MarketVue® Report
Paroxysmal Nocturnal Hemoglobinuria (PNH) (U.S.), 2022

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Paroxysmal Nocturnal Hemoglobinuria (PNH) (U.S.), July 2022

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The MarketVue®: Paroxysmal Nocturnal Hemoglobinuria (PNH) market landscape report combines primary (KOL interviews and survey data) and secondary market research to empower strategic decision-making and provide a complete view of the market.
 
Every MarketVue® includes a disease overview, epidemiology (US and EU5), current treatment, unmet needs, pipeline and access and reimbursement chapter.
 
Topics covered in this report:
  • Disease overview: Review the disease pathophysiology and potential druggable targets
  • Epidemiology: Understand prevalence, diagnosed and drug-treated prevalence of the population and key market segments
  • Current treatment: Understand the treatment decision tree and strengths and weaknesses of current on-label and off-label treatment
  • Unmet needs: Identify opportunities to address treatment or disease management gaps
  • Pipeline analysis: Compare current and emerging therapy clinical development strategy; their performance on efficacy, safety, and delivery metrics; and their potential to address unmet needs
  • Value and access: Gain insights into the drug pricing landscape and payer controls within the disease market or analogous markets
 
Methodology:
Research for the MarketVue®: Paroxysmal Nocturnal Hemoglobinuria (PNH) report is supported by 8 qualitative interviews with key opinion leaders, a quantitative survey with 23 U.S. physicians and secondary research.
 
Geographies covered:
United States plus epidemiology for EU5 (France, Germany, Italy, Spain, United Kingdom)
 
Key companies mentioned:
  • Apellis Pharmaceuticals
  • Hoffmann-La Roche
  • Novartis
  • Regeneron
  • Alexion/AstraZenenca
  • Amgen
  • Biocryst
  • Arrowhead
 
Key drugs mentioned:
  • Ravulizumab (Ultomiris)
  • Eculizumab (Soliris)
  • Pegcetacoplan (Empaveli)
  • Crovalimab (PiaSky)
  • Iptacopan / LNP023 (Fabhalta)
  • Pozelimab (Veopoz)
  • Cemdisiran / ALN-CC5
  • Danicopan / ALXN2040 (Voydeya)
  • ABP-959 / Eculizumab biosimilar
  • Vemircopan / ALXN2050
  • BCX-9930
  • Aro-C3
1. DISEASE OVERVIEW
A rare, acquired disease marked by life-threatening hemolysis, anemia, and thrombosis
Figure 1.1. Select clinical manifestations of PNH
Figure 1.2. Simplified overview of PNH clonal expansion
The complement cascade has multiple drug targets for treating PNH
Figure 1.3 PNH drug targets in the alternative complement pathway
2. EPIDEMIOLOGY & PATIENT POPULATIONS
Disease definition
Figure 2.1. Diagnosed prevalent cases of PNH by region
Table 2.1 Diagnosed prevalent and drug-treated patients in the US and EU5
3. CURRENT TREATMENT
Overview
Figure 3.1. Treatment goals for PNH
Figure 3.2. PNH Current Therapy Patient Share, U.S.
Persistently low hemoglobin levels and extravascular hemolysis is a key downside of anti-C5 therapy
Table 3.1. Upsides and downsides of current PNH treatments
Diagnosis and treatment of PNH
Figure 3.3. Treatment algorithm for PNH
Complement inhibition has taken PNH from a terminal illness to chronic disease management
Table 3.2. Evolution of PNH treatment
Pegcetacoplan is capturing patient share mostly as second-line therapy
Figure 3.4. Percentage of U.S. hematologists who are currently prescribing pegcetacoplan (n=23)
Figure 3.5. Percentage of pegcetacoplan users (n=12) using the therapy by line, U.S. Pegcetacoplan use by line of therapy, U.S.
The convenience of pegcetacoplan’s subcutaneous infusion administration is a matter of debate
Figure 3.6. Percentage U.S. of hematologists agree that pegcetacoplan’s dosing and administration are preferable over that of ravulizumab (n=24)
Key treatment dynamics that shape disease management and drug use in PNH
Table 3.3. Must-know PNH treatment dynamics for now and the future
The PNH market stands to become highly fragmented within the next 5-7 years
Figure 3.7. Important dynamics of PNH market evolution
4. UNMET NEED
Overview
Figure 4.1. Top unmet needs in PNH
Figure 4.2. Hematologist-reported unmet needs in PNH
Persistent anemia is a notable areas that needs improvement beyond eculizumab and ravulizumab
Table 4.1 Hematologist-reported levels of minimally accepted and suboptimal Hb in treated PNH patients
5. PIPELINE ANALYSIS
Overview
Figure 5.1. Percentage of hematologists (n=23) rating PNH therapy target as “promising”
Figure 5.2. Number of emerging PNH therapies whose clinical trial primary end points target top unmet needs
The late-stage pipeline for PNH is very active
Table 5.1. Emerging PNH therapies in Phase 3
The early-stage pipeline is also active and focused on largely similar mechanisms
Table 5.2. Emerging PNH therapies in Phase 1/2
Alexion is expanding its PNH franchise while Novartis aims to shake things up with oral therapy
6. VALUE & ACCESS
Overview
Table 6.1. Current PNH therapy pricing, U.S.
Table 6.2. Typical U.S. commercial payer coverage of PNH therapies
Eculizumab in PNH
Overview
Figure 6.1. Annual cost (WAC) of Soliris (000s), 2007 – 2021
Figure 6.2. Key market access pillars for Soliris in PNH
Ravulizumab and pegcetacoplan in PNH
Overview
Figure 6.2. Strategies used by ravulizumab and pegcetacoplan to compete with eculizumab
Figure 6.4. PNH patients by insurance type. U.S.
7. METHODOLOGY
Primary market research approach
Epidemiology methodology
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