria

    • Tim Bossie says:

      Thank you for reading the post and your question. At this time we do not know where you and your family might be able to receive Spinraza. We do not think it is in your area as of yet. However, please continue to ask your doctor or medical team about it.

  10. Shabbir says:

    My granddaughter of age 4.5 months is diagnosed SMA 1. Can we find this medicine in Pakistan. Does this medicine target to stop deterioration process or it also reverse the same

  11. Tom Duffy says:

    My daughter starts injections in two days. Insurance finally acquiesced and we are thrilled. She is 27 and type 3. Are there any other adults in a similar situation who have had results, positive or negative, to share?

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