https://mb.cision.com/Public/18595/3835417/b827dbaf6a1a5101_800x800ar.png
** UCB receives CHMP positive opinion of zilucoplan for the treatment of ad=
ults with generalized myasthenia gravis in Europe
------------------------------------------------------------
=C2=B7 The Committee for Medicinal Products for Human Use (CHMP) positive o=
pinion^1 is based on the pivotal Phase 3 RAISE study in generalized myasthe=
nia gravis (gMG) in adult patients which demonstrated that treatment with z=
ilucoplan resulted in statistically significant and clinically meaningful i=
mprovements in gMG-specific efficacy outcomes^2
=C2=B7 If approved by the European Commission zilucoplan will be the first =
once-daily subcutaneous (SC) targeted peptide inhibitor of complement compo=
nent 5 (C5 inhibitor) and the only gMG-targeted therapy for self-administra=
tion by adult patients with AChR antibody positive gMG
=C2=B7 CHMP positive opinion in Europe follows recent FDA approval of rozan=
olixizumab-noli for the treatment of generalized myasthenia gravis (gMG) in=
adult patients in the U.S. who are anti-acetylcholine receptor (AChR) or a=
nti-muscle-specific tyrosine kinase (MuSK) antibody positive3
=C2=B7 UCB=E2=80=99s two different medicines for gMG, each with a distinct =
mechanism of action, aim to offer a unique portfolio of treatments that emb=
ody our commitment to addressing the gMG community=E2=80=99s unmet needs
Brussels (Belgium) Friday 15 September 2023 =E2=80=93 UCB (Euronext Brussel=
s: UCB), a global biopharmaceutical company, today announced that the Commi=
ttee for Medicinal Products for Human Use (CHMP) of the European Medicines =
Agency (EMA) has issued a positive opinion recommending granting marketing =
authorization for zilucoplan in the European Union (EU) as an add-on to sta=
ndard therapy for the treatment of generalized myasthenia gravis (gMG) in a=
dult patients who are anti acetylcholine receptor (AChR) antibody positive.=
^1
The CHMP=E2=80=99s positive opinion is now being reviewed by the European C=
ommission, which grants centralized marketing authorizations for medicinal =
products in the EU. Feedback from the commission is anticipated before the =
end of the year.=C2=A0
Following approval, zilucoplan will be the first once-daily subcutaneous (S=
C), targeted peptide inhibitor of complement component 5 (C5 inhibitor) and=
the only self-administered gMG therapy for use by adult patients with AChR=
antibody positive gMG.
As a C5 inhibitor, zilucoplan inhibits complement-mediated damage to the ne=
uromuscular junction through its targeted dual mechanism of action.^2 Benef=
its of SC self-administration can include reduced traveling time to and fro=
m hospitals, decreased interference with work obligations, and increased in=
dependence. Unlike monoclonal antibody C5 inhibitors, as a peptide, zilucop=
lan can be used concomitantly with intravenous immunoglobulin and plasma ex=
change, without the need for supplemental dosing.^2
UCB=E2=80=99s RAISE study^2, published earlier this year in the Lancet Neur=
ology journal, demonstrated that zilucoplan delivered rapid, consistent, st=
atistically significant and clinically meaningful benefits in different pat=
ient-and-clinician-reported outcomes - Myasthenia Gravis-Activities of Dail=
y Living (MG-ADL) score, Quantitative Myasthenia Gravis (QMG) score, Myasth=
enia Gravis Composite (MGC) score and Myasthenia Gravis Quality of Life 15-=
item scale (MG-QoL15r)* - at week 12 in a broad population of mild to sever=
e adult patients with AChR antibody positive gMG. Additionally, rapid impro=
vements in fatigue were observed as an exploratory endpoint.=C2=A0
=E2=80=9CUntil now, people living with gMG have only had access to C5 thera=
py intravenously, which can be inconvenient and time-consuming. This positi=
ve CHMP opinion for zilucoplan is a significant step towards our goal of de=
livering a treatment to address the unmet needs of people living with gMG=
=E2=80=9D, said Iris Loew-Friedrich, Executive Vice-President and Chief Med=
ical Officer at UCB. =E2=80=9CIf approved, we hope zilucoplan, a self-admin=
istered, once daily, subcutaneous targeted C5 inhibitor, will be able to he=
lp a broad population of mild to severe adult patients with AChR-antibody p=
ositive gMG.We would like to extend our thanks to the patients, care partne=
rs, and investigators who participated in the RAISE study, and to our emplo=
yees and collaborators, whose dedication and commitment to the gMG communit=
y made this important milestone possible.=E2=80=9D
gMG is a rare, chronic, heterogeneous, unpredictable autoimmune disease cha=
racterized by dysfunction and damage at the neuromuscular junction (NMJ).^4=
,5,6 gMG has a global prevalence of 100=E2=80=93350 cases per every 1 milli=
on people.^5=C2=A0
The CHMP positive opinion recommending the approval of zilucoplan is suppor=
ted by safety and efficacy data from the Phase 3 RAISE study (NCT04115293),=
published in The Lancet Neurology in May 2023.^2 The primary endpoint for =
the RAISE study was change from baseline to Week 12 in the Myasthenia Gravi=
s-Activities of Daily Living (MG-ADL) score. A statistically significant an=
d clinically meaningful difference favoring zilucoplan in comparison to pla=
cebo was observed in the MG-ADL total score change from baseline: least squ=
ares mean change =E2=88=924=C2=B739 [95% CI =E2=80=935=C2=B728 to =E2=80=93=
3=C2=B750] vs =E2=88=922=C2=B730 [=E2=80=933=C2=B717 to =E2=80=931=C2=B743]=
, least squares mean difference =E2=88=922=C2=B709 [=E2=88=923=C2=B724 to =
=E2=88=920=C2=B795]; p=3D0=C2=B70004. Secondary endpoints included change f=
rom baseline to Week 12 in QMG, MGC and MG-QoL15r. A statistically signific=
ant and clinically meaningful difference favoring zilucoplan compared to pl=
acebo was observed in the QMG total score change from baseline to Week 12 (=
p<0.0001), least squares mean change =E2=88=926.19 [95% CI =E2=88=927.29 to=
=E2=88=925.08] vs =E2=88=923.25 [=E2=88=924.32 to =E2=88=922.17]. Change f=
rom baseline to Week 12 in MGC in comparison to placebo was clinically mean=
ingful and statistically significant. MG-QoL 15r change from baseline to We=
ek 12 compared to placebo was also statistically significant.^2=C2=A0Change=
from baseline to week 12 in the Neuro-QoL short-form fatigue scale was an =
exploratory end point, therefore, p value was nominal, not multiplicity con=
trolled.
The most common adverse events (reported in at least 10% of patients treate=
d with zilucoplan) were injection-site bruising, headache, diarrhea and MG =
worsening.^2
=E2=80=9CWith this CHMP positive opinion of zilucoplan, we are very proud a=
nd excited to expand our support to the gMG community. Following the FDA ap=
proval and strong momentum with our FcRn blocker rozanolixizumab-noli in th=
e U.S., and with our tailored patient support services and commitment to wi=
despread access, I am confident that UCB will be the only company able to d=
eliver a portfolio of two targeted therapies with different mechanisms of a=
ction and the experience to provide truly individualized transformational p=
atient value to people living with this often-debilitating rare disease.=E2=
=80=9D said Jean-Christophe Tellier, CEO, UCB.
Zilucoplan is also currently under review by the Japanese Pharmaceuticals a=
nd Medical Devices Agency (PMDA), the U.S. Food and Drug Administration (FD=
A), the Australian Therapeutic Goods Administration (TGA) and Health Canada=
for the treatment of adults with gMG. Responses from the PMDA and FDA are =
expected by the end of Q4 2023. Responses from the TGA and Health Canada ar=
e expected by H1 2024. Orphan designation was granted by the European Commi=
ssion in 2022 to zilucoplan for the treatment of myasthenia gravis.^7=C2=A0
=C2=A0=C2=A0 =C2=A0
The CHMP positive opinion of zilucoplan follows the recent FDA approval in =
the U.S. of rozanolixizumab-noli for the treatment of generalized myastheni=
a gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR)=
or anti-muscle-specific tyrosine kinase (MuSK) antibody positive^3. Rozano=
lixizumab-noli is currently only approved in the U.S. and is under review b=
y the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) and the Eu=
ropean Medicines Agency (EMA) for the treatment of adults with gMG. Respons=
es from regulatory agencies to these submissions are expected during H2 202=
3 and H1 2024.
In progressing a portfolio of medicines for the treatment of gMG, with the =
aim of providing HCPs the option of addressing either complement activation=
or pathogenic antibodies for appropriate patients, UCB hopes to offer a co=
mprehensive portfolio of targeted therapeutics, embodying a commitment to a=
ddressing the gMG community=E2=80=99s unmet needs.
About zilucoplan
Zilucoplan is a once-daily SC, self-administered peptide inhibitor of compl=
ement component 5 (C5 inhibitor). As a C5 inhibitor, zilucoplan inhibits co=
mplement-mediated damage to the neuromuscular junction through its targeted=
dual mechanism of action.^2=C2=A0
The safety and efficacy of zilucoplan have not been established and it is n=
ot currently approved for use in any indication by any regulatory authority=
worldwide.
About Generalized Myasthenia Gravis (gMG)
gMG is a rare autoimmune disease with a global prevalence of 100=E2=80=9335=
0 cases per every 1 million people.^5 People living with gMG can experience=
a variety of symptoms, including severe muscular weakness that can result =
in double vision, drooping eyelids, difficulty with swallowing, chewing and=
talking, as well as life-threatening weakness of the muscles of respiratio=
n.^4,8
In gMG, pathogenic autoantibodies can impair synaptic transmission at the n=
euromuscular junction (NMJ) by targeting specific proteins on the post-syna=
ptic membrane.^9 This disrupts the ability of the nerves to stimulate the s=
keletal muscle and results in a weaker contraction. gMG can occur in any ra=
ce, gender or age.^4,8
About the RAISE study^2
The RAISE study (NCT04115293) was a multi-center, Phase 3, randomized, doub=
le-blind, placebo-controlled study to confirm the efficacy, safety profile,=
and tolerability of zilucoplan in adult patients with anti-acetylcholine r=
eceptor (AChR) antibody positive gMG. Patients were randomized in a 1:1 rat=
io to receive daily subcutaneous (SC) injections of 0.3 mg/kg zilucoplan or=
placebo for 12 Weeks.
The primary endpoint for the RAISE study was change from baseline to Week 1=
2 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score. Secon=
dary endpoints included change from baseline in the Quantitative Myasthenia=
Gravis (QMG) score, the Myasthenia Gravis Composite (MGC) and the Myasthen=
ia Gravis Quality of Life 15 revised (MG-QoL15r) score from baseline to Wee=
k 12, time to first rescue therapy, the proportion of patients with minimal=
symptom expression (MSE) (defined as MG-ADL of 0 or 1 without rescue thera=
py), the proportion with a =E2=89=A53-point reduction in MG-ADL without res=
cue therapy and the proportion with a =E2=89=A55-point reduction in QMG wit=
hout rescue therapy, all measured at Week 12. The secondary safety endpoint=
was incidence of TEAEs. Patients who completed the RAISE trial had the pos=
sibility to enter the open-label extension study, RAISE-XT (NCT04225871).^2=
=C2=A0
For more information about the trial visit https://clinicaltrials.gov/ct2/s=
how/NCT04115293.=C2=A0
* The threshold for clinical meaningfulness for MG-QoL 15r has not be estab=
lished
For further information, contact UCB:=C2=A0
Global Rare Disease Communications
Jim Baxter
T+32.2.473.78.85.01=C2=A0
jim.baxter@ucb.com=C2=A0
Corporate Communications, Media Relations
Laurent Schots=C2=A0
T+32.2.559.92.64=C2=A0
Laurent.schots@ucb.com=C2=A0
Investor Relations
Antje Witte =C2=A0 =C2=A0=C2=A0
T +32.2.559.94.14=C2=A0
antje.witte@ucb.com
About UCB=C2=A0
UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company =
focused on the discovery and development of innovative medicines and soluti=
ons to transform the lives of people living with severe diseases of the imm=
une system or of the central nervous system. With approximately 8,600 peopl=
e in approximately 40 countries, the company generated revenue of =E2=82=AC=
5.5 billion in 2022. UCB is listed on Euronext Brussels (symbol: UCB). Foll=
ow us on Twitter: @UCB_news.
Forward looking statements=C2=A0
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t limitation, statements containing the words =E2=80=9Cbelieves=E2=80=9D, =
=E2=80=9Canticipates=E2=80=9D, =E2=80=9Cexpects=E2=80=9D, =E2=80=9Cintends=
=E2=80=9D, =E2=80=9Cplans=E2=80=9D, =E2=80=9Cseeks=E2=80=9D, =E2=80=9Cestim=
ates=E2=80=9D, =E2=80=9Cmay=E2=80=9D, =E2=80=9Cwill=E2=80=9D, =E2=80=9Ccont=
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essful as UCB may have believed at the moment of acquisition. Also, UCB or =
others could discover safety, side effects or manufacturing problems with i=
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ny market, or at any particular time, nor can there be any guarantee that s=
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UCB is providing this information, including forward-looking statements, on=
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UCB is following the worldwide developments diligently to assess the financ=
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References:
1. EMA CHMP Confirmation. Data on file, UCB September 2023.
2. Howard JF Jr et al. Safety and efficacy of zilucoplan in patients with g=
eneralised myasthenia gravis (RAISE): a randomised, double-blind, placebo-c=
ontrolled, phase 3 studyLancet Neurol. 2023;22:395-406.=C2=A0
3. US Food and Drug Administration. Novel Drug approvals for 2023. https://=
www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therap=
eutic-biological-products/novel-drug-approvals-2023. Accessed September 202=
3.
4. National Institute of Neurological Disorders and Stroke. 2022. Myastheni=
a Gravis Fact Sheet. https://www.ninds.nih.gov/myasthenia-gravis-fact-sheet=
. Accessed September 2023.
5. Punga AR, et al. Epidemiology, diagnostics, and biomarkers of autoimmune=
neuromuscular junction disorders. Lancet Neurol. 2022;21(2):176-88.
6. Howard JF. Myasthenia gravis: The role of complement at the neuromuscula=
r junction. Ann N Y Acad Sci. 2018;1412:113-128.
7. European Medicines Agency. EU/3/22/2650: Orphan designation for the trea=
tment of myasthenia gravis. https://www.ema.europa.eu/en/medicines/human/or=
phan-designations/eu-3-22-2650. Accessed August 2023
8. Myasthenia Gravis Foundation of America. MG Quick Facts. https://myasthe=
nia.org/MG-Education/MG-Quick-Facts. Accessed September 2023.
9. Juel VC, Massey JM. Myasthenia gravis. Orphanet J Rare Dis. 2007;2:44.
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