Novartis's oral complement factor B inhibitor Fabhalta has rapidly transformed the treatment landscape for paroxysmal nocturnal hemoglobinuria (PNH) in just 15 months since its December 2023 launch, according to new research from Spherix Global Insights.
The independent market intelligence firm's latest tracking study, based on insights from 51 U.S. hematologists, reveals Fabhalta has gained substantial traction in a market historically dominated by complement 5 (C5) inhibitors like Soliris and Ultomiris. Physician consideration for Fabhalta as a first-line therapy has doubled year-over-year, while second-line consideration has increased by one-third.
Shifting Treatment Paradigm in PNH
PNH is a rare and debilitating blood disorder affecting approximately 8,000 to 10,000 people in North America and Western Europe. Patients typically suffer from severe fatigue, and without treatment, PNH can progress to serious complications including hemolytic anemia, chronic kidney disease, and potentially fatal thrombosis.
The research highlights a growing consensus among hematologists that all PNH patients should receive a complement or factor inhibitor, regardless of disease burden. This represents a significant shift in treatment philosophy and reflects increasing confidence in newer therapeutic options.
"With the expansion of oral treatment options, medication adherence is becoming a stronger driver in prescribing decisions," notes the Spherix report. This trend benefits Fabhalta, which has now surpassed Empaveli, Voydeya, and PiaSky in recent prescription volume.
Competitive Landscape Evolution
While Alexion (now part of AstraZeneca) remains the most respected industry partner in the PNH space with its established treatments Soliris and Ultomiris, Novartis has made notable gains in awareness and influence. The company now ranks as the second-most impactful in physician prescribing decisions.
The PNH treatment landscape has diversified significantly with several newer entrants:
- Fabhalta (iptacopan) from Novartis: An oral factor B inhibitor
- Voydeya (ravulizumab-csyz) from Alexion/AstraZeneca: A long-acting C5 inhibitor
- PiaSky from Genentech/Roche: A novel complement pathway inhibitor
- Empaveli (pegcetacoplan) from Apellis: A C3 inhibitor
Biosimilar Disruption on the Horizon
The imminent introduction of biosimilar versions of eculizumab (Soliris) is expected to create significant market disruption. Hematologists project that up to half of their Soliris patients may transition to biosimilar eculizumab—either Bkemv from Amgen or Epysqli from Samsung Bioepis—by the end of 2025.
Interestingly, physicians anticipate that biosimilar availability may actually expand the overall treatment pool for PNH patients and trigger a ripple effect on other branded therapies as patients progress beyond C5 inhibitors.
"While many [physicians] indicate that they will not strongly resist payer-mandated switches, some indicate they are considering self-administered options such as Empaveli or Fabhalta more strongly as a way to sidestep the complexities associated with biosimilars," the report states.
Future Treatment Landscape
Looking ahead, there is mounting enthusiasm for Regeneron's investigational therapy pozelimab/cemdisiran, with physician awareness increasing tenfold year-over-year. Doctors view this potential treatment as a significant advance over existing options, and a majority expect to prescribe it within six months of approval.
Nearly half of PNH patients are considered potential candidates for the Regeneron therapy, positioning it as a likely disruptor to established treatments like Ultomiris and Soliris upon its introduction.
Clinical Implications
The evolving treatment landscape offers new hope for PNH patients, who historically had limited therapeutic options. The shift toward oral therapies may improve treatment adherence, while the introduction of biosimilars could potentially increase access to effective treatments for more patients.
Dr. Sarah Hendry, Hematology Franchise Head at Spherix Global Insights, emphasized the importance of these market dynamics: "With PNH treatment options expanding and biosimilars entering the market, understanding these dynamics is more crucial than ever for optimizing patient care."
Market Intelligence
Spherix's research combines its quarterly RealTime Dynamix tracking study with its first annual chart audit study in hematology, Patient Chart Dynamix: Paroxysmal Nocturnal Hemoglobinuria (US), released in late 2024. The latter analyzed 211 PNH patient charts from 88 U.S.-based hematologists to provide detailed insights into current treatment patterns.
These comprehensive studies offer pharmaceutical manufacturers strategic intelligence to navigate the increasingly complex PNH market, identify emerging opportunities, and address persistent challenges in patient care as the therapeutic landscape continues to evolve rapidly.
