EXTON, PA, June 26, 2024 (GLOBE NEWSWIRE) -- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disorder in which red blood cells prematurely break apart, a process known as hemolysis. While the symptoms can vary significantly from person to person, patients often report fatigue and pain. Chronic hemolysis can also result in serious complications such as blood clots and renal disease.[1]
Treatment for PNH has historically revolved around complement inhibition, with Alexion/AstraZeneca’s Soliris (eculizumab) and Ultomiris (ravulizumab) leading the market. In 2021, Apellis’ Empaveli (pegcetacoplan) gained approval, adding a new mode of administration as well as a new mechanism of action to the mix.
Despite the availability of these treatments, more than one-half of US-based hematologists (n=55) surveyed this spring by Spherix Global Insights as part of its Market Dynamix™: Paroxysmal Nocturnal Hemoglobinuria (US) series reported a high unmet need for new treatment options. Indeed, PNH ranked among the top three hematological diseases in terms of unmet need, with physicians reporting that many of their patients were not being optimally managed or continued to endure severe disease burden with extravascular hemolysis and transfusion dependency. In the words of one physician in the Spherix community:
“There are several challenges [we face in managing PNH patients]:
- Accurate and timely diagnosis
- Prompt approval of medication for patients to have immediate treatment and reduction of disease process
- Ongoing approval of treatment
- Alternate options if the patient struggles with side effects.” – US Hematologist
However, 2024 has already been an exciting year for the PNH market, with significant developments over the past few months. First, Novartis received an indication in PNH for Fabhalta (iptacopan), followed by Alexion/AstraZeneca receiving approval for its new add-on PNH treatment Voydeya (danicopan). On June 21st, Genentech/Roche also received an approval for its new agent PiaSky (crovalimab) for PNH.
Spherix is closely monitoring these developments with its Launch Dynamix™: Innovations in PNH (US) series, tracking key performance indicators (KPIs) such as awareness, familiarity, and perceptions among hematologists. Since March 2024, Spherix has been tracking Fabhalta, with Voydeya added in June 2024. Coverage of PiaSky will begin once it is launched and established in the market.
With only a few months on the market so far, Fabhalta has achieved strong familiarity and awareness amongst physicians, and those who have prescribed the agent note high satisfaction with the drug. More notably, hematologists are projecting increased use of the agent and market share growth over the following six months – driven largely by its clinical data and convenient oral dosing.
Spherix's continued research will not only track market evolution, but the development of new agents aiming to improve patient outcomes and quality of life. As Fabhalta, Voydeya, and now PiaSky emerge on the treatment landscape, Spherix will provide insights on how these new therapies are influencing treatment patterns, physician preferences, and patient outcomes. Later in the year, Spherix plans to initiate a patient chart audit study to unveil true treatment paradigms and where patient needs remain in its Patient Chart Dynamix™: PNH (US) study.
Launch Dynamix™ is an independent service providing a quarterly deep dive into promotional activity, barriers to uptake, and patient types gravitating to the launch brand.
Market Dynamix™ is an independent service providing analysis of markets anticipated to experience a paradigm shift within the next three to five years. Insights highlight market size, current treatment approaches, unmet needs, and expert opinions on the likely disruption introduced by pipeline agents.
Patient Chart Dynamix™ is an independent, data-driven service unveiling real treatment management patterns through rigorous analysis of large-scale patient chart audits. Insights reveal the “why” behind treatment decisions and integrate specialists’ attitudinal & demographic data to highlight differences between stated and actual treatment patterns.
About Spherix Global Insights
Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle.
The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community.
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[1] https://rarediseases.org/rare-diseases/paroxysmal-nocturnal-hemoglobinuria/
