EVRYSDI (Page 5 of 6)

14.3 Pre-Symptomatic SMA

Study 3 is an open-label, single-arm, multicenter clinical study to investigate the efficacy, safety, pharmacokinetics, and pharmacodynamics of EVRYSDI in infants up to 6 weeks of age (at first dose) who have been genetically diagnosed with SMA but do not yet present with symptoms.

At the time of an interim analysis, a total of 18 patients with pre-symptomatic SMA were enrolled in Study 3. The efficacy in pre-symptomatic SMA patients was evaluated in 7 patients who had been treated with EVRYSDI for at least 12 months: four patients had 2 copies of the SMN2 gene, 2 patients had 3 copies, and 1 patient had 4 or more copies. Of these 7 patients, the median age at first dose was 35 days (range: 16 to 40 days), 71% were female, 100% were Caucasian.

The 6 patients with 2 or 3 copies of SMN2 achieved the following motor milestones as measured by the HINE-2 at Month 12: 6 (100%) patients achieved sitting (5 patients could pivot/rotate and 1 patient achieved stable sit); 4 (67%) patients could stand (3 patients could stand unaided and 1 patient could stand with support), and 3 (50%) patients could walk independently. All 6 patients were alive at 12 months without permanent ventilation.

16 HOW SUPPLIED/STORAGE AND HANDLING

16.1 How Supplied

Each amber glass bottle of EVRYSDI is packaged with a bottle adapter, two 6 mL reusable oral syringes, and two 12 mL reusable oral syringes. EVRYSDI for oral solution is a light yellow, yellow, greyish yellow, greenish yellow, or light green powder. Each bottle contains 60 mg of risdiplam (NDC 50242-175-07).

Additional 1 mL reusable oral syringes will be provided by the pharmacy as detailed in the Instruction for Constitution.

16.2 Storage and Handling

Store the dry powder at 20°C to 25°C (68°F to 77°F), excursions permitted between 15°C to 30°C (59°F to 86°F) [see USP controlled room temperature]. Keep in the original carton.

Keep the constituted oral solution of EVRYSDI in the original amber bottle to protect from light. Store in a refrigerator at 2°C to 8°C (36°F to 46°F) [see Dosage and Administration (2.4)].

17 PATIENT COUNSELING INFORMATION

Advise the patient to read the FDA-approved patient labeling (Patient Information and Instructions for Use).

Pregnancy and Fetal Risk

Inform pregnant women and women of reproductive potential that, based on animal studies, EVRYSDI may cause fetal harm [see Use in Specific Populations (8.1)].

Discuss with women of childbearing age whether they are pregnant, might be pregnant, or are trying to become pregnant.

Advise women of childbearing potential to use effective contraception during treatment with EVRYSDI and for at least 1 month after stopping EVRYSDI.

Advise a female patient to immediately inform the prescriber if she is pregnant or planning to become pregnant [see Use in Specific Populations (8.3)].

Potential Effects on Male Fertility

Advise male patients that their fertility may be compromised while on treatment with EVRYSDI [see Use in Specific Populations (8.3)].

Instructions for Preparation of Oral Solution

Advise patients to ensure that EVRYSDI is in liquid form when received from the pharmacy.

Instruct patients/caregivers to take EVRYSDI after a meal or after breastfeeding at approximately the same time each day. However, instruct caregivers to not mix EVRYSDI with formula or milk.

Instruct patients/caregivers to take EVRYSDI immediately after it is drawn up into the reusable oral syringe [see Dosage and Administration (2.1)].

EVRYSDI® [risdiplam]

Distributed by:
Genentech, Inc.
A Member of the Roche Group
1 DNA Way
South San Francisco, CA 94080-4990

EVRYSDI is a registered trademark of
Genentech, Inc.©2022 Genentech, Inc. All rights reserved.

This Patient Information has been approved by the U.S. Food and Drug Administration. Approved: 5/2022
Patient InformationEVRYSDI® [ev-RIZ-dee] (risdiplam)for oral solution
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. If you are pregnant, or are planning to become pregnant, ask your healthcare provider for advice before taking this medicine. EVRYSDI may harm your unborn baby.
  • are a woman who can become pregnant:
    • Before you start your treatment with EVRYSDI, your healthcare provider may test you for pregnancy. Because EVRYSDI may harm your unborn baby, you and your healthcare provider will decide if taking EVRYSDI is right for you during this time.
    • 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.
  • are an adult male planning to have children: EVRYSDI may affect a man’s ability to have children (fertility). If this is of concern to you, make sure to ask a healthcare provider for advice.
  • are breastfeeding or plan to breastfeed. It is not known if EVRYSDI passes into breast milk and may harm your baby. If you plan to breastfeed, discuss with your healthcare provider about the best way to feed your baby while on treatment with EVRYSDI.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Keep a list of them to show your healthcare provider, including your pharmacist, when you get a new medicine.
How should I take EVRYSDI?See the detailed Instructions for Use that comes with EVRYSDI for information on how to take or give EVRYSDI oral solution.
  • You should receive EVRYSDI from the pharmacy as a liquid that can be given by mouth or through a feeding tube. The liquid solution is prepared by your pharmacist or other healthcare provider. 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.
Taking EVRYSDI
  • Your healthcare provider will tell you how long you or your child needs to take EVRYSDI. Do not stop treatment with EVRYSDI unless your healthcare provider tells you to.
  • For infants and children, your healthcare provider will determine the daily dose of EVRYSDI needed based on your child’s age and weight. For adults, take 5 mg of EVRYSDI daily.
    • Take EVRYSDI exactly as your healthcare provider tells you to take it. Do not change the dose without talking to your healthcare provider.
  • Take EVRYSDI 1 time daily after a meal (or after breastfeeding for a child) at approximately the same time each day. Drink water afterwards to make sure EVRYSDI has been completely swallowed.
  • Do not mix EVRYSDI with formula or milk.
  • If you are unable to swallow and have a nasogastric or gastrostomy tube, EVRYSDI can be given through the tube.
  • If you miss a dose of EVRYSDI:
    • If you remember the missed dose within 6 hours of when you normally take EVRYSDI, then take or give the dose. Continue taking EVRYSDI at your usual time the next day.
    • If you remember the missed dose more than 6 hours after you normally take EVRYSDI, skip the missed dose. Take your next dose at your usual time the next day.
  • If you do not fully swallow the dose, or you vomit after taking a dose, do not take another dose of EVRYSDI to make up for that dose. Wait until the next day to take the next dose at your usual time.
Reusable Oral Syringes
  • Your pharmacist will provide you with the reusable oral syringes that are needed for taking your medicine and explain how to use them. Wash the syringes per instructions after use. Do not throw them away.
  • Use the reusable oral syringes provided by your pharmacist (you should receive 2 identical oral syringes) to measure your or your child’s dose of EVRYSDI, as they are designed to protect the medicine from light. Contact your healthcare provider or pharmacist if your oral syringes are lost or damaged.
  • When transferred from the bottle to the oral syringe, take EVRYSDI right away. Do not store the EVRYSDI solution in the syringe. If EVRYSDI is not taken within 5 minutes of when it is drawn up, EVRYSDI should be thrown away from the reusable oral syringe, and a new dose should be prepared.
What are the possible side effects of EVRYSDI? 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, ask your healthcare provider or pharmacist.Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
How should I store EVRYSDI?
  • Store EVRYSDI in the refrigerator between 36°F to 46°F (2°C to 8°C). Do not freeze.
  • Keep EVRYSDI in an upright position in the original amber bottle to protect from light.
  • Throw away (discard) any unused portion of EVRYSDI 64 days after it is mixed by the pharmacist (constitution). Please see the Discard After date written on the bottle label. (See the Instructions for Use that comes with EVRYSDI).
Keep EVRYSDI and all medicines out of the reach of children.
General information about the safe and effective use of EVRYSDI. Medicines are sometimes prescribed for purposes other than those listed in a Patient Information leaflet. Do not use EVRYSDI for a condition for which it was not prescribed. Do not give EVRYSDI to other people, even if they have the same symptoms you have. It may harm them. You can ask your pharmacist or healthcare provider for information about EVRYSDI that is written for health professionals.
What are the ingredients in EVRYSDI?Active ingredient: risdiplamInactive ingredients: ascorbic acid, disodium edetate dihydrate, isomalt, mannitol, polyethylene glycol 6000, sodium benzoate, strawberry flavor, sucralose, and tartaric acid.Distributed by: Genentech, Inc. , A Member of the Roche Group, 1 DNA Way, South San Francisco, CA 94080-4990EVRYSDI is a registered trademark of Genentech, Inc.©2022 Genentech, Inc. All rights reserved.For more information, go to www.EVRYSDI.com or call 1-833-387-9734.

INSTRUCTIONS FOR USE
EVRYSDI® [ev-RIZ-dee]

(risdiplam) for oral solution

Please read and understand this Instructions for Use and the Patient Information leaflet before you start taking EVRYSDI for information about EVRYSDI and how to prepare and give EVRYSDI through an oral syringe, gastrostomy tube (G-tube), or nasogastric tube (NG-tube).

If you have any questions about how to take EVRYSDI, contact your healthcare provider.

EVRYSDI should come as a liquid in a bottle when you receive it from the pharmacy. Do not take EVRYSDI and contact your pharmacist if the medicine in the bottle is a powder.

Each EVRYSDI carton contains (see Figure A):

Image1 Cap
Figure A
(click image for full-size original)
Image1 Bottle adapter
Image1 EVRYSDI bottle
Image2 Reusable oral syringes
Image1 Instructions for Use (not shown)
Image1 Prescribing Information and Patient Information (not shown)
Reusable Oral Syringe Overview (see Figure B)
Figure B
(click image for full-size original)

Important information about EVRYSDI

  • Ask your healthcare provider to show you the correct oral syringe you should use and how to measure your prescribed daily dose.
  • Always use the reusable oral syringes that come with EVRYSDI to measure your prescribed daily dose. If your carton does not contain two identical syringes, contact your pharmacist.
  • Always take EVRYSDI exactly as your healthcare provider tells you to take it.
  • Take EVRYSDI 1 time daily after a meal at approximately the same time each day.
  • Do not take EVRYSDI if the bottle adapter is not in the bottle. If the bottle adapter is not in the bottle, contact your pharmacist.
  • Do not mix EVRYSDI into food or liquids. Do not mix EVRYSDI with formula or milk.
  • Do not take EVRYSDI if the bottle or oral syringes are damaged.
  • 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.
  • If you spill EVRYSDI, dry the area with a dry paper towel and then clean with water. Throw away the paper towel in the trash and wash your hands well with soap and water.
  • If there is not enough EVRYSDI left in the bottle for your prescribed dose, throw away (discard) the bottle with remaining EVRYSDI and used oral syringes according to your local requirements.
  • Use a new bottle of EVRYSDI to get your prescribed dose.Do not mix EVRYSDI from the new bottle with the bottle you are currently using.

How to store EVRYSDI

  • Store EVRYSDI in the refrigerator between 36°F to 46°F (2°C to 8°C). Do not freeze.
  • Store EVRYSDI in the original amber bottle in an upright position with the cap tightly closed.
  • Throw away (discard) any unused portion of EVRYSDI 64 days after constitution. Please see the Discard After date written on the bottle label (see Figure C).
  • Ask your pharmacist for the Discard After date if it is not written on the bottle label.
  • Keep EVRYSDI and all medicines and syringes out of the reach of children.
Figure C
(click image for full-size original)

A) Preparing and withdrawing your dose

How to prepare your dose of EVRYSDI

Figure DFigure DStep A1 Remove the cap by pushing it down and then twisting the cap to the left (counterclockwise) (See Figure D).Do not throw away the cap.
Figure EFigure EStep A2Push the plunger of the oral syringe all the way down to remove any air in the oral syringe (See Figure E).
Figure F
(click image for full-size original)
Step A3 Place the EVRYSDI bottle on a flat surface. While keeping the bottle in an upright position, insert the syringe tip into the bottle adapter (See Figure F).
Figure GFigure GStep A4 Carefully turn the bottle upside down with the syringe tip firmly inserted into the bottle adapter (See Figure G).
Figure H
(click image for full-size original)
Step A5 Slowly pull back on the plunger to withdraw your prescribed dose of EVRYSDI.The top of the black plunger stopper must line up with the mL marking on the oral syringe for your prescribed daily dose (See Figure H).After the correct dose is withdrawn, hold the plunger in place to keep the plunger from moving.
Figure I
(click image for full-size original)
Step A6Continue to hold the plunger in place to keep the plunger from moving. Leave the oral syringe in the bottle adapter and turn the bottle to an upright position. Place the bottle onto a flat surface. Remove the oral syringe from the bottle adapter by gently pulling straight up on the oral syringe while holding the plunger in place (See Figure I).
Figure J
(click image for full-size original)
Step A7 Hold the oral syringe with the syringe tip pointing up. Check the EVRYSDI in the oral syringe. If there are large air bubbles in the oral syringe (See Figure J) or if you have drawn up the wrong dose of EVRYSDI, insert the syringe tip firmly into the bottle adapter while the bottle is in an upright position. Push the plunger all the way down so that EVRYSDI flows back into the bottle and repeat Steps A4 through A7.Take or give EVRYSDI right away after it is drawn up into the oral syringe. If it is not taken within 5 minutes, throw away EVRYSDI liquid from your oral syringe into the household trash. Do this by pushing the plunger all the way down to remove EVRYSDI from the oral syringe. Prepare a new dose starting with Step A2.
Figure KFigure KStep A8 Put the cap back on the bottle. Turn the cap to the right (clockwise) to tightly close the bottle (See Figure K). Do not remove the bottle adapter from the bottle.

If you are taking your dose of EVRYSDI by mouth, follow the instructions in “B) How to take a dose of EVRYSDI by mouth“.

If you are taking your dose of EVRYSDI through a gastrostomy tube, follow the instructions in “C) How to give a dose of EVRYSDI through a gastrostomy tube“.

If you are taking your dose of EVRYSDI through a nasogastric tube, follow the instructions in “D) How to give a dose of EVRYSDI through a nasogastric tube“.

B) How to take a dose of EVRYSDI by mouth

Sit upright when taking a dose of EVRYSDI by mouth.

Figure L
(click image for full-size original)
Step B1 Place the oral syringe into the mouth with the tip along either cheek.Slowly push the plunger all the way down to give the full dose of EVRYSDI (See Figure L).Giving EVRYSDI into the throat or too fast may cause choking.
Figure MFigure MStep B2 Check that there is no EVRYSDI left in the oral syringe (See Figure M).
Figure N
(click image for full-size original)
Step B3 Drink about a tablespoon (15 mL) of water right after taking the prescribed dose of EVRYSDI to ensure the drug has been completely swallowed (See Figure N).Go to Step E for cleaning of the syringe.

C) How to give a dose of EVRYSDI through a gastrostomy tube

If you are giving EVRYSDI through a gastrostomy tube, ask your healthcare provider to show you how to inspect the gastrostomy tube before giving EVRYSDI.

Figure O
(click image for full-size original)
Step C1 Place the oral syringe tip into the gastrostomy tube. Slowly push the plunger all the way down to give the full dose of EVRYSDI (See Figure O).
Figure PFigure PStep C2 Check that there is no EVRYSDI left in the oral syringe (See Figure P).
Figure Q
(click image for full-size original)
Step C3 Flush the gastrostomy tube with 10 mL to 20 mL of water right after giving the prescribed dose of EVRYSDI (See Figure Q).Go to Step E for cleaning of the syringe.

D) How to give a dose of EVRYSDI through a nasogastric tube

If you are giving EVRYSDI through a nasogastric tube, ask your healthcare provider to show you how to inspect the nasogastric tube before giving EVRYSDI.

Figure R
(click image for full-size original)
Step D1 Place the oral syringe tip into the nasogastric tube. Slowly press the plunger all the way down to give the full dose of EVRYSDI (See Figure R).
Figure SFigure SStep D2 Check that there is no EVRYSDI left in the oral syringe (See Figure S).
Figure T
(click image for full-size original)
Step D3 Flush the nasogastric tube with 10 mL to 20 mL of water right after giving the prescribed dose of EVRYSDI (See Figure T).Go to Step E for cleaning of the syringe.

E) How to clean the oral syringe after use

Figure U
(click image for full-size original)
Step E1 Remove the plunger from the oral syringe by pulling the plunger away from the syringe until the plunger comes out of the syringe.Rinse the oral syringe barrel well under clean water (See Figure U).
Figure V
(click image for full-size original)
Step E2 Rinse the plunger well under clean water (See Figure V).
Figure W
(click image for full-size original)
Step E3 Check that the oral syringe barrel and plunger are clean.Place the oral syringe barrel and plunger on a clean surface in a safe place to dry (See Figure W).Wash your hands with soap and water.After the oral syringe barrel and plunger are dry, put the plunger back into the oral syringe barrel and store the syringe with your medicine.

EVRYSDI is a registered trademark of Genentech, Inc.

Distributed by:
Genentech, Inc.
1 DNA Way
South San Francisco, CA 94080-4990
Approved: 4/2021
This Instructions for Use has been Approved by the U.S. Food and Drug Administration.

©2021 Genentech, Inc. All Rights Reserved

EVRYSDI®
(risdiplam) for oral solution

Instructions for Constitution (FOR HEALTHCARE PROVIDERS ONLY)

Each EVRYSDI carton contains (see Figure A):

Image1 Cap
Figure A
(click image for full-size original)
Image1 EVRYSDI bottle
Image2 Reusable oral syringes 12 mL
Image2 Reusable oral syringes 6 mL
Image1 Press-in bottle adapter
Image1 Prescribing Information (not shown)
Image1 Instructions for Constitution (not shown)
Image1 Instructions for Use (not shown)

Note: 1 mL reusable oral syringes will be provided separately.

Important information about EVRYSDI

  • Do not use if the powder expiration date has passed. The powder expiration date is printed on the bottle label.
  • Do not use the medicine if any of the supplies are damaged or missing.
  • Use purified water to constitute the medicine.
  • Select the appropriate oral syringes (1 mL, 6 mL, or 12 mL) based on the patient’s dose and provide instruction to the patient/caregiver on how to administer their dose. The oral syringes provided are intended to be reusable.
    • The 6 mL and 12 mL oral syringes are provided in the carton.
    • The 1 mL oral syringes will be provided separately.
  • Only use the amber oral syringe provided for use with EVRYSDI. Other syringes may not be compatible with the press-in-bottle adapter and may not protect the product from light.

How to store EVRYSDI

  • Store the dry powder at 20°C to 25°C (68°F to 77°F), excursions permitted between 15°C to 30°C (59°F to 86°F) [see USP controlled room temperature]. Keep in the original carton.
  • Store the constituted oral solution of EVRYSDI upright in the original amber bottle in a refrigerator at 2°C to 8°C (36°F to 46°F). Do not freeze.

Important precautions for preparation of EVRYSDI

  • Avoid inhalation and direct contact with skin or mucous membranes with the dry powder and the constituted solution. If such contact occurs, wash thoroughly with soap and water; rinse eyes with water.
  • Wear disposable gloves during the preparation and clean up procedure.

Constitution

Figure BFigure BStep 1 Gently tap the bottom of the bottle to loosen the powder (See Figure B).
Figure CFigure CStep 2 Remove the cap by pushing it down and then twisting to the left (counter-clockwise) (See Figure C). Do not throw away the cap.
Figure DFigure DStep 3 Carefully pour 79 mL of purified water into the medicine bottle (See Figure D).
Figure E
(click image for full-size original)
Step 4 Hold the medicine bottle on a table with one hand.Insert the press-in bottle adapter into the opening by pushing it down with the other hand. Ensure it is completely pressed against the bottle lip (See Figure E).
Figure F
(click image for full-size original)
Step 5 Put the cap back on the bottle. Turn the cap to the right (clockwise) to close the bottle.Ensure it is completely closed and then shake well for 15 seconds (See Figure F).Wait for 10 minutes. You should have obtained a clear solution.If not, shake well again for another 15 seconds.
Figure GFigure GStep 6 Calculate the Discard After date of the oral solution as 64 days after constitution (Note: The day of constitution is counted as day 0. For example, if constitution is on the 1st of April, the Discard After date will be the 4th of June).Write the Discard After date of the solution and the Lot number on the bottle label (See Figure G).Do not dispense the constituted solution if the solution’s Discard After date exceeds the original powder expiration date.
Figure HFigure HStep 7 Remove and discard the peel-off part of the bottle label with the expiration date of the powder (See Figure H).

Selecting the oral syringe for the prescribed daily dose of EVRYSDI

For the calculation of dosing volume, the syringe increments need to be considered. Round the dose volume to the closest increment marked on the selected oral syringe.

Select the correct oral syringe(s) (1 mL, 6 mL, or 12 mL) for the calculated dosing volume according to the table below and remove the other oral syringes.

Dose StrengthSyringe SizeDosing VolumeSyringe Increments
0.75 mg/mL1 mL0.3 mL to 1 mL0.01 mL
6 mL1 mL to 6 mL0.1 mL
12 mL6.2 mL to 6.6 mL0.2 mL

Put the bottle back in its original carton with the correct oral syringes, Prescribing Information, and Instructions for Use.

Store the constituted oral solution of EVRYSDI upright in the original amber bottle in a refrigerator at 2°C to 8°C (36°F to 46°F). Do not freeze. Discard any unused portion 64 days after constitution.

EVRYSDI is a registered trademark of Genentech, Inc.

Distributed by:
Genentech, Inc.
1 DNA Way
South San Francisco, CA 94080-4990
Revision Date: 5/2022
©2022 Genentech, Inc. All Rights Reserved.

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