How One Man With PNH Found a Treatment That Fits His “On-the-Go” Lifestyle

Jason is a patient with PNH who has been compensated for his time. Continue reading for Important Safety Information, including Boxed WARNING and Medication Guide.  

Jason has always loved any kind of vehicle with wheels–especially the ones that are built for speed. Whether getting dirt cars ready for the dirt track, maneuvering remote-controlled (RC) cars around a racetrack, building drift trikes from scratch, or riding down a mountain trail on his bike, he describes himself as “always on the go.”

In fact, Jason was mountain biking with his buddies when he started feeling shortness of breath, one of his first symptoms, which would eventually be diagnosed as paroxysmal nocturnal hemoglobinuria (PNH).¹ For two months, he experienced shortness of breath each time he went biking, and this progressed into what he and his doctor initially thought was bronchitis.

Like many people with rare diseases, the road to a diagnosis was challenging and involved a progression of doctors^2,3–in Jason’s case, from family physician to cardiologist and to, eventually, a hematologist-oncologist, who quickly identified that his symptoms may be due to PNH. By the time he was diagnosed with PNH, Jason’s hemoglobin was a low 7 g/dL,⁴ and he was not feeling like himself–not a familiar or welcome feeling for someone who loves to stay moving.

A startling diagnosis

When Jason received the call with his PNH diagnosis, he says it completely stopped him in his tracks.

“I had left for a vacation cruise when I got the call that I had PNH, and immediately went to the internet to learn more,” says Jason. “What I read was scary. But then I said to myself, ‘Let me just wait to find out more from my doctor.’”

When he met with his doctor, he learned that PNH was a condition that could be managed, and he started therapy.¹

“Until recently, most treatments for PNH involved infusion-based C5 inhibitors,” says Jamie L. Koprivnikar, MD*, a hematologist-oncologist at Hackensack University Medical Center in New Jersey. “These agents were ground-breaking developments for the management of PNH when they came along. However, based on my own experience treating patients with PNH, some patients are looking for treatment options other than infusions. So there still remained some unmet need.”^1,5,6

Jason had a similar experience while on his initial treatment. Though he was extremely grateful that treatments existed, he says he sometimes felt like he could be doing better. “I learned to take it slow,” he says. “But my hemoglobin still wasn’t where I wanted it to be. So I continued to research what other options were available and kept in close touch with my doctor about how I was feeling and whether a switch might be the right choice for me.”

An oral treatment option for PNH

Jason worked with his doctor to develop a treatment plan that suited his lifestyle, which included switching to FABHALTA^® (iptacopan), the first US Food and Drug Administration (FDA)-approved oral treatment for adults with PNH.⁷ FABHALTA is a Factor B inhibitor that may lower a person’s ability to fight infections and increases one’s chance of getting serious infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type b. It is only available through a program called the FABHALTA Risk Evaluation and Mitigation Strategy (REMS).⁷

“FABHALTA is novel because of where it works in the complement system,”⁷ says Dr Koprivnikar. PNH occurs when the complement part of the immune system destroys red blood cells–a process known as hemolysis. FABHALTA controls the two types of hemolysis that occur in PNH: intravascular hemolysis, which happens in the blood vessels; and extravascular hemolysis, which happens most commonly in the liver and spleen.^6,7

Dr Koprivnikar says that having an oral treatment option has been an important advancement in the field. “When FABHALTA was approved by the FDA for adults with PNH, I was excited,” she says. “For patients who are struggling to manage their PNH, I prioritize having a conversation about their needs and goals for treatment. A twice-daily capsule option may be beneficial for many of my patients, as it can fit into their lifestyle and care plan.”⁷

Now on this treatment, Jason says he’s happy with the way he’s feeling–and likes being on a treatment that works well with his routine.

Staying optimistic and connected

 

Today, Jason is feeling confident about his PNH care plan. He continues to race his RC cars and work on passion projects like the drift trike he’s building in his workshop. But he has his ups and downs, where he sometimes has to take things slow. Having an “invisible” disease can be challenging since symptoms aren’t always obvious to others^2,3–but he makes sure to explain what he’s going through and rest when his body tells him to. He also recommends seeking out people who are going through the same thing–connecting with others through online groups or during awareness and fundraising walks.

“Reach out to people that you feel comfortable with so you can share stories and learn from each other,” says Jason. “That’s the best thing. When you talk to real people who have PNH and live with it every day, you learn from them, and it makes things so much easier.”

Jason wants people with PNH to have the resources and support they need to have effective conversations with their doctor and find a treatment plan that works for them.

To learn more about FABHALTA, visit FABHALTA.COM.

*Dr Koprivnikar has been compensated for her time on this article and is a paid consultant for other projects of Novartis Pharmaceuticals Corporation, the manufacturer of FABHALTA.

 

Indication

FABHALTA is a prescription medicine used to treat adults with paroxysmal nocturnal hemoglobinuria (PNH).

It is not known if FABHALTA is safe and effective in children.

Important Safety Information

FABHALTA is a medicine that affects part of the immune system and may lower one’s ability to fight infections. FABHALTA increases the chance of getting serious infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type b. These serious infections may quickly become life-threatening or fatal if not recognized and treated early. Patients must complete or be up to date with the vaccines against Streptococcus pneumoniae and Neisseria meningitidis at least 2 weeks before starting FABHALTA. If patients have not completed these vaccinations and FABHALTA therapy must be started right away, they should receive the required vaccinations as soon as possible. If patients have not been vaccinated and FABHALTA must be started right away, they should also receive antibiotics to take for as long as their health care provider tells them. If patients have been vaccinated against these bacteria in the past, they might need additional vaccinations before starting FABHALTA. Their doctor will decide if they need additional vaccinations. Vaccines do not prevent all infections caused by encapsulated bacteria. Patients should call their doctor or get emergency medical care right away if they have any of these signs and symptoms of a serious infection: fever with or without shivers or chills; fever with chest pain and cough; fever with high heart rate; headache and fever; confusion; clammy skin; fever and a rash; fever with breathlessness or fast breathing; headache with nausea or vomiting; headache with stiff neck or stiff back; body aches with flu-like symptoms; eyes sensitive to light. Health care providers will give their patients a Patient Safety Card about the risk of serious infections. Patients must carry it with them at all times during treatment and for 2 weeks after their last dose of FABHALTA. The risk of serious infections may continue for a few weeks after their last dose of FABHALTA. It is important for patients to show this card to any health care provider who treats them. This will help them diagnose and treat patients quickly.

FABHALTA is only available through a program called the FABHALTA Risk Evaluation and Mitigation Strategy (REMS). Before patients can take FABHALTA, their health care provider must enroll in the FABHALTA REMS program, counsel patients about the risk of serious infections caused by certain bacteria, give patients information about the symptoms of serious infections, make sure that patients are vaccinated against serious infections caused by encapsulated bacteria and that they receive antibiotics if they need to start FABHALTA right away and are not up to date on vaccinations, and give patients a Patient Safety Card about the risk of serious infections.

Patients should not take FABHALTA if they are allergic to FABHALTA or any of the ingredients in FABHALTA or if they have a serious infection caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, or Haemophilus influenzae type b when starting FABHALTA.

Before taking FABHALTA, patients should tell their health care provider about all their medical conditions, including if they have an infection or a fever, have liver problems, are pregnant or plan to become pregnant (it is not known if FABHALTA will harm an unborn baby), or are breastfeeding or plan to breastfeed, as it is not known if FABHALTA passes into breast milk. Patients should not breastfeed during treatment and for 5 days after their final dose of FABHALTA.

Patients should tell their health care provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking FABHALTA with certain other medicines may affect the way FABHALTA works and may cause side effects. Patients should know the medicines they take and the vaccines they receive. Patients should keep a list of them to show their health care provider and pharmacist when they get a new medicine.

If patients have PNH and stop taking FABHALTA, their health care provider will need to monitor them closely for at least 2 weeks after stopping FABHALTA. Stopping treatment with FABHALTA may cause a breakdown of red blood cells due to PNH. Symptoms or problems that can happen due to breakdown of red blood cells include: decreased hemoglobin level in the blood; blood in the urine; shortness of breath; trouble swallowing; tiredness; pain in the stomach (abdomen); blood clots, stroke, and heart attack; and erectile dysfunction (ED). It is important that patients take FABHALTA exactly as their health care provider tells them to lower the possibility of breakdown of red blood cells due to PNH.

FABHALTA may cause serious side effects, including increased cholesterol and triglyceride (lipid) levels in the blood. Health care providers will do blood tests to check patients’ cholesterol and triglycerides during treatment with FABHALTA and may start patients on cholesterol-lowering medicine if needed.

The most common side effects of FABHALTA include headache; nasal congestion, runny nose, cough, sneezing, and sore throat (nasopharyngitis); diarrhea; pain in the stomach (abdomen); infections (bacterial and viral); nausea; and rash.

Patients should tell their health care provider about any side effect that bothers them or that does not go away. These are not all the possible side effects of FABHALTA. Patients should call their health care provider for medical advice about side effects.

Patients are encouraged to report negative side effects of prescription drugs to the FDA by visiting www.fda.gov/medwatch or calling 1-800-FDA-1088.

Please see full Prescribing Information, including Boxed WARNING and Medication Guide.

References:

1. Dingli D, Matos JE, Lehrhaupt K, et al. The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey. Ann Hematol. 2022;101(2):251-263. doi:10.1007/s00277-021-04715-5
2. Bektas M, Copley-Merriman C, Khan S, Sarda SP, Shammo JM. Paroxysmal nocturnal hemoglobinuria: patient journey and burden of disease. J Manag Care Spec Pharm. 2020 Dec;26(12-b Suppl):S8-S14. doi: 10.18553/jmcp.2020.26.12-b.s8
3. Marwaha S, Knowles JW, Ashley EA. A guide for the diagnosis of rare and undiagnosed disease: beyond the exome. Genome Med. 2022;14(1):23. Published 2022 Feb 28. doi:10.1186/s13073-022-01026-w
4. Cleveland Clinic. Low Hemoglobin. Accessed September 19, 2024. https://my.clevelandclinic.org/health/symptoms/17705-low-hemoglobin
5. Debureaux PE, Kulasekararaj AG, Cacace F, et al. Categorizing hematological response to eculizumab in paroxysmal nocturnal hemoglobinuria: a multicenter real-life study. Bone Marrow Transplant. 2021;56(10):2600-2602. doi:10.1038/s41409-021-01372-0
6. Cançado RD, Araújo A da S, Sandes AF, et al. Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria. Hematol Transfus Cell Ther. 2021;43(3):341-348. doi:10.1016/j.htct.2020.06.006
7. FABHALTA (iptacopan) Prescribing Information. Novartis Pharmaceuticals Corp.