Important Safety Information

• 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.

--Brand Name--
US Brand Name: Evrysdi

--Description--
Risdiplam is used to treat spinal muscular atrophy, a rare nerve and muscle disorder that is characterized by loss of lower motor neurons and progressive muscle wasting, often leading to early death.

This medicine is available only with your doctor's prescription.

This product is available in the following dosage forms:

• Tablet
• Powder for Solution

--Before Using--
In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies
Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Pediatric
Appropriate studies performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of risdiplam in children (newborn infants and older). Safety and efficacy have been established.

Geriatric
Appropriate studies on the relationship of age to the effects of risdiplam have not been performed in the geriatric population.

Breastfeeding
There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Drug Interactions
Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Other Interactions
Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other Medical Problems
The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

Liver disease—Use with caution. The effects may be increased because of slower removal of the medicine from the body.

--Proper Use--
Take this medicine only as directed by your doctor. Do not take more of it, do not take it more often, and do not take it for a longer time than your doctor ordered.

This medicine should come with a patient information leaflet and patient instructions. Read and follow these instructions carefully. Ask your doctor if you have any questions.

You may take the medicine with or without food at the same time each day.

Swallow the tablet whole with water. Do not chew, cut, or crush it.

Measure the oral liquid with the oral syringe and take it within 5 minutes. Throw away any unused medicine after 5 minutes. Do not save leftover medicine.

If you are giving theoral liquid to an infant, give it before or after breastfeeding. Do not mix it with milk or formula.

To use the oral liquid:

• Remove the cap by pushing it down and twisting the cap to the left.
• Push the plunger of the oral syringe all the way down to remove any air. Place the bottle on a flat surface and insert the syringe tip into the bottle adapter.
• Carefully turn the bottle upside down with the syringe tip firmly inserted into the adapter. Slowly pull back on the plunger until the top of the plunger lines up with the marking on the barrel of the syringe that matches the needed dose. Hold the plunger in place to keep it from moving.
• Turn the bottle into an upright position. Remove the oral syringe from the bottle adapter by pulling straight up. Check for large air bubbles in the syringe. If you see air bubbles, insert the syringe tip into the bottle adapter and push the plunger all the way down to let the medicine flow back into the bottle. Repeat the steps to withdraw your prescribed medicine dose.
• Put the cap back on. Do not remove the bottle adapter.
• Place the tip of the syringe into the mouth against the inside of the cheek. Slowly push the plunger all the way down to give full dose. Drink about 15 milliliters (mL) of water after taking the medicine.
• If you are giving this medicine through a gastrotomy or nasogastric tube: Place the oral syringe tip into the tube. Slowly push the plunger all the way down to give full dose. Flush the tube with 10 to 20 mL of water.

If you are unable to swallow he tablet whole:

• Wash your hands before and after preparing or taking this medicine.
• Put the tablet in a small cup with a teaspoon (5 mL) of room temperature non-chlorinated drinking water (eg, filtered water). Do not mix it with any liquids other than non-chlorinated drinking water.
• Gently swirl the cup until the tablet and water are fully mixed. It may take up to 3 minutes.
• Swallow the mixture right away. Add at least 15 mL of non-chlorinated drinking water into the cup to make sure there is no medicine left. Drink the mixture right away. Do not give the mixture via nasogastric or gastrotomy tube.
• Throw away mixture 10 minutes after mixing it.

Do not let the tablet or oral liquid touch your skin or eyes. If it gets on your skin, wash the area with soap and water. If it got in your eyes, rinse your eyes with water.

If the tablet mixture or oral liquid is spilled, use a dry paper towel to dry the area and clean it with soap and water.

Dosing

The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

For spinal muscular atrophy:
►For oral dosage form (solution):

• Adults and children 2 years of age and older weighing 20 kilograms (kg) or more—5 milligrams (mg) once a day.
• Children 2 years of age and older weighing less than 20 kg—Dose is based on body weight and must be determined by your doctor. The dose is usually 0.25 milligram per kilogram (mg/kg) of body weight once a day.
• Infants 2 months to less than 2 years of age—Dose is based on body weight and must be determined by your doctor. The dose is usually 0.2 mg/kg of body weight once a day.
• Infants younger than 2 months of age—Dose is based on body weight and must be determined by your doctor. The dose is usually 0.15 mg/kg of body weight once a day.

►For oral dosage form (tablets):

• Adults and children 2 years of age and older weighing 20 kilograms (kg) or more—5 milligrams (mg) once a day.
• Children younger than 2 years of age and older weighing less than 20 kg—Use of oral liquid is recommended.

Missed Dose

If you miss a dose of this medicine, take it as soon as possible. However, if it is almost time for your next dose, skip the missed dose and go back to your regular dosing schedule. Do not double doses.

If you miss a dose and it is within 6 hours of your usual dose schedule, take it as soon as possible, and then go back to your regular schedule. If you miss a dose and it is more than 6 hours of your usual dose schedule, skip the missed dose and take your next dose at the regular time.

Storage

Keep out of the reach of children.

Do not keep outdated medicine or medicine no longer needed.

Ask your healthcare professional how you should dispose of any medicine you do not use.

Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light. Keep from freezing.

Store the oral liquid in the refrigerator. Do not freeze. If needed, you may store it at room temperature for a combined total of 5 days. You may put it back in the refrigerator. Throw away the oral liquid if it has been kept at room temperature for more than 5 days.

Throw away any unused oral liquid 64 days after mixing it.

--Precautions--
It is very important that your doctor check your child's progress at regular visits to make sure this medicine is working properly. Blood tests may be needed to check for unwanted effects.

Using this medicine while you are pregnant can harm your unborn baby. If you are a woman who can bear children, your doctor may give you a pregnancy test before you start using this medicine to make sure you are not pregnant. Use an effective form of birth control during treatment and for at least 1 month after the last dose.

If you plan to have children, talk with your doctor before using this medicine. Some men using this medicine have become infertile (unable to have children).

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or non-prescription (over-the-counter [OTC]) medicines and herbal or vitamin supplements.

--Side Effects--
Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur:

More common

• Body aches or pain
• chest pain or tightness
• chills
• cough
• ear congestion
• fever
• headache
• loss of voice
• runny or stuffy nose
• sneezing
• sore throat
• trouble breathing
• unusual tiredness or weakness

Less common

• Bladder pain
• bloody or cloudy urine
• difficult, burning, or painful urination
• frequent urge to urinate
• lower back or side pain

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

• Canker sores
• constipation
• diarrhea
• skin rash
• sores, ulcers, or white spots on the lips or tongue or inside the mouth
• vomiting

Less common

• Difficulty in moving
• joint pain
• muscle pain or stiffness

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.