First Oral Medication Approved for the Treatment of Spinal Muscular Atrophy

EvrysdiTM is the first oral medication approved for adults and children two months and up with spinal muscular atrophy, a genetic condition affecting the muscles used in movement.  It is available in a liquid form.  Some of the most common side effects include fever, rash, and diarrhea.  For more information about how to administer or any questions about EvrysdiTM, contact your primary care provider or pharmacist.

Share This Post

If you are anything like me, when I think of muscles, I picture someone at the gym flexing in the mirror, taking their daily gym selfie -#ifyoudontpostitdiditreallyhappen!  But our muscles do way more than lift weights.

There are three types of muscle tissue – cardiac, smooth, and skeletal.  All of these muscles work to help the body function.  But what happens if muscles are weak or start to waste away?  When this happens to the muscles used in movement, this is known as spinal muscular atrophy.

There are a few treatment options available for spinal muscular atrophy, but recently the U.S. Federal Drug and Administration (FDA) approved the first oral medication.  Check out below to learn more!

What is Spinal Muscular Atrophy?

Spinal muscular atrophy is a genetic disorder where the muscles used for movement become weak or waste away.  This condition occurs when there is a loss of motor neurons (specialized nerve cells) that control muscle movement. 

 Worldwide, spinal muscular atrophy affects 1 per 8,000 to 10,000 people.  There are multiple types of spinal muscular atrophy:

Type 0

  • Rarest and most severe form
  • Evident before birth
  • Most do not survive past infancy due to respiratory failure

Type 1

  • Most common form
  • Also known as Werdnig-Hoffmann disease
  • Most children cannot control their head movements and may have swallowing problems
  • Most do not survive past early childhood due to respiratory failure

Type 2

  • Develops in children between 6 and 12 months old
  • Also known as Dubowitz disease
  • Most individuals typically cannot stand or walk without support
  • Many live into their twenties or thirties

Type 3

  • Also known as Kugelberg-Welander disease
  • Develops after early childhood
  • Individuals can stand or walk on their own, but over time it becomes more difficult, and they may need to use a wheelchair
  • Individuals have a normal life expectancy

Type 4

  • Rare
  • Begins in early adulthood
  • Individuals experience mild to moderate muscle weakness, mild breathing problems, and tremors
  • Individuals have a normal life expectancy

What is EvrysdiTM?

EvrysdiTM (risdiplam) is a recently approved medication for spinal muscular atrophy in adults and children two months old and up.  EvrysdiTM is the first oral medication available for spinal muscular atrophy available in a liquid form, allowing for at-home treatment.

EvrysdiTM comes in an amber bottle and stays in the refrigerator in an upright position. Check with your pharmacist or primary care provider for full instructions and training on how to take or administer EvrysdiTM if you have questions.

How Does it Work?

Remember that a loss of motor neurons causes spinal muscular atrophy.  EvrysdiTM works by helping to maintain and make motor neurons, leading to improved motor function.

What are the Side Effects?

The most common side effects include:

  • Fever
  • Diarrhea
  • Rash

Other common side effects explicitly seen in infant-onset spinal muscular atrophy include runny nose/sneezing/sore throat/cough, constipation, lung infection, and vomiting.

This is not a full list of side effects for EvrysdiTM.  For more information, contact your primary care provider or pharmacist.

What Do the Studies Say About EvrysdiTM?

The FIREFISH trial looked at infants with type 1 spinal muscular atrophy and investigated the safety and efficacy of EvrysdiTM dosing.  This trial found improvement in the majority of the infant’s ability to breathe without permanent support and improvement in slightly less than half of the infant’s ability to sit without support.

The SUNFISH trial looked at children and adults with types 2 or 3 spinal muscular atrophy, investigating the safety and efficacy of EvrysdiTM dosing.  The results showed improvement in motor function in children and adults. 

The Bottom Line

Spinal muscular atrophy is a genetic condition affecting the muscles involved in movement.  It is caused by a loss of motor neurons, specialized neurons that control muscle movement.

EvrysdiTM is the first oral medication approved for the treatment of spinal muscular atrophy.  It works by helping to make or maintain motor neurons, improving motor function.

Some of the most common side effects include fever, rash, and diarrhea.

The safety and efficacy of EvrysdiTM were studied in infants and adults with type 1, 2, or 3 spinal muscular atrophy in the FIREFISH and SUNFISH trials.

For more information on how to administer or any questions about EvrysdiTM, contact your primary care provider or pharmacist.

References:

Spinal muscular atrophy. Genetics Home Reference website. https://ghr.nlm.nih.gov/condition/spinal-muscular-atrophy. Updated October 2018. Accessed on September 16, 2020.

About Evrysdi. EvrysdiTM website. https://www.evrysdi.com/about-evrysdi.html. Accessed on September 16, 2020.

EvrysdiTM. Instructions for use. Genentech; 2020.

EvrysdiTM. Package insert. Genentech; 2020.

Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy (FIREFISH). U.S. National Library of Medicine website. https://clinicaltrials.gov/ct2/show/NCT02913482. Updated August 24, 2020. Accessed September 16, 2020.

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants (SUNFISH). U.S. National Library of Medicine website. https://clinicaltrials.gov/ct2/show/NCT02908685. Updated July 22, 2020. Accessed September 16, 2020.

Sesame Care

Find the best price for great doctors and specialists

  • Thousands of doctors and specialists
  • $13,000,000+ saved by patients
  • 95% patient satisfaction
  • 4.3 on TrustPilot
     

Popular Destinations

Health

Medication

Telehealth Reviews

Shop

Pharmacist Membership

About Us

Pharmacy Near Me

Recent Articles

Share On:

Subscribe To Our Newsletter

Get updates and learn from the best

More To Explore

How Often Should You Get an Eye Exam?

There is no “one size fits all” answer when it comes to how often you should get an eye exam, as the recommended frequency

The different types of lie bumps: Transient lingual papillitis, fungiform papillae, and other forms

Transient lingual papillitis is a condition where there are transient swellings on your tongue called lie bumps. They can be caused

Iron Infusions: Everything You Need to Know

Iron-deficiency anemia is a common condition that can cause fatigue, shortness of breath, and other health problems. If you are suffering from iron-deficiency anemia,

What Is Genomic Medicine?

Genomic medicine uses our genetic information to provide individualized health care. Various gene therapies can potentially cure rare diseases or conditions. Genetic testing allows

Chigger Bite: Everything You Need to Know 

Do you know what chiggers are? If not, you're not alone. Many people have never heard of chiggers before, but they can be a

What Are the Benefits of Manuka Honey?

Manuka honey is a unique type of honey that is produced in New Zealand and Australia as it is made by bees from the

How Often Can I Take Tylenol?

We’ve all suffered from mild to moderate pain or a fever at some point in our lives, and for many of us, we’ve likely

Coffee: Potential Benefits and Adverse Effects

In this article, we will explore coffee's history, its potential benefits, and if it is safe for everyone to consume.

Parosmia: What it is, Symptoms, Causes, and Treatment

Parosmia is an olfactory disorder where you have a distorted perception of smell and you may smell odors differently than you should. It can

What Is Levofloxacin?

Whether you’re currently fighting off pneumonia, have recently been exposed to inhalational anthrax, or are hoping to prevent infection with the plague (yes, that

Brown Recluse Spider Bites: What You Need to Know

In this article, we will discuss the symptoms of being bitten by a brown recluse spider, when to seek medical attention, and how to

What Is the Drug Levofloxacin Used For?

What happens when your doctor prescribes an antibiotic for a minor infection and it doesn’t work, or you come down with a serious case

Comparison of Azithromycin and Clarithromycin

Azithromycin and clarithromycin are both macrolide antibiotics used to treat a number of different bacterial infections, including upper respiratory tract infections, lower respiratory tract

MCHC: What It Is, Symptoms, Causes, Treatment, and More

Mean corpuscular hemoglobin concentration (MCHC) is a measure of the average amount of hemoglobin in red blood cells with hemoglobin being responsible for transporting

What are the Symptoms of Coffee Withdrawal?

Due to the prevalence of coffee, tea, soft drinks, and energy drinks, caffeine is one of the most popular drugs in the world and

Everything You Ever Wanted to Know About Quercetin

Quercetin is a powerful antioxidant and quercetin supplements are often taken as a way to support good health. Quercetin can also

How Much Does the Shingrix Vaccine Cost?

About 99 percent of American adults have had chickenpox, and many incorrectly assume that this makes them immune to shingles later in life. Shingles,

All You Need to Know About Ringworm: Symptoms, Causes, Diagnosis, and Treatment

Ringworm is a fungal infection that can be seen on the skin. It can affect both children and adults, but it's most common in

CBD Oil: What You Need to Know

CBD oil is a popular natural remedy used for many common ailments. CBD is short for cannabidiol and it is one of over 100 chemical

The Top 7 Best CBD Lotions of 2023

CBD, or cannabidiol, is a cannabinoid that is found in cannabis and hemp plants. It has many supposed beneficial properties for the skin, making