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** UCB receives CHMP positive opinion for rozanolixizumab for treatment of =
adults 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 MycarinG study in generalized myas=
thenia gravis (gMG) in adult patients,^2 which demonstrated treatment with =
rozanolixizumab resulted in statistically significant and clinically meanin=
gful improvements in gMG-specific outcomes compared to placebo,^2 including=
everyday activities such as breathing, talking, swallowing, and being able=
to rise from a chair^3
=C2=B7 If approved by the European Commission, rozanolixizumab will be the =
first emerging therapy approved in Europe for adults with both anti-acetylc=
holine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antib=
ody-positive gMG, the two most common subtypes of gMG=C2=A0
=C2=B7 The decision follows CHMP positive opinion for UCB=E2=80=99s zilucop=
lan in Europe for the treatment of adult patients with gMG earlier this yea=
r, alongside similar U.S. FDA and Japanese MHLW approvals of rozanolixizuma=
b and zilucoplan for the treatment of gMG in adult patients^4,5,6,7
=C2=B7 UCB is the first and only company to offer a gMG-focused portfolio, =
providing patients and clinicians the option of two targeted therapies for =
both anti-AChR and anti-MuSK antibody-positive gMG
=C2=A0
Brussels (Belgium) 10 November 2023, 18:00 CET =E2=80=93 UCB (Euronext Brus=
sels: UCB), a global biopharmaceutical company, today announced that the Co=
mmittee for Medicinal Products for Human Use (CHMP) of the European Medicin=
es Agency (EMA) has issued a positive opinion recommending granting marketi=
ng authorization for rozanolixizumab as an add-on to standard therapy for t=
he treatment of generalized myasthenia gravis (gMG) in adult patients who a=
re anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kina=
se (MuSK) antibody positive.^1=C2=A0
Rozanolixizumab 140 mg/ml solution for injection is a humanized IgG4 monocl=
onal antibody that binds to the neonatal Fc receptor (FcRn), resulting in t=
he reduction of circulating IgG.^2 If approved by the European Commission, =
rozanolixizumab will be the first emerging therapy approved in Europe for a=
dults with both anti-AChR and anti-MuSK antibody-positive gMG, the two most=
common subtypes of gMG.
In September 2023, the Committee for Medicinal Products for Human Use (CHMP=
) of the European Medicines Agency (EMA) also issued a positive opinion rec=
ommending granting marketing authorization for UCB=E2=80=99s zilucoplan in =
the European Union (EU) as an add-on to standard therapy for the treatment =
of gMG in adult patients who are anti-AChR antibody-positive^4. Zilucoplan =
is a once-daily subcutaneously (SC)-injected, self-administered peptide inh=
ibitor of complement component 5 (C5 inhibitor).^8
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.
=E2=80=9CThere is a significant need to bring more targeted, well-tolerated=
, effective treatment options that address the pathophysiology of gMG disea=
se. If approved by the European Commission, UCB will be the first and only =
company to offer a gMG-focused portfolio with rozanolixizumab and zilucopla=
n, providing patients and clinicians the option of two targeted therapies. =
We believe that the inclusion of both anti-muscle-specific tyrosine kinase =
(MuSK) antibody positive patients and anti-AChR positive patients within th=
e CHMP marketing authorization recommendation for rozanolixizumab could sup=
port clinicians to tailor their prescribing decisions to meet individual ne=
eds of their patients.=E2=80=99 said Iris Loew-Friedrich, Executive Vice-Pr=
esident and Chief Medical Officer at UCB. =E2=80=98This latest European gMG=
regulatory milestone, alongside approvals for both zilucoplan and rozanoli=
xizumab in the U.S. and Japan in recent months, further reinforces the comm=
itment we have made to the gMG community to help transform their experience=
s, outcomes and expectations. We are truly proud and excited for the future=
.=E2=80=9D
The CHMP opinion for rozanolixizumab is supported by safety and efficacy da=
ta from the pivotal Phase 3 MycarinG study (NCT03971422), published in The =
Lancet Neurology in May 2023.^2 The primary efficacy endpoint was the compa=
rison of the change from baseline between treatment groups (rozanolixizumab=
7mg/kg, rozanolixizumab 10mg/kg) or placebo in the MG-ADL score at Day 43.=
MG-ADL is a measurement tool that assesses the impact of gMG on daily func=
tions of 8 items that are typically affected in gMG. These include activiti=
es such as breathing, talking, swallowing, and being able to rise from a ch=
air.^3 Each item is assessed on a 4-point scale where a score of 0 represen=
ts normal function and a score of 3 represents loss of ability to perform t=
hat function. A total score ranges from 0 to 24, with the higher scores ind=
icating more impairment. Reductions in MG-ADL score from baseline to Day 43=
were greater in the rozanolixizumab 7 mg/kg group (least-squares mean chan=
ge =E2=80=933.37 [SE 0.49]) and in the rozanolixizumab 10 mg/kg group (=E2=
=80=933.40 [0.49]) than with placebo (=E2=80=930.78 [0.49]; for 7 mg/kg, le=
ast-squares mean difference =E2=88=922.59 [95% CI =E2=88=924.09 to =E2=88=
=921.25], p<0.001; for 10 mg/kg, =E2=88=922.62 [=E2=88=923.99 to =E2=88=921=
.16], p<0.001).^2
Secondary efficacy endpoints included change from baseline to Day 43 in the=
Myasthenia Gravis Composite (MG-C) and the Quantitative Myasthenia Gravis =
(QMC) scores. The MG-C is a 10-item instrument that measures the symptoms a=
nd signs of MG based on physician examination and patient history. Items ar=
e related to ptosis, double vision, eye closure, talking, chewing, swallowi=
ng, breathing, neck flexion, shoulder abduction, and hip flexion. Each item=
is scored on an ordinal scale with four possible categories and weighted. =
The total score ranges from 0 to 50, with higher scores indicating more sev=
ere impairments. The MG-C is composed of items originating from other scale=
s (i.e., QMG, MMT, MG-ADL). A statistically significant difference favoring=
rozanolixizumab compared to placebo was observed in the MG-C score change =
from baseline to Day 43 [least squares mean difference] -3.90 (95% CI (=E2=
=88=926.63 to =E2=88=921.25), p<0.001 for rozanolixizumab 7mg/kg; least-squ=
ares mean difference =E2=88=925.53 (95% CI =E2=88=928.30 to =E2=88=922.97),=
p<0.001 for rozanolixizumab 10mg/kg.^2=C2=A0
The QMG is a 13-item categorical grading system that assesses muscle weakne=
ss. Each item is assessed on a 4-point scale where a score of 0 represents =
no weakness and a score of 3 represents severe weakness. A total possible s=
core ranges from 0 to 39, where higher scores indicate more severe impairme=
nt.^9 A statistically significant difference favoring rozanolixizumab compa=
red to placebo was observed in the QMG total score change from baseline to =
Day 43 [least squares mean difference -3.48 (95% CI (=E2=88=925.61 to =E2=
=88=921.58), p<0.001 for rozanolixizumab 7mg/kg; least-squares mean differe=
nce =E2=88=924.76 (95% CI =E2=88=926.82 to =E2=88=922.86), p<0.001 for roza=
nolixizumab 10mg/kg.^2=C2=A0
The most common adverse reactions (reported in at least 10% of patients tre=
ated with rozanolixizumab) were headache, diarrhea and pyrexia.^1=C2=A0
gMG is a rare, chronic, heterogeneous, unpredictable autoimmune disease cha=
racterized by dysfunction and damage at the neuromuscular junction (NMJ).^1=
0,11,12 gMG has a global prevalence of 100=E2=80=93350 cases per every 1 mi=
llion people.^11=C2=A0
=E2=80=9CWith the news of the CHMP=E2=80=99s positive opinion of rozanolixi=
zumab, we are very proud and excited to potentially provide the gMG communi=
ty with further treatment options and new hope. Following recent approvals =
in the U.S. and Japan, it is our commitment to bring widespread access of i=
nnovative treatment options to a broader patient population living with mya=
sthenia gravis. And, with our two different medicines for gMG, each with a =
distinct mechanism of action, UCB offers the community a unique portfolio o=
f treatments that embodies our commitment to addressing the gMG community=
=E2=80=99s unmet needs.=E2=80=9D said Jean-Christophe Tellier, CEO, UCB. =
=E2=80=9CWe would like to take this time to extend our gratitude to the pat=
ients, care partners and investigators who participated in the MycarinG stu=
dy, and to our employees and collaborators for their dedication and support=
to the gMG community.=E2=80=9D=C2=A0
This announcement follows approval of rozanolixizumab and zilucoplan by the=
Japanese Ministry of Health, Labour and Welfare (MHLW) for treatment of gM=
G in adult patients (only for patients who inadequately respond to steroids=
or other immunosuppressants), and approval of rozanolixizumab by the U.S. =
Food and Drug Administration (FDA) for the treatment of gMG in adult patien=
ts who are anti-AChR or anti-MuSK antibody positive.^7,5 Orphan designation=
was granted by the European Commission in 2020 to rozanolixizumab for the =
treatment of myasthenia gravis and successfully maintained after having rec=
eived the positive CHMP Opinion.^13=C2=A0
The CHMP=E2=80=99s positive opinion for rozanolixizumab is now being review=
ed by the European Commission, which grants centralized marketing authoriza=
tions for medicinal products in the EU. Feedback from the European Commissi=
on is anticipated during Q1 2024.=C2=A0
About rozanolixizumab
Rozanolixizumab 140 mg/ml solution for injection is a subcutaneously admini=
stered, humanized monoclonal antibody that specifically binds, with high af=
finity, to human neonatal Fc receptor (FcRn). It has been designed to block=
the interaction of FcRn and Immunoglobulin G (IgG), accelerating the catab=
olism of antibodies and reducing the concentration of pathogenic IgG autoan=
tibodies.^2
In June 2023, rozanolixizumab-noli was approved by the FDA, for the treatme=
nt of gMG in adult patients who are anti-acetylcholine receptor (AChR) or a=
nti-muscle-specific tyrosine kinase (MuSK) antibody-positive, having been g=
ranted Priority Review for its Biologic License Application (BLA).^5
In September 2023, rozanolixizumab was granted approval by the Japanese Min=
istry of Health, Labour and Welfare (MHLW) for the treatment of generalized=
myasthenia gravis (gMG) in adult patients (only for patients who inadequat=
ely respond to steroids or other immunosuppressants).^7
Rozanolixizumab is currently under review by the Center of Drug Evaluation =
of the China National Medical Products Administration, the Australian Thera=
peutic Goods Administration (TGA), Health Canada and Switzerland (Swissmedi=
c) for the treatment of adults with gMG. Responses from regulatory agencies=
to these submissions are expected during H2 2023 and H1 2024.=C2=A0
About zilucoplan
Zilucoplan is a once-daily SC, self-administered peptide inhibitor of compl=
ement component 5 (C5 inhibitor). As the only once-daily generalized myasth=
enia gravis (gMG) target therapy for self-administration by adult patients =
with anti-acetylcholine receptor (AChR) antibody-positive gMG, zilucoplan i=
nhibits complement-mediated damage to the neuromuscular junction through it=
s targeted mechanism of action.^8
In October 2023, zilucoplan was approved by the U.S. Food and Drug Administ=
ration (FDA) for the treatment of gMG in adult patients who are anti-acetyl=
choline receptor (AchR) antibody-positive.^6=C2=A0
In September 2023, the Committee for Medicinal Products for Human Use (CHMP=
) of the European Medicines Agency (EMA) issued a positive opinion recommen=
ding granting marketing authorization for zilucoplan in the European Union =
(EU) as an add-on to standard therapy for the treatment of gMG in adult pat=
ients who are anti-AChR antibody-positive.^4 A final decision on approval i=
n the EU is expected before the end of the year, in line with the EMA=E2=80=
=99s standard review timeline.=C2=A0
Also in September 2023, the Japanese Ministry of Health, Labour and Welfare=
(MHLW) approved zilucoplan for the treatment of gMG in adult patients (onl=
y for patients who inadequately respond to steroids or other immunosuppress=
ants).^7
Zilucoplan is currently under review by the Australian Therapeutic Goods Ad=
ministration (TGA) and Health Canada for the treatment of adults with gMG. =
Responses from regulatory agencies to these submissions are expected during=
H2 2023 and H1 2024.
Orphan designation was granted by the FDA in 2019 to zilucoplan for the tre=
atment of myasthenia gravis.^14
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.^11 People living with gMG can experienc=
e a variety of symptoms, including severe muscular weakness that can result=
in double vision, drooping eyelids, difficulty with swallowing, chewing an=
d talking, as well as life-threatening weakness of the muscles of respirati=
on.^10,15
In gMG, pathogenic autoantibodies can impair synaptic transmission at the n=
euromuscular junction (NMJ) by targeting specific proteins on the post-syna=
ptic membrane.^16 This disrupts the ability of the nerves to stimulate the =
skeletal muscle and results in a weaker contraction. gMG can occur in any r=
ace, gender or age.^10,15
About the MycarinG study^2
The MycarinG study (NCT03971422) is a multi-center, Phase 3, randomized, do=
uble-blind, placebo-controlled study evaluating the efficacy and safety of =
rozanolixizumab in adult patients with gMG, with an open-label extension.=
=C2=A0
The primary endpoint for the MycarinG study is change in the Myasthenia Gra=
vis-Activities of Daily Living (MG-ADL) score, an eight-item patient-report=
ed scale developed to assess MG symptoms and their effects on daily activit=
ies. Additional endpoints include changes in the Myasthenia Gravis composit=
e (MG-C) score, the Quantitative MG (QMG) score, patient-reported outcomes =
at Day 43 and adverse events (AEs). The majority of patients taking part in=
the MycarinG study opted to enroll in any future extensions to this clinic=
al trial. As a result, UCB is exploring the potential for further extension=
studies into this treatment.
For more information about the trial, visit https://clinicaltrials.gov/ct2/=
show/NCT03971422.=C2=A0
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
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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cial decisions or governmental investigations, safety, quality, data integr=
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stems.=C2=A0
Given these uncertainties, you should not place undue reliance on any of su=
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UCB is providing this information, including forward-looking statements, on=
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References:
1. EMA CHMP Confirmation. Data on file, UCB November 2023.
2. Bril V. Efficacy and safety of rozanolixizumab in patients with generali=
sed myasthenia gravis: a randomised, double-blind, placebo-controlled, adap=
tive Phase 3 study MycarinG study. Lancet Neurol. 2023;22(5):383-94
3. Wolfe Gl, et al. Myasthenia gravis activities of daily living profile. N=
eurology. 1992;52(7):1487-9
4. CHMP Positive Opinion: Zilbrysq https://www.ema.europa.eu/en/medicines/h=
uman/summaries-opinion/zilbrysq. Date accessed November 2023
5. RYSTIGGO^=C2=AE U.S. Prescribing Information
6. ZILBRYSQ^=C2=AE U.S. Prescribing Information.
7. Data on file: Japan MHLW, 25 September 2023.
8. Howard JF Jr, et al. Safety and efficacy of zilucoplan in patients with =
generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-=
controlled, phase 3 study. Lancet Neurol. 2023;22(5):395-406.=C2=A0
9. Regnault A, et al. Measuring Overall Severity of Myasthenia Gravis (MG):=
Evidence for the Added Value of the MG Symptoms PRO. Neurol Ther. 2023; 12=
:1573=E2=80=931590.
10. National Institute of Neurological Disorders and Stroke. 2022. Myasthen=
ia Gravis Fact Sheet. https://www.ninds.nih.gov/health-information/disorder=
s/myasthenia-gravis?search-term=3Dmyasthenia%20gravis%20fact%20sheet. Date =
accessed November 2023
11. Punga AR, et al. Epidemiology, diagnostics, and biomarkers of autoimmun=
e neuromuscular junction disorders. Lancet Neurol. 2022;21(2):176-88.
12. Howard JF. Myasthenia gravis: The role of complement at the neuromuscul=
ar junction. Ann N Y Acad Sci. 2018;1412:113-128.
13. European Medicines Agency. 2020. EU/3/20/2272: Orphan designation for t=
he treatment of myasthenia gravis. https://www.ema.europa.eu/en/medicines/h=
uman/orphan-designations/eu3202272. Date accessed November 2023
14. US Food and Drug Administration. https://www.accessdata.fda.gov/scripts=
/opdlisting/oopd/detailedIndex.cfm?cfgridkey=3D699319. Date accessed Novemb=
er 2023.
15. Myasthenia Gravis Foundation of America. MG Quick Facts. https://myasth=
enia.org/MG-Education/MG-Quick-Facts. Date accessed November 2023
16. Juel VC, Massey JM. Myasthenia gravis. Orphanet J Rare Dis. 2007;2:44.
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