Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Trends

Growth Drivers

• Increasing regulatory approvals for novel therapies: The paroxysmal nocturnal hemoglobinuria treatment sector is experiencing a surge in approvals for therapies boosted by the rising demands for treatments. PNH is classified as a rare disease and Rare Disease Advisor estimates PNH cases in the U.S. to be around 0.6 to 6.1 cases per million people. Growing research and awareness of the rare blood disorder has hastened clinical trials and approval rates by regulatory bodies, allowing pharmaceutical companies to distribute treatment options in the markets. Fast-track approval of new biologics and gene therapies usher in advanced treatments such as complement inhibitors to the paroxysmal nocturnal hemoglobinuria (PNH) treatment market swiftly. For instance, in February 2024, Chugai Pharmaceuticals reported China to be the first country to approve Crovalimab for PNH treatment, and the drug is poised to improve patient convenience by allowing self-injection every four weeks.
• Advancements in complement inhibitors: The growing research and development of complement inhibitors such as C5 inhibitors ravulizumab and eculizumab benefit the global paroxysmal nocturnal hemoglobinuria treatment sector. These biologics target the root cause of PNH patients. Additionally, therapeutic breakthroughs improving patient outcomes and convenience assist the paroxysmal nocturnal hemoglobinuria (PNH) treatment market’s growth. For instance, in October 2023, the FDA approved the complement C5 inhibitor Zilucoplan under the market name Zilbrysq. As investments in research increase, it is positioned to boost studies on next-generation complement inhibitors that can improve the efficacy of treatments.
• Expansion of precision medicine and gene therapy: Global healthcare trends are witnessing a favorable push for precision medicine to benefit the paroxysmal nocturnal hemoglobinuria (PNH) treatment market. Advancements in CRISPR gene editing can address the genetic mutations responsible for PNH. For instance, in January 2021 the creation of animal models for PNH using CRISPR gene editing advanced the understanding of the mechanism of pathogenesis of PNH. Additionally, emerging therapies have the potential to provide curative solutions that can reduce lifelong dependency on medication. For instance, in December 2023, Novartis presented 48-week research results from the Phase 3 APPLY-PNH trial that long-term efficacy of Fabhalta (Iptacopan) in adults suffering from PNH.

Challenges

• Limited awareness and diagnosis: The awareness of PNH remains limited owing to the rarity of the disorder. Emerging paroxysmal nocturnal hemoglobinuria (PNH) treatment markets are facing barriers in the growth of the paroxysmal nocturnal hemoglobinuria treatment sector owing to a lack of awareness, lesser diagnosis, and volatile healthcare infrastructure. Delayed diagnosis can prevent patients from being administered timely treatment and also reduce applications of novel therapeutics. Additionally, complications of PNH overlap with other conditions such as anemia that can add to challenges in diagnosis.
• High cost of treatment: The high cost of treatment associated with PNH can hinder the paroxysmal nocturnal hemoglobinuria (PNH) treatment market’s growth curve. Patients diagnosed with PNH often develop anemia which adds to treatment costs. Additionally, the complicated treatment procedures require specialists and a medical team that can add to the costs. The difficulties in marrow transplantation add to the challenges in treatment. The high price can deter patients from seeking treatment and put a strain on healthcare systems. As cost containment pressures rise globally, pharmaceutical companies face the issue of balancing affordability with profits.