High satisfaction reported with Fabhalta in PNH patient interviews
All participants previously on Soliris or Ultomiris preferred newer oral therapy
Most people with paroxysmal nocturnal hemoglobinuria (PNH) treated with Fabhalta (iptacopan) in clinical trials reported high satisfaction and a meaningful easing of symptoms with the oral therapy, and generally preferred it over other older treatments.
In fact, all patients previously treated with the PNH therapies Soliris (eculizumab) or Ultomiris (ravulizumab-cwvz) said Fabhalta was preferred.
That’s according to a new study based on interviews involving dozens of participants treated with the now-approved therapy in three Phase 3 trials.
“Participants on the whole reported a positive treatment experience with [Fabhalta], specifically noting the convenience of oral administration, treatment efficacy, fewer doctor visits, and the lack of need for transfusions,” the researchers wrote. “All participants who were asked reported that they were either satisfied or very satisfied with [Fabhalta] treatment, and all participants who were asked about their experience with other PNH treatments reported that they preferred [Fabhalta] over their previous treatments because of its oral administration and treatment schedule.”
The study, “Patient-Reported Meaningful Change in Symptoms and Impacts of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Three Phase III Clinical Trials of Iptacopan,” was published in The Patient – Patient-Centered Outcomes Research, a journal of the International Academy of Health Preference Research. The work was funded by Novartis, the company that markets Fabhalta. Four of the 15 authors work for Novartis, while another six are employees of another company compensated by the pharmaceutical for their work on the study.
An acquired disorder, PNH is marked by blood cell destruction driven by the abnormal activation of part of the immune system called the complement cascade. Fabhalta works by blocking the activity of a complement protein called factor B. The twice-daily oral medication is approved in the U.S. to treat adults with PNH, regardless of whether or not they have received prior treatments.
Researchers interview participants in Fabhalta clinical trials
Fabhalta’s approval was based on data from two Phase 3 clinical trials: APPLY-PNH (NCT04558918) and APPOINT-PNH (NCT04820530). APPLY-PNH enrolled adults with PNH who were experiencing substantial disease activity on prior treatments, while APPOINT-PNH had recruited adults with PNH who had not been previously treated. Participants who completed either of these trials had the option to continue into an ongoing open-label extension study (NCT04747613), in which all are being treated with Fabhalta and monitored for long-term outcomes. That study is expected to wrap up in late 2027.
Data from these trials have indicated that most patients given Fabhalta had no need for blood transfusions. The results also indicated that, irrespective of prior treatment, Fabhalta could boost the levels of hemoglobin, the protein that red blood cells use to transport oxygen through the body.
Here, the researchers sought to better understand patients’ individual experiences with the therapy. To that end, the team interviewed 61 people who participated in at least one of these studies.
“Collecting data on the associated change in disease experience helps further understand the effectiveness of this novel treatment and ensures the patient voice is captured and included in the drug development program,” the researchers wrote.
Data from the interviews indicated that the vast majority of patients saw their PNH symptoms ease with Fabhalta. For example, all but one of 43 patients who discussed fatigue said this symptom was less severe with Fabhalta.
Similar rates of improvement were seen for other signs and symptoms of PNH, including tiredness, weakness, shortness of breath, and pale or yellow skin. Consistent with the easing of symptoms, most patients reported that Fabhalta treatment made it easier for them to carry out day-to-day activities and engage in social functions.
“Significantly, these participants reported that these improvements were meaningful to them, with meaningful improvements reported by both participants who had received [other treatments for PNH] and those who had not,” the researchers wrote.
The team noted that these improvements are consistent with trial data showing Fabhalta’s ability to boost hemoglobin levels and limit the need for transfusions.
Patients highlighted convenience, ease of using oral therapy
Among the 22 patients who were asked to rate their overall satisfaction with Fabhalta, nearly two-thirds said they were very satisfied, and the rest reported being satisfied.
There were 18 patients who had previously been on other PNH therapies, namely Soliris or Ultomiris, and who were asked to compare their satisfaction with Fabhalta against those prior treatments. All of these patients said they preferred Fabhalta over the previous therapy they had received.
It’s much more comfortable to take pills rather than having an injection in your arm. … [I am] much, much more independent [with Fabhalta], because I can take the tablets for myself.
The researchers noted that patients frequently highlighted the convenience of an oral therapy as a major benefit of Fabhalta. Soliris is given intravenously, or via an infusion directly into the bloodstream, every two weeks after the initial loading doses. Ultomiris can be administered by intravenous infusion or a subcutaneous, or under-the-skin, injection.
“It’s much more comfortable to take pills rather than having an injection in your arm,” one patient said during an interview, also noting the “time component.” Having an oral therapy meant this patient was “much, much more independent, because I can take the tablets for myself.”
Another patient reported being “absolutely very satisfied,” saying Fabhalta “allows an enormously better quality of life compared with the previous drug.”
The scientists highlighted that this study was limited to interviews from a small number of patients who might not represent the broader population of people with PNH. Also, certain topics were discussed in some interviews but not others, further limiting the amount of data available.
Still, the team said these findings shed light on patients’ experiences with Fabhalta and “support and contextualize the positive efficacy results demonstrated in the … [Fabhalta] clinical trials.”
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