US Healthcare Professionals

FIREFISH

Redefining possibilities for infants with Type 1 SMA

FIREFISH is a 2-part, open-label study of Evrysdi in 62 infants aged 2 to 7 months with Type 1 SMA. Part 1 explored the dose and safety of Evrysdi (N=21). Part 2 measured the effectiveness and safety of Evrysdi (N=41).

The effectiveness of Evrysdi in infantile-onset SMA was observed using 2 measurements: Bayley Scales of Infant and Toddler Development-Third Edition (BSID-III) and the Hammersmith Infant Neurological Examination-Module 2 (HINE-2).

Learn more about the trial
What does BSID-III measure?
What does HINE-2 measure?
5-year FIREFISH results

Changing the course of SMA

Infants with Type 1 SMA exceeded expectations for development compared with infants who did not receive treatment
FIREFISH PART 2
MAIN MEASUREMENT
AFTER 1 YEAR

of infants (12/41)*

FIREFISH PART 2
MAIN MEASUREMENT
AFTER 1 YEAR

of infants (12/41)*
 were able to sit without support for at least 5 seconds, as measured by BSID-III

FIREFISH PARTS 1 AND 2
POOLED ANALYSIS
FIREFISH PARTS 1 AND 2

POOLED ANALYSIS

AFTER 1 YEAR

of infants (19/58)*

of infants (19/58)*
were able to sit without support for at least 5 seconds, as measured by BSID-III

AFTER 2 YEARS

of infants (35/58)*

of infants (35/58)*
were able to sit without support for at least 5 seconds, as measured by BSID-III

 were able to sit without support for at least 5 seconds, as measured by BSID-III

Infants with Type 1 SMA are typically not able to sit on their own without treatment

*Infants taking the recommended dose of Evrysdi.

The Bayley Scales of Infant and Toddler Development–Third Edition (BSID-III) is a gross motor scale that assesses a range of physical abilities such as sitting, rolling, and crawling.

Remarkable achievement of key milestones not typically seen in infants without treatment with Type 1 SMA

Of the infants taking the recommended dose of Evrysdi
AFTER 2 YEARS

of infants (23/58) were able to sit without support for 30 seconds, as measured by BSID‑III

AFTER 2 YEARS

of infants (16/58) were able to stand, as measured by HINE‑2

  • 9/58 could stand supporting weight
  • 7/58 could stand with support
  • 0/58 could stand unaided

The Hammersmith Infant Neurological Examination–Module 2 (HINE-2) assesses 8 developmental milestones for infants, including head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking.

Slowing the progression of SMA

EVRYSDI PROLONGED SURVIVAL
AFTER 1 YEAR

of infants (54/62)*

AFTER 2 YEARS

of infants (52/62)*

were alive and able to breathe without permanent support†

Without treatment, no more than 25% of infants with Type 1 SMA are expected to survive beyond 14 months without permanent breathing support

*Infants taking Evrysdi (all dose strengths).
†Permanent support was defined as having a tracheostomy (a surgery where a tube is inserted in the front of the throat into the windpipe) or more than 21 days of either non-invasive ventilation support (16 or more hours a day) or being intubated (a procedure where a breathing tube is inserted down the throat and into the windpipe) to help with breathing, in the absence of an acute reversible event.

EXPLORATORY OBSERVATIONS SUGGEST

Infants taking Evrysdi had better feeding and swallowing abilities compared with infants without treatment*

OF THE INFANTS TAKING THE RECOMMENDED DOSE OF EVRYSDI, AFTER 2 YEARS:

of infants (48/52)
were able to feed orally*

of infants (50/52)
were able to swallow*

Without treatment, 87% of infants with Type 1 SMA typically require feeding
support via a feeding tube by 18 months old

This information is considered exploratory, which means the clinical trial was not specifically designed to show a treatment effect on feeding and swallowing. Data should be interpreted with caution.

*Includes infants who could eat by mouth or in combination with a feeding tube.

EXPLORATORY OBSERVATIONS SUGGEST

Infants taking Evrysdi were able to breathe without permanent support and sit, and some were able to stand

AFTER 5 YEARS

of infants (36/58)*† were able to sit without support for at least 5 seconds, as measured by BSID-III

AFTER 5 YEARS

of infants (34/58)*† were able to sit without support for 30 seconds, as measured by BSID-III

AFTER 5 YEARS

of infants (12/58)*† were able to stand

  • 5/58 could stand supporting weight
  • 3/58 could stand with support
  • 4/58 could stand unaided

As measured by HINE-2

Evrysdi prolonged survival

Recommended-dose cohort (n=58)*

AFTER 5 YEARS

of infants were alive and able to
breathe without permanent support*§

This information is considered exploratory, which means the clinical study was not specifically designed to demonstrate that Evrysdi caused these results. Data should be interpreted with caution.

*Infants taking the recommended dose of Evrysdi.
†The analyses at Year 1 and Year 5 include the pooled population with children from Part 1 (high-dose cohort, n=17) and all children from Part 2 (n=41). Results at Year 1 (data cutoff: November 14, 2019) are based on the assessment of 2 independent central readers, and those at Year 5 (data cutoff: December 22, 2023) are based on the assessment of the site clinical evaluator. Any children not assessed were included as non-responders (BSID-III, n=11; HINE-2 n=10).
‡Percentage based on statistical analysis.
§Permanent support was defined as having a tracheostomy (a surgery where a tube is inserted in front of the throat into the windpipe) or more than 21 days of either non-invasive ventilation support (16 or more hours a day) or being intubated (a procedure where a breathing tube is inserted down the throat and into the windpipe) to help with breathing, in the absence of an acute reversible event.

EXPLORATORY OBSERVATIONS SUGGEST

Infants taking Evrysdi had better feeding and swallowing abilities compared with infants without treatment*

OF THE INFANTS TAKING THE RECOMMENDED DOSE, AFTER 5 YEARS:

of infants (42/46) were able to feed orally†

of infants (46/48) were able to swallow‖

Without treatment, 87% of infants with Type 1 SMA typically require feeding support via a feeding tube by 18 months old

This information is considered exploratory, which means the clinical study was not specifically designed to show a treatment effect on feeding and swallowing. Data should be interpreted with caution.

*Includes infants who could eat by mouth or in combination with a feeding tube.
†These calculations are based on the number of patients assessed for feeding or swallowing at 5 years.

A closer look at the FIREFISH trial

A 2-part, open label study in infants with Type 1 SMA

The study included

62 infants with Type 1 SMA

The main measurement recorded
after 1 year of taking Evrysdi:

  • Sitting without support for at least 5 seconds*
  • Survival without permanent breathing support†

58 infants (aged 2 to 7 months)

who received the recommended dose of Evrysdi in Parts 1 and 2 were included in a pooled analysis that evaluated the effectiveness of Evrysdi

PART 1

Explored the recommended dose of Evrysdi in 21 infants (aged 3 to 7 months)

PART 2

Measured the safety and effectiveness of Evrysdi in 41 infants (aged 2 to 7 months) at the recommended dose

*Measured by Item 22 of the Bayley Scales of Infant and Toddler Development–Third Edition (BSID-III) gross motor scale.
†Permanent support was defined as having a tracheostomy (a surgery where a tube is inserted in the front of the throat into the windpipe) or more than 21 days of either non-invasive ventilation support (16 or more hours a day) or being intubated (a procedure where a breathing tube is inserted down the throat and into the windpipe) to help with breathing, in the absence of an acute reversible event.

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Important Safety Information and Indication

What is Evrysdi?

Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults.

  • Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:
    • are pregnant or plan to become pregnant, as Evrysdi may harm your unborn baby. Ask your healthcare provider for advice before taking this medicine
    • are a woman who can become pregnant:
      • Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy
      • Talk to your healthcare provider about birth control methods that may be right for you. Use birth control while on treatment and for at least 1 month after stopping Evrysdi
      • Pregnancy Registry. There is a pregnancy registry for women who take Evrysdi during pregnancy. The purpose of this registry is to collect information about the health of the pregnant woman and her baby. If you are pregnant or become pregnant while receiving Evrysdi, tell your healthcare provider right away. Talk to your healthcare provider about registering with the Evrysdi Pregnancy Registry. Your healthcare provider can enroll you in this registry or you can enroll by calling 1-833-760-1098 or visiting https://www.evrysdipregnancyregistry.com.
    • are an adult male. Evrysdi may affect a man’s ability to have children (fertility). Ask a healthcare provider for advice before taking this medicine
    • are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby
  • Tell your healthcare provider about all the medicines you take
  • If you were prescribed Evrysdi for Oral Solution, you should receive Evrysdi from the pharmacy as a liquid. If the medicine in the bottle is a powder, do not use it. Contact your pharmacist for a replacement
  • Avoid getting Evrysdi on your skin or in your eyes. If Evrysdi gets on your skin, wash the area with soap and water. If Evrysdi gets in your eyes, rinse your eyes with water
  • The most common side effects of Evrysdi include:
    • For later-onset SMA:
      • fever
      • diarrhea
      • rash
    • For infantile-onset SMA:            
      • fever
      • diarrhea
      • rash
      • runny nose, sneezing, and sore throat (upper respiratory infection)
      • lung infection (lower respiratory infection)
      • constipation
      • vomiting
      • cough

These are not all of the possible side effects of Evrysdi. For more information on the risk and benefits profile of Evrysdi, ask your healthcare provider or pharmacist.

You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.

Please see full Prescribing Information for additional Important Safety Information.

    • Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.

      Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.

    • Atrophy

      The medical term for getting smaller, which is generally what happens to muscles when they are not stimulated by nerve cells.

    • BSID-III

      Stands for the Bayley Scales of Infant and Toddler Development–Third Edition, which is a tool used to assess a range of physical abilities, such as sitting, rolling, and crawling.

    • CI

      Stands for confidence interval, which shows how much a clinical result is likely to vary.

    • DMT

      Stands for disease-modifying treatment, which in the case of SMA, stimulates production of SMN2 protein.

    • FDA

      Stands for Food and Drug Administration.

    • G-tube

      Stands for gastrostomy tube, which is a tube inserted through the belly that brings nutrition directly to the stomach.

    • Gene

      The basic unit of heredity passed from parent to child, made up of sequences of DNA (deoxyribonucleic acid). They contain information that leads to the development of physical traits such as hair color.

    • GI system

      Stands for gastrointestinal system, also called the digestive system, which refers to the group of organs that take in food and liquids and break them down into energy the body can use.

    • HINE-2

      Stands for Hammersmith Infant Neurological Examination–Module 2, which is a tool used to assess 8 developmental milestones for infants, including head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking.

    • Investigational

      Term used to describe medical treatments that are currently being studied in clinical trials to determine how well they work and how safe they are to take.

    • Larynx

      Also known as the voice box, is a hollow tube in the middle of the neck, above the windpipe and esophagus. The larynx allows you to breathe and make sounds.

    • MFM-32

      Stands for Motor Function Measure-32 Items, which is a tool designed to capture changes in head, trunk, and limb motor movements using 32 elements in a broad range of people, including those who cannot walk. It uses 3 main categories: standing/transfer movements, upper/lower body movements, and hand/foot movements.

    • Molecule

      The smallest particle of a substance, made up of one or more atoms.

    • NG-tube

      Stands for nasogastric tube, which is a thin, soft tube that goes in through the nose, down the throat, and into the stomach to provide nutrition or medicine to those who cannot consume by mouth.

    • Non-invasive

      Term used to describe medicines or medical procedures that do not require inserting anything (such as a needle) through the skin or into a body opening.

    • Oral

      Refers to anything having to do with the mouth.

    • PAL

      Stands for Partnership and Access Liaison.

    • Presymptomatic

      When someone has not yet displayed any symptoms but may have underlying presence of the disease, as shown through testing or other means.

    • Previously treated

      When someone has taken either an approved or investigational medication specifically for SMA in the past.

    • Progression

      When the symptoms of SMA get worse over time.

    • Progressive disease

      Any condition that gets worse over time instead of improving.

    • Protein

      Large, complex molecules that play many important roles in the body, including making up the structure of cells and ensuring proper function of tissues and organs.

    • RULM

      Stands for Revised Upper Limb Module, which is a tool specifically designed for people 2.5 years and older living with SMA to evaluate strength in arm movements and the ability to perform certain tasks. It includes tests, such as picking up objects, like coins/tokens, tearing paper, raising a cup to the mouth, and opening a plastic container.

    • Scoliosis

      A sideways curve of the spine. Instead of appearing straight, the spine of a person with scoliosis will have an abnormal S or C shape that leads to their hips or shoulders appearing uneven.

    • Sedation

      A state of calmness, relaxation, or sleepiness as a result of medications called sedatives. Sedation can be used to help you relax or fall asleep for a medical procedure.

    • SMA

      Stands for spinal muscular atrophy, which is a genetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement.

    • SMN

      Stands for survival motor neuron, which is a type of protein that your muscles need to function.

    • Spinal cord

      A tube of tissue that runs from your brain down to your lower back and acts as an important part of your nervous system. The spinal cord carries nerve signals from your brain to the rest of your body, which allow you to feel sensations such as pressure or pain.

    • Type 1 SMA

      SMA symptoms that are present at 6 months of age or earlier. This is sometimes called “infantile onset.” Type 1 SMA is characterized by generalized muscle weakness, a weak cry, and muscle distress, often accompanied by failure to meet developmental milestones such as sitting up unassisted.

    • Type 2 SMA

      SMA symptoms that appear between the ages of 6 to 18 months. People with Type 2 SMA cannot stand or walk without help, but they can sit without support.

    • Type 3 SMA

      SMA symptoms that appear after 18 months of age. People with Type 3 SMA can walk independently but may have difficulty doing so or performing other movements such as running, rising from a chair, or climbing stairs.