As the PNH Market Continues to Fill, Spherix Announces New Suite of Consulting Services to Explore Persistent Unmet Needs and Track New Launches

Spherix Global Insights releases study on evolving market landscape, coupled with new research on familiarity and adoption of Novartis Fabhalta and Alexion/AstraZeneca Voydeya.

EXTON, PA, June 26, 2024 (GLOBE NEWSWIRE) — Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder in which red blood cells break prematurely, a process known as hemolysis. Although symptoms can vary greatly from person to person, patients often report fatigue and pain. Chronic hemolysis can also lead to serious complications such as blood clots and kidney disease.(1)

Treatment of 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 delivery as well as a new mechanism of action to the mix.

Despite the availability of these treatments, more than half of U.S.-based hematologists (n=55) surveyed this spring by Spherix Global Insights in its study Market Dynamix™: Paroxysmal nocturnal hemoglobinuria (United States) series reported a high, unmet need for new treatment options. Indeed, PNH ranks among the top three hematologic diseases in terms of unmet need, with physicians reporting that many of their patients were not optimally managed or continued to carry a high burden of morbidity due to extravascular hemolysis and transfusion dependence. In the words of a doctor in the Spherix community:

“There are several challenges (that we face in the care of patients with PNH):

Accurate and timely diagnosis
Rapid drug approval so patients benefit from immediate treatment and reduction of the disease process
Continued approval of treatment
Alternative options if the patient suffers from side effects. » – American 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 which received approval for its new complementary PNH treatment, Voydeya (danicopan). June 21st^stGenentech/Roche also received approval for its novel agent PiaSky (crovalimab) for PNH.

Spherix is closely following these developments with its Launch of Dynamix™:Innovations in the field of PNH (WE) series, tracking key performance indicators (KPIs) such as awareness, familiarity and perceptions among hematologists. Since March 2024, Spherix follows Fabhalta, and Voydeya was 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, Fabhalta has gained great familiarity and notoriety among doctors, and those who have prescribed the agent report being very satisfied with this drug. Most notably, hematologists anticipate increased use of the agent and growth in its market share over the next six months – largely due to its clinical data and convenient oral dosing.

Spherix’s ongoing research will track not only market developments, but also the development of new agents aimed at improving patient outcomes and quality of life. As Fabhalta, Voydeya and now PiaSky emerge across the therapeutic landscape, Spherix will provide insight into how these new therapies influence treatment patterns, physician preferences and patient outcomes. Later this year, Spherix plans to launch a patient record audit study to unveil true treatment paradigms and determine where patient needs remain on the agenda. Dynamix™ patient file: HPN (United States) study.

Launch Dynamix™ is an independent service providing quarterly in-depth analysis of promotional activity, barriers to adoption, and patient types gravitating to the launch brand.

Market Dynamix™ is an independent service that provides analysis on markets expected to experience a paradigm shift over the next three to five years. The information highlights market size, current therapeutic approaches, unmet needs and expert opinions on likely disruptions introduced by pipeline agents.

Dynamix of patient records™ is an independent, data-driven service that uncovers real treatment management models through rigorous analysis of large-scale patient record audits. The insights reveal the “why” behind treatment decisions and integrate specialist behavioral and demographic data to highlight differences between reported and actual treatment patterns.

About Spherix Global Insights

Spherix is a leading independent market intelligence and consulting company that delivers business value to the global life sciences industry, throughout the brand lifecycle.

The seasoned team of Spherix experts provides an unbiased and holistic view of the rapidly evolving specialty market landscape, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology and hematology. Spherix customers stay ahead with the insight of the broad Spherix physician community.

As a trusted advisor and industry thought leader, Spherix’s unparalleled market insights and advisory services enable clients to make better decisions and unlock growth opportunities.

To learn more about Spherix Global Insights, visit spherixglobalinsights.com or connect via LinkedIn.

For more details on Spherix’s leading market research reports and interactive dashboard offerings, visit or register here: https://clientportal.spherixglobalinsights.com

NOTICE: All company, brand or product names mentioned in this press release are registered trademarks of their respective owners. The findings and opinions expressed herein are based on Spherix Global Insight’s analysis and do not imply a relationship or endorsement.

(1) https://rarediseases.org/rare-diseases/paroxysmal-nocturnal-hemoglobinuria/

CONTACT: Tucker Hurtado, Hematology Launch Franchise Head Spherix Global Insights 4848794284 [email protected]