As the PNH Market Continues to Crowd, Spherix Announces New Suite of Advisory Services to Explore Continued Unmet Needs and Track New Launches
26 Junio 2024 - 3:00AM
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.
As a trusted advisor and industry thought leader, Spherix’s
unparalleled market insights and advisory services empower clients
to make better decisions and unlock opportunities for growth.
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release are trademarks of their respective holders. The findings
and opinions expressed within are based on Spherix Global Insight's
analysis and do not imply a relationship with or endorsement.
[1]
https://rarediseases.org/rare-diseases/paroxysmal-nocturnal-hemoglobinuria/
Tucker Hurtado, Hematology Launch Franchise Head
Spherix Global Insights
4848794284
tucker.hurtado@spherixglobalinsights.com