CHMP recommends EU approval of Roche’s PiaSky for people with PNH, a rare, life-threatening blood condition
28 Juni 2024 - 1:00PM
UK Regulatory
CHMP recommends EU approval of Roche’s PiaSky for people with PNH,
a rare, life-threatening blood condition
- If approved, PiaSky will be
the first monthly subcutaneous (SC) treatment for paroxysmal
nocturnal haemoglobinuria (PNH) in the EU
- Additionally, with the
option to self-administer, PiaSky may provide an alternative to
existing intravenous (IV) C5 inhibitors, potentially helping to
reduce treatment burden1
- The recommendation is based
on the COMMODORE 2 study results, where SC PiaSky given every month
demonstrated equivalent disease control and comparable safety to IV
eculizumab given every two
weeks2,3
Basel, 28 June 2024 - Roche (SIX: RO, ROG; OTCQX: RHHBY)
announced today that the European Medicines Agency’s (EMA)
Committee for Medicinal Products for Human Use (CHMP) has adopted a
positive opinion for PiaSky® (crovalimab) for the treatment of
paroxysmal nocturnal haemoglobinuria (PNH). The CHMP has
recommended PiaSky, a novel recycling monoclonal antibody that
inhibits the complement protein C5, for use in adults and
adolescents (12 years of age or older with a weight of 40 kg) who
are either new to, or have been previously treated with C5
inhibitors. If approved, PiaSky will be the first monthly
subcutaneous (SC) treatment for PNH in the European Union, with the
option to self-administer following adequate training. This may
provide an alternative option to current C5 inhibitors that require
regular intravenous (IV) infusions, potentially helping to reduce
treatment burden and disruption to the lives of people with PNH and
their caregivers.1 A final decision regarding the
approval of PiaSky is expected from the European Commission in the
near future.
“People living with PNH face lifelong treatment, often requiring
frequent intravenous infusions and time-consuming clinic visits,”
said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and
Head of Global Product Development. “With the option to
self-administer once a month, today’s recommendation may therefore
offer patients and caregivers in Europe more freedom in their
day-to-day lives.”
PNH is a rare and life-threatening blood condition, which
affects approximately 20,000 people worldwide. In PNH, red blood
cells are destroyed by the complement system – part of the innate
immune system. This causes symptoms such as anaemia, fatigue and
blood clots, and can lead to kidney disease. C5 inhibitors –
treatments that block part of the complement system cascade – have
been shown to be effective in treating PNH. PiaSky is a novel C5
inhibitor that is recycled within the bloodstream, allowing the
medicine to bind and inhibit the C5 protein multiple times and to
act longer in the body with a small volume of medicine. This
enables SC administration every four weeks, following an initial IV
infusion and weekly SC loading doses in the first month of
treatment.1,2,4-6
The CHMP recommendation is based on the results from the Phase
III COMMODORE 2 study in people with PNH who have not been
previously treated with C5 inhibitors. Results from the study
demonstrated that PiaSky, administered as SC injections every four
weeks, achieved disease control and was well-tolerated. PiaSky was
non-inferior with comparable safety to eculizumab, an existing
standard of care C5 inhibitor, given intravenously every two weeks.
The rate of adverse events in people treated with PiaSky was
similar to treatment with eculizumab (78% versus 80%,
respectively). The application included supportive data from two
additional Phase III studies, the COMMODORE 1 study, in people with
PNH switching from currently approved C5 inhibitors, and the
COMMODORE 3 study in people new to C5 inhibitor treatment in
China.2,7,8,9
PiaSky is the first monthly SC treatment approved in the US,
Japan and China for people with PNH based on results of the
COMMODORE studies.2,7,9
PiaSky is being investigated in a
broad clinical development programme, including five ongoing Phase
III studies and three earlier phase studies in complement-mediated
diseases, including PNH, atypical haemolytic uremic syndrome and
sickle cell disease.2,7,9-14
About PiaSky® (crovalimab)
PiaSky® (crovalimab) is a novel recycling monoclonal antibody that
inhibits the complement protein C5 and is designed to block the
complement system – a vital part of the innate immune system that
acts as the body’s first line of defence against infection. PiaSky
has been engineered by Chugai Pharmaceutical Co. Ltd to address
certain needs of people living with complement-mediated diseases,
including providing patients with a potential for
self-administration following adequate training.
PiaSky works by binding to C5, blocking the last step of the
complement cascade and delivering rapid and sustained complement
inhibition. It is also recycled within the bloodstream, enabling
small volume subcutaneous administration every four weeks. In
addition, PiaSky binds to a different C5 binding site from current
treatments, which has the potential to provide a treatment option
for people with specific C5 gene mutations who do not respond to
current therapies.
PiaSky is a monthly subcutaneous treatment approved in the US,
Japan and China for people with paroxysmal nocturnal
haemoglobinuria, based on the Phase III COMMODORE studies. It is
also being evaluated in atypical haemolytic uremic syndrome and
sickle cell disease.1,2,7,9-14
About the COMMODORE 2 study
The COMMODORE 2 study is a Phase III, randomised, open-label study
evaluating the efficacy and safety of PiaSky® (crovalimab) versus
eculizumab in people with paroxysmal nocturnal haemoglobinuria
(PNH) who have not been treated previously with C5 inhibitors. The
study’s co-primary efficacy endpoints measure transfusion avoidance
and control of haemolysis (the ongoing destruction of red blood
cells measured by lactate dehydrogenase levels). The adults
enrolled in the study were randomised in a 2:1 ratio to be treated
with either subcutaneous (SC) PiaSky every four weeks or
intravenous eculizumab every two weeks. The participants who were
less than 18 years old were included in a non-randomised treatment
arm and were treated with SC PiaSky every four
weeks.3
About Roche
Founded in 1896 in Basel, Switzerland, as one of the first
industrial manufacturers of branded medicines, Roche has grown into
the world’s largest biotechnology company and the global leader in
in-vitro diagnostics. The company pursues scientific excellence to
discover and develop medicines and diagnostics for improving and
saving the lives of people around the world. We are a pioneer in
personalised healthcare and want to further transform how
healthcare is delivered to have an even greater impact. To provide
the best care for each person we partner with many stakeholders and
combine our strengths in Diagnostics and Pharma with data insights
from the clinical practice.
In recognising our endeavour to pursue a long-term perspective
in all we do, Roche has been named one of the most sustainable
companies in the pharmaceuticals industry by the Dow Jones
Sustainability Indices for the fifteenth consecutive year. This
distinction also reflects our efforts to improve access to
healthcare together with local partners in every country we
work.
Genentech, in the United States, is a wholly owned member of the
Roche Group. Roche is the majority shareholder in Chugai
Pharmaceutical, Japan.
For more information, please visit www.roche.com.
All trademarks used or mentioned in this release are protected
by law.
References
1Fukuzawa T, et al. Long lasting neutralisation of C5 by
SKY59, a novel recycling antibody, is a potential therapy for
complement-mediated diseases. 2017; Sci Rep 7, 1080.
2Roth A, et al. The Phase III, Randomised COMMODORE 2
Trial: Results from a Multicentre Study of Crovalimab vs Eculizumab
in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Naïve to
Complement Inhibitors. Presentation at European Hematology
Association (EHA) Annual Congress; 2023 June 08-13. Abstract
#S181.
3 COMMODORE 2 (NCT04434092). [Internet; cited June 2024]
Available at:
https://www.clinicaltrials.gov/ct2/show/NCT04434092.
4Grand View Research. Paroxysmal nocturnal
hemoglobinuria (PNH) treatment market size, share and trends
analysis report by treatment and segment forecasts, 2018 – 2025.
[Internet; cited June 2024]. Available at:
https://www.grandviewresearch.com/industry-analysis/paroxysmal-nocturnal-hemoglobinuria-pnh-market.
5 National Organization for Rare Diseases. Paroxysmal
nocturnal hemoglobinuria. [Internet; cited June 2024]. Available
at:
https://rarediseases.org/rare-diseases/paroxysmal-nocturnal-hemoglobinuria/.
6Harder M, et al. Incomplete inhibition by eculizumab:
mechanistic evidence for residual C5 activity during strong
complement activation. Blood. 2017;129:970-980.
7Scheinberg P, et al. Phase III Randomised, Multicentre,
Open-Label COMMODORE 1 Trial: Comparison of Crovalimab Vs
Eculizumab in Complement Inhibitor-Experienced Patients with
Paroxysmal Nocturnal Hemogobinuria (PNH). Presentation at European
Hepatology Association (EHA) Annual Congress; 2023 June 08-13.
Abstract #S183.
8COMMODORE 1 (NCT04432584). [Internet; cited June 2024]
Available at:
https://www.clinicaltrials.gov/ct2/show/NCT04432584.
9Liu H, et al. Six-month Crovalimab Extension in the
Phase III COMMODORE 3 Study: Updated Efficacy and Safety Results in
Complement Inhibitor-Naive Patients with Paroxysmal Nocturnal
Hemoglobinuria. Poster presentation at European Hematology
Association (EHA) Annual Congress; 2023 June 08-13. Abstract
#P785.
10COMMUTE-p (NCT04958265). [Internet; cited June 2024]
Available at:
https://www.clinicaltrials.gov/ct2/show/NCT04958265.
11COMMUTE-a (NCT04861259). [Internet; cited June 2024]
Available at:
https://www.clinicaltrials.gov/ct2/show/NCT04861259.
12Nishimura J, et al. Crovalimab for Treatment of
Patients With Paroxysmal NocturnalHemoglobinuria And Complement C5
Polymorphism – Experience From The Composer Phase I/II Study. EHA
Library. 2020; Abstract PB1992.
13CROSSWALK-a (NCT04912869) ClinicalTrials.gov.
[Internet; cited June 2024]. Available at:
https://clinicaltrials.gov/ct2/show/NCT04912869.
14CROSSWALK-c (NCT05075824) ClinicalTrials.gov.
[Internet; cited June 2024]. Available at:
https://clinicaltrials.gov/ct2/show/NCT05075824.
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