New Spinal Muscular Atrophy Treatments 2021



Article last updated on 7/29/2021


What is Spinal Muscular Atrophy?

Spinal Muscular Atrophy (SMA) is an inherited disorder that affects nerves and muscles, which causes muscles to become increasingly weak. The cause is a shortage of the protein SMN, or ‘survival of motor neuron’. This protein is crucial for the function of nerves that control muscle activity. Without this protein, the motor neuron cells shrink and eventually die. This leads to muscle weakness seen in SMA.1

Treatment for Spinal Muscular Atrophy

There are different SMA medications approved that help to increase and maintain the amount of SMN protein in the body. Thereby it is expected to improve motor neuron function and slow down the progression of SMA. This type of SMA therapy is often called “SMN-based” or “SMN-enhancing” approach.1,2

Approved treatments work by either replacing the function of the missing or nonworking SMN1 gene with a new, working copy of an SMN gene (gene replacement therapy), or by fixing splicing errors in genes such as SMN2 (disease-modifying therapy).1,2


Is there a cure for Spinal Muscular Atrophy?

Currently, there are no cures for spinal muscular atrophy, but research is ongoing to find new treatments.


What are new Spinal Muscular Atrophy treatments?

There are several approved new treatments for SMA. Here are some of them:

Evrysdi (risdiplam)3,4

Evrysdi (risdiplam) is a disease-modifying therapy used to treat SMA in adults and children 2 months of age and older.3 It is a daily oral medication that can be given by mouth or feeding tube. Evrysdi (risdiplam) must be taken for the duration of the patient’s life.4 Evrysdi (risdiplam) was approved for the treatment of people with SMA by:3 The Food and Drug Administration (FDA), USA on August 7, 2020 The European Medicines Agency (EMA), Europe on March 26, 2021 Health Canada on April 15, 2021

Zolgensma (onasemnogene abeparvovec)5,6

Zolgensma (onasemnogene abeparvovec) is a gene replacement therapy, or gene therapy, for spinal muscular atrophy (SMA). It is used to treat patients with all forms and types of SMA who are under 2 years of age at the time of dosing.5 It is a one-time-only dose that is given through an intravenous infusion that takes about an hour.6 Zolgensma (onasemnogene abeparvovec) was approved for the treatment of children aged 2 years and below with SMA by:5 Food and Drug Administration (FDA), USA on May 24, 2019 European Medicines Agency (EMA) on May 18, 2020 Health Canada on December 16, 2020

Spinraza (nusinersen)7,8

Spinraza (nusinersen) is an antisense oligonucleotide used to treat SMA patients of all ages and types of SMA.7

Nusinersen is injected directly into the spinal canal (intrathecally). Patients receive four “loading doses” within the first 2 months of treatment. Once those loading doses are completed, a maintenance dose will be given every 4 months for the duration of the patient’s life.8

Spinraza (nusinersen) was approved for the treatment of children and adults with spinal muscular atrophy (SMA) by:7

  • The Food and Drug Administration (FDA), USA on December 23, 2016
  • The European Medicines Agency (EMA) on May 30, 2017
  • The Therapeutic Goods Administration (TGA), Australia on November 2, 2017
  • Health Canada on August 29, 2017
  • Medsafe, New Zealand on August 23, 2018
  • National Medical Products Administration (NMPA), China in February 2019

If you are trying to access new SMA drugs that are approved outside of your country of residence, we might be able to help you access it with the support of your treating doctor. You can read more about the medicines we can help you and your doctor access and about their price below:



Why access and buy treatments for SMA with TheSocialMedwork?

TheSocialMedwork is registered in The Hague with the Dutch Ministry of Health as an independent medicines intermediary (registration number 6730 BEM) and as an international pharmacy (registration number 16258 G). We have helped patients from over 85 countries to access thousands of medicines that are not yet approved in their home country, including patients suffering from Psoriatic Arthritis. With a prescription from your treating doctor, you can count on our expert team to safely and legally guide you to access Psoriatic Arthritis medicines in your country. If you or someone you know are looking to access a medicine that is not yet approved where you live, we will support you. Contact us for more information.


References:

  1. clevelandclinic.org
  2. cureSMA.org
  3. Evrysdi (risdiplam)
  4. FDA Evrysdi
  5. Zolgensma (onasemnogene abeparvovec)
  6. FDA Zolgensma
  7. Spinraza (nusinersen)
  8. FDA Spinraza

Disclaimer: This article is not meant to influence or impact the care provided by your treating physician. Please do not make changes to your treatment without first consulting your healthcare provider. This article is not intended to diagnose or treat illness or to influence treatment options. TheSocialMedwork is as diligent as possible in compiling and updating the information on this page. However, TheSocialMedwork does not guarantee the correctness and completeness of the information provided on this page.