STACEY: Celebrating my son’s 6th birthday was a happy time for the whole family and knowing that TRIKAFTA^® was approved for children his age with the same mutations made me feel even better. Hi, everybody. I’m Stacey.

MASSI: Hi, I’m Massimo, but everybody calls me Massi.

STACEY: I’m going to share why we decided TRIKAFTA was right for our son, Massimo, and what it’s meant for us. Massi is a smiley, happy kid who other parents and kids just love to be around. He’s energetic and loves playing soccer, basketball, and baseball. He always has a positive attitude and he’s living his life with CF to the fullest.

VOICE-OVER: TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (or CF) in patients aged 2 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA.

Talk to your doctor to learn if you have an indicated CF gene mutation.

It is not known if TRIKAFTA is safe and effective in children under 2 years of age.

Did you have any hesitations about starting Massimo on TRIKAFTA?

STACEY: The short answer is yes. I don’t tend to give my kids medicine without doing the research. I just want to make sure I’m prepared and understand what to watch for, including potential benefits and possible side effects. I guess it all goes back to when Massi was first diagnosed.

It was a very emotional time and we had a lot of hard days. I can recall our doctor telling me that I needed to stay positive because of the research underway for potential medicines that might help Massi. Staying informed and talking to our care team gave me a lot of hope.

Do you have any advice for other parents?

STACEY: I’d say find a routine that works for you and your child and stick to it. We established a routine early on and that made it easier for Massi to stay on track with his medicine. Also, if your child can have the opportunity to live a life that’s defined by more than their CF, it’s worth doing everything you can.

Start the conversation with your care team about potential treatments. I know that helped me feel more confident that TRIKAFTA was the right decision for our family.

What helped us make the decision to start TRIKAFTA?

When we were talking to our care team about TRIKAFTA, for us, what was really important was, first of all, was he in fact going to qualify for it with his particular mutations?

Once we got past that, it got more serious. What were the side effects of TRIKAFTA? We had all heard, anecdotally, some of the great outcomes, but what was our care team seeing firsthand? You know, they’re the ones on the ground seeing all the patients that are taking TRIKAFTA. What were they seeing?

For us, what was really important were considering the risk/benefit of taking a drug like TRIKAFTA. Were the risks or the side effects going to outweigh the benefit? In their opinion, when we were talking it through and making an educated decision about it, the benefits for Massi outweighed the risks. We started as soon as we could.

Before I get to more of your questions, please listen to this Important Safety Information.

Before taking TRIKAFTA, tell your doctor about all of your medical conditions, including if you are allergic to TRIKAFTA or any ingredients in TRIKAFTA. See the Patient Information for a list of ingredients. Have kidney problems; have or have had liver problems; are pregnant or plan to become pregnant. It is not known if TRIKAFTA will harm your unborn baby. You and your doctor should decide if you will take TRIKAFTA while you are pregnant. Are breastfeeding or planning to breastfeed. It is not known if TRIKAFTA passes into your breast milk. You and your doctor should decide if you will take TRIKAFTA while you are breastfeeding.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

How has treatment been going so far?

STACEY: Any improvement for Massi was going to be a win for us. So from early on, we made sure to create a routine that helped him stay on track so he’d reach his treatment goals. I’m happy to say that he’s definitely seen an improvement. He’s in first grade. He’s doing really well. He loves to read. And it’s fun just to celebrate those normal victories that you come to know with your kids in school. It makes me glad we started TRIKAFTA when we did.

Thanks for listening to our story. I hope it answered some of your questions and gave you ideas for what to discuss with your doctor. Please keep watching for some more Important Safety Information.

VOICE-OVER: TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works. The dose of TRIKAFTA may need to be adjusted when taken with certain medicines. Ask your doctor or pharmacist for a list of these medicines if you are not sure.

Especially tell your doctor if you take: antibiotics such as rifampin (RIFAMATE^®, RIFATER^®) or rifabutin (MYCOBUTIN^®). Seizure medicines such as phenobarbital, carbamazepine (TEGRETOL^®, CARBATROL^®, EQUETRO^®), or phenytoin (DILANTIN^®, PHENYTEK^®). St. John’s wort. Antifungal medicines including ketoconazole, itraconazole (such as SPORANOX^®), posaconazole (such as NOXAFIL^®), voriconazole (such as VFEND^®), or fluconazole (such as DIFLUCAN^®). Antibiotics including telithromycin, clarithromycin (such as BIAXIN^®), or erythromycin (such as ERY-TAB^®)

Avoid food or drink that contains grapefruit while you are taking TRIKAFTA.

TRIKAFTA can cause serious side effects, including liver damage and worsening of liver function in people with severe liver disease that can be serious and may require transplantation. Liver damage has also happened in people without liver disease.

High liver enzymes in the blood, which is a common side effect in people treated with TRIKAFTA. These can be serious and may be a sign of liver injury. Your doctor will do blood tests to check your liver before you start TRIKAFTA, every 3 months during your first year of taking TRIKAFTA, and every year while you are taking TRIKAFTA. Your doctor may do blood tests to check the liver more often if you have had high liver enzymes in your blood in the past.

Call your doctor right away if you have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach, or abdominal, area; yellowing of your skin or the white part of your eyes; loss of appetite; nausea or vomiting; dark, amber-colored urine.

Serious allergic reactions have happened to people who are treated with TRIKAFTA. Call your healthcare provider or go to the emergency room right away if you have any symptoms of an allergic reaction. Symptoms of an allergic reaction may include: rash or hives; tightness of the chest or throat or difficulty breathing; swelling of the face, lips, and/or tongue, or difficulty swallowing; or light-headedness or dizziness.

Abnormality of the eye lens, also known as cataract, which has happened in some children and adolescents treated with TRIKAFTA. If you are a child or adolescent, your doctor should perform eye examinations before and during treatment with TRIKAFTA to look for cataracts.

The most common side effects of TRIKAFTA include headache; upper respiratory tract infection, or common cold, including stuffy and runny nose; stomach, or abdominal, pain; diarrhea; rash; increase in liver enzymes; increase in a certain blood enzyme called creatine phosphokinase; flu, or influenza; inflamed sinuses; or increase in blood bilirubin.

Tell your doctor if you have any side effect that bothers you or that does not go away.

These are not all the possible side effects of TRIKAFTA. For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

To learn more about TRIKAFTA and if it could be right for you, please talk to your healthcare provider and visit TRIKAFTA.com. Please see the links for Important Safety Information and full Prescribing Information, including Patient Information below.

This video is brought to you by Vertex Pharmaceuticals Incorporated.