JEN: Clayton is a, kind of a stereotypical 5-year-old. He loves to be active. Probably his favorite activities are playing outdoors, riding his bike. Um, he also loves imaginary play.

JEN, DALE, AND CLAYTON: Action!

DALE: Yeah! Nicely done, kiddo!

JEN: Hi, I'm Jen.

DALE: And I'm Dale. And we have a son named Clayton who is 5 years old.

JEN: We're going to answer some questions that other parents may have about TRIKAFTA. We'll also talk about how we decided TRIKAFTA was right for our son and how it's going.

Clayton's Story

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, or 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.

JEN: Finding out that Clayton has CF was a shock. But I feel like now, as time has gone by, that shock has gone away. And I've actually become very appreciative of how close-knit and active the CF community is. There has been so much research and so many advancements in how it's treated. It's been pretty neat over the last 5 years of Clayton's life to see a lot of those things come about.

Why did you start treatment with TRIKAFTA when you did?

JEN: At the time, we had been keeping tabs on the pipeline of CF treatments and what was in clinical trials. And so, we knew from his diagnosis and the gene mutations that he has, he wasn't eligible for any of the modulators that were on the market at the time. So, when Clayton's doctor said that TRIKAFTA was available and Clayton was eligible, that got us really excited. So, we were like, “All right, let's try this and see if it helps. And if this is something that works for him, it could be a really big deal.”

DALE: We had also heard about TRIKAFTA from others in the CF community here in Pittsburgh who were taking it. Hearing other people's experiences really gave us a sense of hope and anticipation. Before we get to more of your questions, please listen to this Important Safety Information.

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 or rifabutin
• seizure medicines such as phenobarbital, carbamazepine, or phenytoin
• St. John’s wort
• antifungal medicines including ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole
• antibiotics including telithromycin, clarithromycin, or erythromycin

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

Please stay tuned for additional Important Safety Information at the end of this video.

What has Clayton taking TRIKAFTA meant to you?

JEN: From Clayton's standpoint, it's just another part of his routine. It's another treatment and he takes it. But for Dale and me, it's treating the underlying cause. And that gives us a lot of hope when we think about the future.

Did you have any hesitations? How did you get past them?

JEN: I think what helped us was we had been told about potential side effects by Clayton's care team, so we felt like we knew what to watch out for. Also, having a good relationship with them and having all the different phone numbers and contact info, we knew if there was an issue, we could reach out and they would help us understand what needs to happen. And then just hearing the experiences of others with CF who were taking TRIKAFTA, as well as Clayton's doctor's experience prescribing it to others.

What was it like getting started? How does Clayton stay on track with treatment?

DALE: I remember the first time I helped with giving Clayton his TRIKAFTA. I had this thought of like, “So, this is it. This is what everyone's been talking about. And now it's Clayton's turn.” It was a very surreal moment.

At this point we've got taking TRIKAFTA down pat. We'll plan out what fat-containing food he'll eat right after, and we've started getting him involved with that. The whole routine has become something that we all really enjoy doing together. Homemade pizza is one of our favorites.

Clayton takes TRIKAFTA every 12 hours. When it's time for treatment, I start by measuring out a teaspoon, or 5 milliliters, of a soft food or liquid, usually yogurt or applesauce, into a small bowl. I make sure it's at room temperature. Next, I shake the packet, open it, dump it into the yogurt, and mix it all together. Then I make sure he finishes all of it within an hour.

In terms of staying on track when he first started, at that point he was already used to his other oral treatments. So, for him, it was just a different type of treatment. He's also very big on routine. He actually won't start eating until he takes his TRIKAFTA. There's been just a handful of times when I've maybe forgotten to get it ready, and he'll speak up and say, “Where's my TRIKAFTA?”

Do you have any tips for other caregivers?

JEN: So I think early on, um, CF definitely seems kind of all-encompassing. That you're figuring out the treatment and it's a big focus of life. And I think, over the years we've been able to adapt, and it's become part of our routine and part of our family but doesn't define what we do, um, and that Clayton and us, we are able to participate in the different activities that we enjoy doing, and it's just kind of part of our normal but not this heavy cloud weighing over us.

What’s next for Clayton?

DALE: From his perspective, it's his next adventure. And for us, it's great to see him come into his own and enjoy being a kid.

JEN: And we've done everything that the doctors have said and have had a few bumps along the way but have kept at it. And thankfully, he's a happy, thriving 5-year-old.

Thanks for listening to our story! I hope it answered some of your concerns and gave you ideas for questions to ask your doctor.

DALE: Keep watching for some more Important Safety Information.

Additional 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.

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.