APRIL: I’ve waited basically my entire life for something like this. I mean, I was speechless. We all cried. So if you’re eligible and considering it, at least have that conversation with your doctor.

Hi, I’m April and I have cystic fibrosis. I have one copy of the F508 delta mutation. And I’m here to tell you my story. My CF is a big part of me. It always comes first, but I don’t let it really define me as a person.

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.

APRIL: I love being outdoors and just staying as active as I can, so archery started as a sport to get me active and going. Also, I know it’s important for people with cystic fibrosis to be active. I fell in love with it instantly. Also, working out when you go to the gym—I feel like it helps you focus more. It’s not always about my CF, it’s about me and how I want to be as an individual.

When I learned I was eligible

I’ve waited basically my entire life for something like this. Although my F508 delta mutation is common, my other mutation is pretty rare. So when I found out that I was eligible, I was speechless. We all cried. I cried. My family cried. I always get emotional about it.

I had concerns about the possible side effects, so I asked my doctors what they were and what things would need to be monitored.

For the side effects, as soon as something happens, you call your doctor immediately. Just be very transparent to each other and explain exactly how you’re feeling. It was really important to have that discussion.

Before telling you more of my story, take a moment to listen to this Important Safety Information.

VOICE-OVER: 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.

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.

Please see additional Important Safety Information later in this video and at TRIKAFTA.com.

My experience on TRIKAFTA

Keep in mind that your results may be different from what is mentioned in this story.

APRIL: The biggest change I saw was my lung function. It’s the highest it has been in a while. I’ve also noticed improvements in my respiratory symptoms. And I did start gaining weight on the drug, which is huge for people with cystic fibrosis. So when I went to the doctors and discussed with them, they were so excited. They were like, That is great, that is what you need. I mean, that is something I really fought for—to gain weight—my whole life.

Body mass index (BMI)* was evaluated in a study of people with one F508del mutation and another mutation defined in the study who took either TRIKAFTA or placebo.

*BMI=a measure of someone's weight in relation to their height.

I pay more attention to my diet, adding in some healthier fats, balancing out any junk food. I did experience a side effect. But before I tell you about that, take a look at some more Important Safety Information.

VOICE-OVER: 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; 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; 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.

My experience with side effects on TRIKAFTA

APRIL: My care team said they’d monitor me while I’m on treatment and that I should call them if I experience a side effect at any point. I did get a rash. My doctors and I decided to monitor it, and eventually it went away. It helped me that we discussed what to expect before starting. I’m grateful that I have doctors who are dedicated and know me very well.

Here’s what’s next for me!

I’m just so excited that I am going to be a science teacher so I can teach them about genetics and cystic fibrosis, so it’ll be pretty fun. I do want to inspire people. I want people to know that you’re not, you’re not the sick person. You’re more than that.

What I’d say if you’re considering TRIKAFTA

I’m always looking to do whatever I can for my CF. And after talking with my doctor, I knew that this was the right move for me. So if you’re eligible and considering it, at least have that conversation with your doctor.

VOICE-OVER: 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.