For Patients and Caregivers

The first and only at-home treatment for SMA

The first and only at-home treatment for SMA

Evrysdi®️ (risdiplam) is self administered or delivered with help from caretakers
Evrysdi®️ (risdiplam) is a liquid solution icon

A liquid solution

Evrysdi®️ (risdiplam) is taken via oral syringe icon

Taken via oral syringe; either by mouth or feeding tube

Evrysdi®️ (risdiplam) can be self-administered or delivered with help from a caregiver

Self-administered or delivered with help from a caregiver

The liquid solution should be refrigerated (never frozen) in the original amber bottle to protect from light and should be stored in an upright position with the cap tightly closed to avoid spillage.

Evrysdi®️ (risdiplam) should be stored in 36°F to 46°F and should never be frozen

Evrysdi should be refrigerated between 36°F and 46°F for up to 64 days.

If refrigeration is not available, Evrysdi can be kept at room temperature up to 104°F for a combined total of 5 days. It can be removed from, and returned to, a refrigerator. The total combined time out of refrigeration should not exceed 5 days. Any portion of Evrysdi that has been kept above 104˚F should be discarded.

How long can Evrysdi be stored in the refrigerator?

Evrysdi lasts up to 64 days in the refrigerator at a temperature of 36°F to 46°F. After 64 days, any unused product should be discarded. The liquid solution should never be frozen, and should be stored in an upright position with the cap tightly closed to avoid spillage. An expiration date will be indicated on the bottle after constitution by the pharmacist. When traveling, Evrysdi should be stored in an insulated cooler with ice packs at the recommended temperature of 36°F to 46°F.

Can Evrysdi be kept at room temperature?

Evrysdi can be kept at room temperature up to 104°F for a combined total of 5 days. It can be removed from, and returned to, a refrigerator. The total combined time out of refrigeration should not exceed 5 days. Any portion of Evrysdi that has been kept above 104˚F should be discarded.

What should patients and caregivers do if there is an issue with their order (incomplete order or delay in shipment), or if any of the contents of the shipment (bottles, oral syringes, etc) are damaged?

Advise patients or caregivers to contact their specialty pharmacy right away.

Will the syringes provided work with various feeding tubes (ie, G-tube, NG-tube)?

Each monthly shipment of Evrysdi will include ENFit® compatible oral/enteral syringes. The enclosed syringes will work with a variety of feeding tubes, including ENFit® feeding tubes. Specialty pharmacies stock the most common adapters so that syringes will work with most oral/enteral feeding tubes. If the specialty pharmacy does not provide the correct adapter, they will work with you, your patients, and/or caregivers to acquire the appropriate adapter.

What if the bottle of Evrysdi is leaking?

If a patient or caregiver notices leakage on any bottle, advise them to dry off the bottle with a dry paper towel then rinse the closed bottle with water. Store the bottle out of sight and out of reach of children. Call Genentech Product Complaints at 1-800-334-0290 to coordinate free replacement product and further instructions.

Can Evrysdi be picked up from a retail pharmacy?

Evrysdi cannot be filled at a retail pharmacy. Evrysdi will be shipped directly to the patient’s home after it has been prepared by a specialty pharmacy.

Evrysdi®️ (risdiplam) dosing guide for information on how to dose and store

Dosing guide

Find helpful information for how to dose and store Evrysdi

Download

Indication

Evrysdi is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

 

Interactions with Substrates of MATE Transporters

  • Based on in vitro data, Evrysdi may increase plasma concentrations of drugs eliminated via MATE1 or MATE2-K, such as metformin
  • Avoid coadministration of Evrysdi with MATE (multidrug and toxin extrusion) substrates. If coadministration cannot be avoided, monitor for drug-related toxicities and consider dosage reduction of the coadministered drug if needed

Pregnancy & Breastfeeding

  • Evrysdi may cause embryofetal harm when administered to a pregnant woman. In animal studies, administration of Evrysdi during pregnancy and/or lactation resulted in adverse effects on development. Advise pregnant women of the potential risk to the fetus
  • Pregnancy testing is recommended prior to initiating Evrysdi. Advise female patients to use contraception during treatment with Evrysdi and for at least 1 month after the last dose
  • There is a pregnancy exposure registry that monitors pregnancy and fetal/neonatal/infant outcomes in women exposed to Evrysdi during pregnancy. Physicians are encouraged to register patients and pregnant women are encouraged to register themselves by calling 1-833-760-1098 or visiting www.evrysdipregnancyregistry.com
  • The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Evrysdi and any potential adverse effects on the breastfed infant

Potential Effects on Male Fertility

  • Counsel male patients that fertility may be compromised by treatment with Evrysdi. Male patients may consider sperm preservation prior to treatment

Most Common Adverse Reactions

  • The most common adverse reactions in later-onset SMA (incidence in at least 10% of patients treated with Evrysdi and more frequent than control) were fever, diarrhea, and rash
  • The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, adverse reactions with an incidence of at least 10% were upper respiratory tract infection (including nasopharyngitis, rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting, and cough
  • The safety profile for presymptomatic patients is consistent with the safety profile for symptomatic SMA patients treated with Evrysdi in clinical trials

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.

 

 

    • Evrysdi® (risdiplam) Prescribing Information. Genentech, Inc.

      Evrysdi® (risdiplam) Prescribing Information. Genentech, Inc.

    • Data on file. Genentech USA, Inc.

      Data on file. Genentech USA, Inc.

    • NIH US National Library of Medicine. Spinal muscular atrophy. https://ghr.nlm.nih.gov/condition/spinal-muscular-atrophy. Accessed June 7, 2021.

      NIH US National Library of Medicine. Spinal muscular atrophy. https://ghr.nlm.nih.gov/condition/spinal-muscular-atrophy. Accessed June 7, 2021.

    • Farrar MA, Park SB, Vucic S, et al. Emerging therapies and challenges in spinal muscular atrophy. Ann Neurol. 2017;81(3):355-368.

      Farrar MA, Park SB, Vucic S, et al. Emerging therapies and challenges in spinal muscular atrophy. Ann Neurol. 2017;81(3):355-368.

    • Sumner CJ, Crawford TO. Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain. J Clin Invest. 2018;128(8):3219-3227.

      Sumner CJ, Crawford TO. Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain. J Clin Invest. 2018;128(8):3219-3227.

    • Cure SMA. Voice of the patient report: spinal muscular atrophy (SMA). https://curesma.wpengine.com/wp-content/uploads/2018/01/SMA-VoP-for-publication-1-22-2018.pdf. Published January 10, 2018. Accessed June 7, 2021.

      Cure SMA. Voice of the patient report: spinal muscular atrophy (SMA). https://curesma.wpengine.com/wp-content/uploads/2018/01/SMA-VoP-for-publication-1-22-2018.pdf. Published January 10, 2018. Accessed June 7, 2021.

    • Prior TW, Leach ME, Finanger E. Spinal Muscular Atrophy. In: Adam MP, Ardinger HH, Pagon RA, et al., eds. GeneReviews®. Seattle (WA): University of Washington, Seattle; February 24, 2000.

      Prior TW, Leach ME, Finanger E. Spinal Muscular Atrophy. In: Adam MP, Ardinger HH, Pagon RA, et al., eds. GeneReviews®. Seattle (WA): University of Washington, Seattle; February 24, 2000.

    • Hamilton G, Gillingwater TH. Spinal muscular atrophy: going beyond the motor neuron. Trends Mol Med. 2013;19(1):40-50.

      Hamilton G, Gillingwater TH. Spinal muscular atrophy: going beyond the motor neuron. Trends Mol Med. 2013;19(1):40-50.

    • Baranello G, Bloespflug-Tanguy O, Darras BT, et al. FIREFISH Part 1: 24-month safety and exploratory outcomes of risdiplam (RG7916) in infants with Type 1 spinal muscular atrophy (SMA). Supplemental presentation at: 2020 Virtual World Muscle Society; September 28-October 2, 2020; Virtual.

      Baranello G, Bloespflug-Tanguy O, Darras BT, et al. FIREFISH Part 1: 24-month safety and exploratory outcomes of risdiplam (RG7916) in infants with Type 1 spinal muscular atrophy (SMA). Supplemental presentation at: 2020 Virtual World Muscle Society; September 28-October 2, 2020; Virtual.

    • Day JW, Annoussamy M, Baranello G, et al. SUNFISH Part 1: 24-month safety and exploratory outcomes of risdiplam (RG7916) treatment in patients with Type 2 or 3 spinal muscular atrophy (SMA). Presentation at: 2020 Virtual SMA Conference; June 8-12, 2020; Virtual.

      Day JW, Annoussamy M, Baranello G, et al. SUNFISH Part 1: 24-month safety and exploratory outcomes of risdiplam (RG7916) treatment in patients with Type 2 or 3 spinal muscular atrophy (SMA). Presentation at: 2020 Virtual SMA Conference; June 8-12, 2020; Virtual.

    • Chiriboga CA, Bruno C, Duong T, et al. JEWELFISH: safety and pharmacodynamic data in non-naïve patients with spinal muscular atrophy (SMA) receiving treatment with risdiplam. Presentation at: 2020 Virtual CureSMA Conference; June 8-12, 2020; Virtual.

      Chiriboga CA, Bruno C, Duong T, et al. JEWELFISH: safety and pharmacodynamic data in non-naïve patients with spinal muscular atrophy (SMA) receiving treatment with risdiplam. Presentation at: 2020 Virtual CureSMA Conference; June 8-12, 2020; Virtual.

    • Chiriboga CA, Bruno C, Duong T, et al. JEWELFISH: safety and pharmacodynamic data in non-naïve patients with spinal muscular atrophy (SMA) receiving treatment with risdiplam. Presentation at: 2021 Virtual World Muscle Society (WMS) Conference; September 21-24, 2021; Virtual.

      Chiriboga CA, Bruno C, Duong T, et al. JEWELFISH: safety and pharmacodynamic data in non-naïve patients with spinal muscular atrophy (SMA) receiving treatment with risdiplam. Presentation at: 2021 Virtual World Muscle Society (WMS) Conference; September 21-24, 2021; Virtual.

    • Poirier A, Weetall M, Heinig K, et al. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol Res Perspect. 2018;6(6):1-12.

      Poirier A, Weetall M, Heinig K, et al. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol Res Perspect. 2018;6(6):1-12.

    • Baranello G, Darras BT, Day JW, et al. Risdiplam in Type 1 spinal muscular atrophy. N Engl J Med. 2021;384:915-923. doi: 10.1056/NEJMoa2009965.

      Baranello G, Darras BT, Day JW, et al. Risdiplam in Type 1 spinal muscular atrophy. N Engl J Med. 2021;384:915-923. doi: 10.1056/NEJMoa2009965.

    • Darras BT, Baranello G, Boespflug-Tanguy O, et al. FIREFISH Part 1: 24-month safety and exploratory outcomes of risdiplam in infants with Type 1 spinal muscular atrophy (SMA). Presentation at: 2021 Virtual MDA Conference; March 15-18, 2021; Virtual.

      Darras BT, Baranello G, Boespflug-Tanguy O, et al. FIREFISH Part 1: 24-month safety and exploratory outcomes of risdiplam in infants with Type 1 spinal muscular atrophy (SMA). Presentation at: 2021 Virtual MDA Conference; March 15-18, 2021; Virtual.

    • Oskoui M, Day JW, Deconinck N, et al. SUNFISH Part 2: 24-month efficacy and safety of risdiplam in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA). Presentation at: 2021 Virtual MDA Conference; March 15-18, 2021; Virtual.

      Oskoui M, Day JW, Deconinck N, et al. SUNFISH Part 2: 24-month efficacy and safety of risdiplam in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA). Presentation at: 2021 Virtual MDA Conference; March 15-18, 2021; Virtual.

    • Mercuri E, Barisic N, Boespflug-Tanguy O, et al. SUNFISH Part 2: efficacy and safety of risdiplam (RG7916) in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA). Presentation at: 2020 Virtual American Academy of Neurology (AAN) Conference; April 25-May 1, 2020; Virtual.

      Mercuri E, Barisic N, Boespflug-Tanguy O, et al. SUNFISH Part 2: efficacy and safety of risdiplam (RG7916) in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA). Presentation at: 2020 Virtual American Academy of Neurology (AAN) Conference; April 25-May 1, 2020; Virtual.

    • Clinicaltrials.gov. A study of risdiplam in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (RAINBOWFISH). [NCT03779334]. https://clinicaltrials.gov/ct2/show/NCT03779334. Last update posted June 7, 2021. Accessed June 7, 2021.

      Clinicaltrials.gov. A study of risdiplam in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (RAINBOWFISH). [NCT03779334]. https://clinicaltrials.gov/ct2/show/NCT03779334. Last update posted June 7, 2021. Accessed June 7, 2021.

    • Bérard C, Payan C, Hodgkinson I, et al., and the MFM Collaborative Study Group. A motor function measure scale for neuromuscular diseases. Construction and validation study. Neuromuscul Disord. 2005;15:463-470.

      Bérard C, Payan C, Hodgkinson I, et al., and the MFM Collaborative Study Group. A motor function measure scale for neuromuscular diseases. Construction and validation study. Neuromuscul Disord. 2005;15:463-470.

    • Trundell D, Le Scouiller S, Le Goff L, et al. Assessment of the validity and reliability of the 32-item Motor Function Measure in individuals with Type 2 or non-ambulant Type 3 spinal muscular atrophy. PLoS One. 2020;15(9):e0238786. doi: 10.1371/journal. pone.0238786.

      Trundell D, Le Scouiller S, Le Goff L, et al. Assessment of the validity and reliability of the 32-item Motor Function Measure in individuals with Type 2 or non-ambulant Type 3 spinal muscular atrophy. PLoS One. 2020;15(9):e0238786. doi: 10.1371/journal. pone.0238786.

    • Vuillerot C, Payan C, Iwaz J, et al. Responsiveness of the motor function measure in patients with spinal muscular atrophy. Arch Phys Med Rehabil. 2013;94(8):1555-1561.

      Vuillerot C, Payan C, Iwaz J, et al. Responsiveness of the motor function measure in patients with spinal muscular atrophy. Arch Phys Med Rehabil. 2013;94(8):1555-1561.

    • Mazzone ES, Mayhew A, Montes J, et al. Revised upper limb module for spinal muscular atrophy: development of a new module. Muscle Nerve. 2017;55:869-874.

      Mazzone ES, Mayhew A, Montes J, et al. Revised upper limb module for spinal muscular atrophy: development of a new module. Muscle Nerve. 2017;55:869-874.

    • Pera MC, Coratti G, Mazzone ES, et al. Revised upper limb module for spinal muscular atrophy: 12 month changes. Muscle Nerve. 2019;59:426-430.

      Pera MC, Coratti G, Mazzone ES, et al. Revised upper limb module for spinal muscular atrophy: 12 month changes. Muscle Nerve. 2019;59:426-430.

    • Mazzella A, Curry M, Belter L, et al. “I have SMA, SMA doesn't have me”: a qualitative snapshot into the challenges, successes, and quality of life of adolescents and young adults with SMA. Orphanet J Rare Dis. 2021;16(1):96.

      Mazzella A, Curry M, Belter L, et al. “I have SMA, SMA doesn't have me”: a qualitative snapshot into the challenges, successes, and quality of life of adolescents and young adults with SMA. Orphanet J Rare Dis. 2021;16(1):96.

    • Wan HWY, Carey KA, D’Silva A, et al. “Getting ready for the adult world”: how adults with spinal muscular atrophy perceive and experience healthcare, transition and well-being. Orphanet J Rare Dis. 2019;14(1):74.

      Wan HWY, Carey KA, D’Silva A, et al. “Getting ready for the adult world”: how adults with spinal muscular atrophy perceive and experience healthcare, transition and well-being. Orphanet J Rare Dis. 2019;14(1):74.

    • Albers CA, Grieve AJ. Test review: Bayley, N. (2006). Bayley Scales of Infant and Toddler Development–Third Edition. San Antonio, TX: Harcourt Assessment. J Psychoeduc Assess. 2007;25(2):180-198.

      Albers CA, Grieve AJ. Test review: Bayley, N. (2006). Bayley Scales of Infant and Toddler Development–Third Edition. San Antonio, TX: Harcourt Assessment. J Psychoeduc Assess. 2007;25(2):180-198.

    • Darras BT, Boespflug-Tanguy O, Day JW, et al, on behalf of the FIREFISH Working Group. FIREFISH Parts 1 and 2: 24-month safety and efficacy of risdiplam in infants with Type 1 SMA. Poster presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 13-16, 2022; Nashville, TN.

      Darras BT, Boespflug-Tanguy O, Day JW, et al, on behalf of the FIREFISH Working Group. FIREFISH Parts 1 and 2: 24-month safety and efficacy of risdiplam in infants with Type 1 SMA. Poster presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 13-16, 2022; Nashville, TN.

    • Finkel RS, Farrar MA, Vlodavets D, et al, on behalf of the RAINBOWFISH Study Group. RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic SMA. Poster presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 13-16, 2022; Nashville, TN.

      Finkel RS, Farrar MA, Vlodavets D, et al, on behalf of the RAINBOWFISH Study Group. RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic SMA. Poster presented at: Muscular Dystrophy Association Clinical and Scientific Conference; March 13-16, 2022; Nashville, TN.

    • Baranello G, Day JW, Deconinck N, et al, on behalf of the SUNFISH Working Group. SUNFISH: efficacy and safety of risdiplam in Types 2 and 3 SMA. Poster presented at: European Paediatric Neurology Society; April 28 - May 2, 2022; Glasgow, UK.

      Baranello G, Day JW, Deconinck N, et al, on behalf of the SUNFISH Working Group. SUNFISH: efficacy and safety of risdiplam in Types 2 and 3 SMA. Poster presented at: European Paediatric Neurology Society; April 28 - May 2, 2022; Glasgow, UK.