https://mb.cision.com/Public/18595/3855610/b87e61056f6b1e26_800x800ar.png
** UCB announces U.S. FDA approval of ZILBRYSQ^=C2=AE (zilucoplan) for the =
treatment of adults with generalized myasthenia gravis
------------------------------------------------------------
=C2=B7 FDA approval of ZILBRYSQ^=C2=AE (zilucoplan) has been granted for th=
e treatment of generalized myasthenia gravis (gMG) in adult patients who ar=
e anti-acetylcholine receptor (AChR) antibody-positive^1
=C2=B7 Zilucoplan is the first once-daily subcutaneous, targeted C5 complem=
ent inhibitor for gMG. It is the only once-daily gMG-target therapy for sel=
f-administration=C2=A0
=C2=B7 U.S. FDA approval of zilucoplan is built on the pivotal Phase 3 RAIS=
E study in gMG^2, which demonstrated that treatment with zilucoplan resulte=
d in statistically significant improvements in MG-specific efficacy outcome=
s compared to placebo
=C2=B7 With the FDA approval of zilucoplan, and with RYSTIGGO^=C2=AE (rozan=
olixizumab-noli) already FDA approved and launched in the U.S^3, UCB is the=
first organization to offer the U.S. gMG community the opportunity to bene=
fit from a choice of two new targeted therapies from a single company
=C2=B7 UCB=E2=80=99s portfolio of two different medicines for gMG, each wit=
h a distinct mechanism of action, offers physicians new and additional choi=
ces, embodying our commitment to addressing the gMG community=E2=80=99s unm=
et needs=C2=A0
=C2=A0
Brussels (Belgium) 17 October 2023=C2=A0=E2=80=93 UCB (Euronext Brussels: U=
CB), a global biopharmaceutical company, today announced that ZILBRYSQ^=C2=
=AE (zilucoplan) has been approved by the U.S. Food and Drug Administration=
(FDA) for the treatment of generalized myasthenia gravis (gMG) in adult pa=
tients who are anti-acetylcholine receptor (AChR) antibody-positive.^1=C2=
=A0
Zilucoplan is the first once-daily subcutaneous (SC), targeted peptide inhi=
bitor of complement component 5 (C5 inhibitor)2. It is the only once-daily =
gMG target therapy for self-administration by adult patients with anti-AChR=
antibody-positive gMG.=C2=A0
As a complement C5 inhibitor, zilucoplan inhibits complement-mediated damag=
e to the neuromuscular junction through its targeted mechanism of action.^2=
Benefits of self-administered treatment compared with=C2=A0intravenously a=
dministered treatments can include reduced traveling time to and from hospi=
tals, decreased interference with work obligations, and increased independe=
nce. Unlike monoclonal antibody C5 inhibitors, as a peptide, zilucoplan can=
be used concomitantly with intravenous immunoglobulin and plasma exchange,=
without the need for supplemental dosing.^2=C2=A0
The FDA approval of zilucoplan^1 is supported by safety and efficacy data f=
rom the RAISE study (NCT04115293), published in The Lancet Neurology in May=
2023.2 The RAISE study was a multi-center, phase 3, randomized, double-bli=
nd, placebo-controlled study to assess the efficacy, safety profile, and to=
lerability of zilucoplan in adult patients with anti-acetylcholine receptor=
(AChR) antibody-positive gMG. Patients were randomized in a 1:1 ratio to r=
eceive daily subcutaneous (SC) injections of 0.3 mg/kg zilucoplan or placeb=
o for 12 weeks. The study demonstrated that zilucoplan delivered rapid, con=
sistent, and statistically significant benefits in different patient-and-cl=
inician reported outcomes at week 12 in a broad population of adult patient=
s with mild-to-severe anti-AChR-antibody positive gMG. The most common adve=
rse reactions (=E2=89=A510%) in patients with gMG were injection site react=
ions, upper respiratory tract infection, and diarrhea.
=E2=80=9CFor people with gMG, the unpredictable nature of the severity and =
frequency of symptoms can be debilitating and can have a substantial impact=
on many aspects of their day-to-day lives. In addition to muscle weakness,=
people living with gMG experience fatigue, affecting their overall quality=
of life,=E2=80=9D said James F. Howard, MD, Distinguished Professor of Neu=
romuscular Disease, Professor of Neurology, Medicine and Allied Health, The=
University of North Carolina at Chapel Hill School of Medicine and lead in=
vestigator in the RAISE trial. =E2=80=9CZilucoplan demonstrated rapid impro=
vements in gMG symptoms at Week 12, with differences seen as early as one w=
eek, and provides a new treatment option for a broad population of AChR ant=
ibody-positive gMG patients. Zilucoplan is designed for continued daily use=
.=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 Several factors are understood to be drivers of gMG disease pathology,=
including the complement cascade, immune cells and pathogenic Immunoglobul=
in G (IgG) autoantibodies.^7
In anti-AChR antibody-positive gMG, pathogenic AChR autoantibodies (IgG1 an=
d IgG3) initiate the classical complement pathway, leading to the cleavage =
of C5 and the MAC (membrane attack complex) formation, damage to the NMJ, l=
oss of AChRs and subsequent impaired synaptic transmission.^6,8,9 Preventin=
g MAC formation reduces damage to the post-synaptic membrane, reduces disru=
ption of ionic channel conductance and helps to preserve neuromuscular tran=
smission.^8 MG has a global prevalence of 100=E2=80=93350 cases per every 1=
million people.^5
=E2=80=9CThis is an important development for the community because, with m=
ore FDA-approved treatments for generalized myasthenia gravis, physicians h=
ave additional tools to treat this disease in individualized ways that are =
the right fit for each individual patient,=E2=80=9D said Samantha Masterson=
, President and Chief Executive Officer of the Myasthenia Gravis Foundation=
of America. =E2=80=9CWe are so grateful to UCB for being part of the myast=
henia gravis community and their continued commitment to finding solutions =
for people living with this chronic, autoimmune, neuromuscular disease.=E2=
=80=9D
With the approval of zilucoplan, alongside the company=E2=80=99s neonatal F=
c receptor (FcRn) blocker RYSTIGGO^=C2=AE (rozanolixizumab-noli), which was=
approved earlier this year by the FDA under Priority Review designation fo=
r the treatment of generalized myasthenia gravis (gMG) in adult patients wh=
o are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine k=
inase (MuSK) antibody-positive, UCB's portfolio provides healthcare profess=
ionals the option of addressing either complement activation or pathogenic =
auto-antibodies for appropriate patients.^3,10
=E2=80=9CWith the FDA approval for zilucoplan, we are very proud and excite=
d to expand our support to the gMG community. Following the FDA approval an=
d strong momentum with our FcRn inhibitor rozanolixizumab-noli and with our=
tailored patient support services and commitment to widespread access, I a=
m confident that UCB is the only company with a portfolio of two targeted t=
herapies with different mechanisms of action and the experience to deliver =
truly individualized transformational patient value to people living with t=
his often debilitating rare disease=E2=80=9D said Jean-Christophe Tellier, =
CEO, UCB. =E2=80=9CWe would like to extend our thanks to the patients, care=
partners, and investigators who participated in the RAISE study, and to ou=
r employees and collaborators, whose dedication and commitment to the gMG c=
ommunity made this important milestone possible.=E2=80=9D
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. The M=
G-ADL is an eight-item patient-reported outcome measure assessing MG sympto=
ms and functional activities related to activities of daily living.^11 Thes=
e include activities such as breathing, talking, swallowing, and being able=
to rise from a chair.^12 Each of the items is scored, from 0 (normal) to 3=
(most severe), providing a total MG-ADL score ranging from 0 to 24, where =
higher scores indicate greater severity of symptoms.^2=C2=A0
The efficacy of zilucoplan was also measured, as a secondary endpoint, usin=
g the Quantitative Myasthenia Gravis (QMG) total score which is a 13-item c=
ategorical grading system that assesses muscle weakness. Each item is asses=
sed on a 4-point scale where a score of 0 represents no weakness and a scor=
e of 3 represents severe weakness. A total possible score ranges from 0 to =
39, where higher scores indicate more severe impairment.^11=C2=A0
At week 12, treatment with zilucoplan demonstrated a statistically signific=
ant improvement from baseline compared to placebo for MG-ADL total score an=
d QMG total score. Other secondary endpoints included the proportion of pat=
ients with improvements of at least 3 and 5 points in the MG-ADL total scor=
e and QMG total score, respectively, at week 12 without rescue therapy.^2
=E2=80=9CUntil now, people living with gMG have only had access to C5 thera=
py intravenously, which can be inconvenient and time consuming. Now, with t=
he option of zilucoplan, a self-administered once-daily, subcutaneous targe=
ted complement C5 inhibitor, we hope a broad population of mild-to-severe a=
dult patients with AChR-antibody-positive gMG will be able to have greater =
independence=E2=80=9D said Iris Loew-Friedrich, Executive Vice-President an=
d Chief Medical Officer at UCB. =E2=80=9CAlongside our FcRn blocker rozanol=
ixizumab-noli, which was approved and launched in the U.S. earlier this sum=
mer, our unique portfolio will support patients and healthcare professional=
s to tailor choice of treatment according to their individual needs.=E2=80=
=9D=C2=A0
About zilucoplan
Zilucoplan is a once-daily, SC, self-administered peptide inhibitor of comp=
lement component 5 (C5 inhibitor). As the only once-daily gMG target therap=
y for self-administration by adult patients with anti-AChR antibody-positiv=
e gMG, zilucoplan inhibits complement-mediated damage to the neuromuscular =
junction through its targeted mechanism of action.^2=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 generalized myas=
thenia gravis (gMG) in adult patients who are anti acetylcholine receptor (=
AChR) antibody-positive.^13 A final decision on approval in the EU is expec=
ted before the end of the year, in line with the EMA=E2=80=99s standard rev=
iew 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 who =
inadequately respond to steroids or other immunosuppressants.^14=C2=A0
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 betwee=
n H2 2023 and H2 2024.=C2=A0
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.5 People living with gMG can experience =
a variety of symptoms, including severe muscular weakness that can result i=
n double vision, drooping eyelids, difficulty with swallowing, chewing and =
talking, as well as life-threatening weakness of the muscles of respiration=
.^4,16
In MG, pathogenic autoantibodies can impair synaptic transmission at the ne=
uromuscular junction (NMJ) by targeting specific proteins on the post-synap=
tic membrane.^17 This disrupts the ability of the nerves to stimulate the m=
uscle and results in a weaker contraction. gMG can occur in any race, gende=
r or age.^4,16
About the RAISE study^2
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.=C2=A0
Secondary endpoints included change from baseline in the Quantitative Myast=
henia Gravis (QMG) score, the Myasthenia Gravis Composite (MGC) and the Mya=
sthenia Gravis Quality of Life 15 revised (MG-QoL15r) score from baseline t=
o Week 12. Other secondary efficacy outcomes, time to first receipt of resc=
ue therapy over 12 weeks, the proportion of patients with minimal symptom e=
xpression (MSE) (defined as MG-ADL of 0 or 1 without rescue therapy), the p=
roportion of patients with a =E2=89=A53-point reduction in MG-ADL without r=
escue therapy and the proportion of patients with a =E2=89=A55-point reduct=
ion in QMG without rescue therapy, all measured at Week 12.=C2=A0
Safety was assessed by the incidence of treatment-emergent adverse events (=
TEAEs).=C2=A0
Patients who completed the RAISE trial had the possibility to enter the ope=
n-label extension study, RAISE-XT (NCT04225871).^2=C2=A0
For more information about the trial, visit https://clinicaltrials.gov/ct2/=
show/NCT04115293.=C2=A0
About rozanolixizumab-noli
In addition to zilucoplan, UCB=E2=80=99s gMG portfolio includes the FDA-app=
roved medicine =C2=A0RYSTIGGO=C2=AE (rozanolixizumab-noli), a subcutaneousl=
y infused monoclonal antibody targeting the neonatal Fc receptor (FcRn).^9=
=C2=A0
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).^3,10=C2=
=A0
In September 2023, rozanolixizumab was approved by the Japanese Ministry of=
Health, Labour and Welfare (MHLW) for the treatment of gMG in adult patien=
ts who inadequately respond to steroids or other immunosuppressants.^14=C2=
=A0
Rozanolixizumab is currently under review by the European Medicines Agency =
(EMA), the Australian Therapeutic Goods Administration (TGA) and Health Can=
ada for the treatment of adults with gMG. Responses from regulatory agencie=
s to these submissions are expected during H2 2023 and H1 2024.=C2=A0
Important Safety Information for ZILBRYSQ^=C2=AE
IMPORTANT SAFETY INFORMATION INCLUDING BOXED WARNING
What is the most important information I should know about ZILBRYSQ?
ZILBRYSQ is a medicine that affects part of your immune system. =C2=A0ZILBR=
YSQ may lower the ability of your immune system to fight certain infections=
.
=C2=B7 ZILBRYSQ increases your chance of getting serious and life-threateni=
ng meningococcal infections. Meningococcal infections may become life-threa=
tening or fatal if not recognized and treated early.
=C2=B7 You must complete or update two types of meningococcal vaccines (f=
or both serogroup B infections and serogroup A, C, W, and Y infections) at =
least 2 weeks before your first dose of ZILBRYSQ if you have not already ha=
d these vaccines.
=C2=B7 If your healthcare provider decided that urgent treatment with ZIL=
BRYSQ is needed, you should receive meningococcal vaccination(s) as soon as=
possible.
=C2=B7 If you have not completed or updated vaccinations for meningococca=
l infections at least 2 weeks before your first ZILBRYSQ dose and ZILBRYSQ =
therapy must be started right away, you must also receive antibiotics.
=C2=B7 If you had a meningococcal vaccine in the past, you might need add=
itional vaccination before starting ZILBRYSQ. Your healthcare provider will=
decide if you need additional meningococcal vaccination.
=C2=B7 Meningococcal vaccines do not prevent all meningococcal infections=
. Call your healthcare provider or get emergency medical care right away if=
you get any of these signs and symptoms of a meningococcal infection:
=EF=82=A7=C2=A0=C2=A0 =C2=A0headache with nausea or vomiting=C2=A0=C2=A0 =
=C2=A0=EF=82=A7=C2=A0=C2=A0 =C2=A0confusion
=EF=82=A7=C2=A0=C2=A0 =C2=A0headache and fever=C2=A0=C2=A0 =C2=A0=EF=82=A7=
=C2=A0=C2=A0 =C2=A0muscle aches with flu-like symptoms
=EF=82=A7=C2=A0=C2=A0 =C2=A0headache with a stiff neck or stiff back=C2=A0=
=C2=A0 =C2=A0=EF=82=A7=C2=A0=C2=A0 =C2=A0eyes sensitive to light
=EF=82=A7=C2=A0=C2=A0 =C2=A0fever=C2=A0=C2=A0 =C2=A0
=EF=82=A7=C2=A0=C2=A0 =C2=A0fever and a rash=C2=A0=C2=A0 =C2=A0
Your healthcare provider will give you a Patient Safety Card about the risk=
of meningococcal infection. Carry it with you at all times during treatmen=
t and for 2 months after your last ZILBRYSQ dose. Your risk of meningococca=
l infection may continue for several weeks after your last dose of ZILBRYSQ=
. It is important to show this card to any healthcare provider who treats y=
ou. This will help them diagnose and treat you quickly.
ZILBRYSQ is only available through a program called the ZILBRYSQ REMS. =C2=
=A0Before you can receive ZILBRYSQ, your healthcare provider must:
=C2=B7 enroll in the ZILBRYSQ REMS.
=C2=B7 counsel you about the risk of meningococcal infection.
=C2=B7 give you the Patient Guide, including information about the signs an=
d symptoms of meningococcal infection.
=C2=B7 give you a Patient Safety Card about your risk of meningococcal infe=
ction, as discussed above.
=C2=B7 make sure that you are vaccinated with two types of meningococcal va=
ccines and, if needed, get revaccinated with the meningococcal vaccines. As=
k your healthcare provider if you are not sure if you need to be revaccinat=
ed.
ZILBRYSQ may also increase the risk of other types of serious infections.=
=C2=A0
=C2=B7 ZILBRYSQ may increase your chance of getting Streptococcus pneumonia=
e and Haemophilus influenzae type b. Your healthcare provider will tell you=
if you should receive the Streptococcus pneumoniae and Haemophilus influen=
zae type b vaccinations.
=C2=B7 Certain people may have an increased risk of gonorrhea infection. Ta=
lk to your healthcare provider about whether you are at risk for gonorrhea =
infection, about gonorrhea prevention, and about regular testing.=C2=A0
Call your healthcare provider right away if you have new signs or symptoms =
of infection.=C2=A0
Who should not use ZILBRYSQ?=C2=A0
Do not use ZILBRYSQ if you have a Neisseria meningitidis infection.
Before you use ZILBRYSQ, tell your healthcare provider about all of your me=
dical conditions, including if you:
=C2=B7 have an infection or fever.
=C2=B7 are pregnant or plan to become pregnant. =C2=A0It is not known if ZI=
LBRYSQ will harm your unborn baby.
=C2=B7 are breastfeeding or plan to breastfeed. It is not known if ZILRYSQ =
passes into your breast milk. Talk to your healthcare provider about the be=
st way to feed your baby if you use ZILBRYSQ.
Tell your healthcare provider about all the medicines you take, including p=
rescription and over-the-counter medicines, vitamins, and herbal supplement=
s.
What are the possible side effects of ZILBRYSQ?
ZILBRYSQ may cause serious side effects, including:
=C2=B7 See =E2=80=9CWhat is the most important information I should know ab=
out ZILBRYSQ?=E2=80=9D
=C2=B7 Inflammation of the pancreas (pancreatitis) and other pancreatic pro=
blems. Pancreatitis and pancreatic cysts have happened in people who use ZI=
LBRYSQ. Your healthcare provider will do blood tests to check your pancreas=
before you start treatment with ZILBRYSQ.
=C2=B7 Call your healthcare provider right away if you have pain in your =
stomach area (abdomen) that will not go away. Your healthcare provider will=
tell you if you should stop using ZILBRYSQ. The pain may be severe or felt=
going from your abdomen to your back. The pain may happen with or without =
vomiting. These may be symptoms of pancreatitis.
The most common side effects of ZILBRYSQ include:
=C2=B7 injection site reactions.
=C2=B7 upper respiratory tract infections.
=C2=B7 diarrhea.
Tell your healthcare provider about any side effect that bothers you or tha=
t does not go away.
These are not all the possible side effects of ZILBRYSQ. For more informati=
on, ask your healthcare provider or pharmacist. Call your doctor for medica=
l advice about side effects. You may report side effects to FDA at www.fda.=
gov/medwatch (https://www.fda.gov/medwatch) or 1-800-FDA-1088. You may also=
report side effects to UCB, Inc. by calling 1-844-599-CARE [2273].
See the detailed Instructions for Use that comes with ZILBRYSQ for informat=
ion on how to prepare and inject a dose of ZILBRYSQ, and how to properly th=
row away (dispose of) used ZILBRYSQ prefilled syringes.
INDICATION
What is ZILBRYSQ?
=C2=B7 ZILBRYSQ is a prescription medicine used to treat adults with a dise=
ase called generalized myasthenia gravis (gMG) who are anti-acetylcholine r=
eceptor (AChR) antibody positive.
=C2=B7 It is not known if ZILBRYSQ is safe and effective in children.
Please see the full Prescribing Information and Medication Guide for ZILBRY=
SQ, including Boxed Warning regarding serious meningococcal infections. Ple=
ase see the Instructions for Use for the ZILBRYSQ Single-Dose Prefilled Syr=
inge. Talk to your healthcare provider about your condition or your treatme=
nt. For more information, go to www.ZILBRYSQ.com (http://www.zilbrysq.com/)=
or call 1-844-599-2273.
Important Safety Information for RYSTIGGO^=C2=AE=C2=A0
IMPORTANT SAFETY INFORMATION AND INDICATION
WHAT IS RYSTIGGO?
RYSTIGGO is a prescription medicine used to treat adults with a disease cal=
led generalized myasthenia gravis (gMG) who are acetylcholine receptor (ant=
i-AChR) antibody positive or muscle-specific tyrosine kinase (anti-MuSK) an=
tibody positive.^18
WHAT IS THE MOST IMPORTANT INFORMATION I SHOULD KNOW ABOUT RYSTIGGO (rozano=
lixizumab-noli)?
RYSTIGGO may cause serious side effects, including:
=C2=B7 Infection: RYSTIGGO may increase the risk of infection. In clinical =
studies, the most common infections were upper respiratory tract infections=
, COVID-19, urinary tract infections, and herpes simplex infections. Your h=
ealthcare provider should check you for infections before starting and duri=
ng treatment with RYSTIGGO. Tell your healthcare provider if you have any h=
istory of infections. Tell your healthcare provider right away if you have =
signs or symptoms of an infection during treatment with RYSTIGGO. Some of t=
he signs and symptoms may include fever, chills, frequent and/or painful ur=
ination, cough, runny nose, wheezing, shortness of breath, fatigue, sore th=
roat, excess phlegm, nasal discharge, back pain, and/or chest pain.^18
=C2=B7 Aseptic Meningitis: RYSTIGGO could cause aseptic meningitis. Tell yo=
ur healthcare provider right away if you develop any signs or symptoms of m=
eningitis during treatment with RYSTIGGO such as severe headache, neck stif=
fness, drowsiness, fever, sensitivity to light, painful eye movements, naus=
ea, and vomiting.^18
=C2=B7 Hypersensitivity Reactions: RYSTIGGO can cause swelling and rash. Yo=
ur healthcare provider should monitor you during and after treatment and di=
scontinue RYSTIGGO if needed. Tell your healthcare provider immediately abo=
ut any undesirable reactions you experience after administration.^17
Before taking RYSTIGGO, tell your healthcare provider about all of your med=
ical conditions, including if you:
=C2=B7 Have a history of infection or think you have an active infection.
=C2=B7 Have received or are scheduled to receive a vaccine (immunization). =
The use of vaccines during RYSTIGGO treatment has not been studied, and the=
safety with live or live-attenuated vaccines is unknown. Administration of=
live or live-attenuated vaccines is not recommended during treatment with =
RYSTIGGO. Completion of age-appropriate vaccines according to vaccination g=
uidelines before starting a new treatment cycle with RYSTIGGO is recommende=
d.
=C2=B7 Are pregnant or plan to become pregnant or are breastfeeding or plan=
to breastfeed.
Tell your healthcare provider about all the medicines you take, including p=
rescription and over-the-counter medicines, vitamins, and herbal supplement=
s.
WHAT ARE THE POSSIBLE SIDE EFFECTS OF RYSTIGGO?
RYSTIGGO may cause serious side effects, including^18:
=C2=B7 See "What is the most important information I should know about RYST=
IGGO?"
The most common side effects of RYSTIGGO include^18:
=C2=B7 headache
=C2=B7 infections
=C2=B7 diarrhea
=C2=B7 fever
=C2=B7 hypersensitivity reactions
=C2=B7 nausea
These are not all the possible side effects of RYSTIGGO. For more informati=
on, ask your healthcare provider or pharmacist. Tell your healthcare provid=
er about any side effect that bothers you or that does not go away. Call yo=
ur healthcare provider for medical advice about side effects. You are encou=
raged to report negative side effects of prescription drugs to the FDA. Vis=
it www.fda.gov/medwatch (https://www.fda.gov/medwatch) or call 1-800-FDA-10=
88. You may also report side effects to UCB, Inc. by calling 1-844-599-CARE=
[2273].
Please see the full Prescribing Information (https://www.ucb-usa.com/RYSTIG=
GO-prescribing-information.pdf) and talk to your healthcare provider about =
your condition or your treatment.
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
U.S. Rare Disease Communications
Daphne Teo
Daphne.teo@ucb.com
T +1-770-880-7655
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
This press release may contain forward-looking statements including, withou=
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=
inue=E2=80=9D and similar expressions. These forward-looking statements are=
based on current plans, estimates and beliefs of management. All statement=
s, other than statements of historical facts, are statements that could be =
deemed forward-looking statements, including estimates of revenues, operati=
ng margins, capital expenditures, cash, other financial information, expect=
ed legal, arbitration, political, regulatory or clinical results or practic=
es and other such estimates and results. By their nature, such forward-look=
ing statements are not guarantees of future performance and are subject to =
known and unknown risks, uncertainties and assumptions which might cause th=
e actual results, financial condition, performance or achievements of UCB, =
or industry results, to differ materially from those that may be expressed =
or implied by such forward-looking statements contained in this press relea=
se. Important factors that could result in such differences include: the gl=
obal spread and impact of COVID-19, changes in general economic, business a=
nd competitive conditions, the inability to obtain necessary regulatory app=
rovals or to obtain them on acceptable terms or within expected timing, cos=
ts associated with research and development, changes in the prospects for p=
roducts in the pipeline or under development by UCB, effects of future judi=
cial decisions or governmental investigations, safety, quality, data integr=
ity or manufacturing issues; potential or actual data security and data pri=
vacy breaches, or disruptions of our information technology systems, produc=
t liability claims, challenges to patent protection for products or product=
candidates, competition from other products including biosimilars, changes=
in laws or regulations, exchange rate fluctuations, changes or uncertainti=
es in tax laws or the administration of such laws, and hiring and retention=
of its employees. There is no guarantee that new product candidates will b=
e discovered or identified in the pipeline, will progress to product approv=
al or that new indications for existing products will be developed and appr=
oved. Movement from concept to commercial product is uncertain; preclinical=
results do not guarantee safety and efficacy of product candidates in huma=
ns. So far, the complexity of the human body cannot be reproduced in comput=
er models, cell culture systems or animal models. The length of the timing =
to complete clinical trials and to get regulatory approval for product mark=
eting has varied in the past and UCB expects similar unpredictability going=
forward. Products or potential products, which are the subject of partners=
hips, joint ventures or licensing collaborations may be subject to differen=
ces disputes between the partners or may prove to be not as safe, effective=
or commercially successful as UCB may have believed at the start of such p=
artnership. UCB=E2=80=99s efforts to acquire other products or companies an=
d to integrate the operations of such acquired companies may not be as succ=
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=
ts products and/or devices after they are marketed. The discovery of signif=
icant problems with a product similar to one of UCB=E2=80=99s products that=
implicate an entire class of products may have a material adverse effect o=
n sales of the entire class of affected products. Moreover, sales may be im=
pacted by international and domestic trends toward managed care and health =
care cost containment, including pricing pressure, political and public scr=
utiny, customer and prescriber patterns or practices, and the reimbursement=
policies imposed by third-party payers as well as legislation affecting bi=
opharmaceutical pricing and reimbursement activities and outcomes. Finally,=
a breakdown, cyberattack or information security breach could compromise t=
he confidentiality, integrity and availability of UCB=E2=80=99s data and sy=
stems.=C2=A0
Given these uncertainties, you should not place undue reliance on any of su=
ch forward-looking statements. There can be no guarantee that the investiga=
tional or approved products described in this press release will be submitt=
ed or approved for sale or for any additional indications or labelling in a=
ny market, or at any particular time, nor can there be any guarantee that s=
uch products will be or will continue to be commercially successful in the =
future.
UCB is providing this information, including forward-looking statements, on=
ly as of the date of this press release and it does not reflect any potenti=
al impact from the evolving COVID-19 pandemic, unless indicated otherwise. =
UCB is following the worldwide developments diligently to assess the financ=
ial significance of this pandemic to UCB. UCB expressly disclaims any duty =
to update any information contained in this press release, either to confir=
m the actual results or to report or reflect any change in its forward-look=
ing statements with regard thereto or any change in events, conditions or c=
ircumstances on which any such statement is based, unless such statement is=
required pursuant to applicable laws and regulations.=C2=A0
Additionally, information contained in this document shall not constitute a=
n offer to sell or the solicitation of an offer to buy any securities, nor =
shall there be any offer, solicitation or sale of securities in any jurisdi=
ction in which such offer, solicitation or sale would be unlawful prior to =
the registration or qualification under the securities laws of such jurisdi=
ction.=C2=A0
References:
1. ZILBRYSQ^=C2=AE U.S. Prescribing Information.
2. 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
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 October 2023.
4. National Institute of Neurological Disorders and Stroke. 2022. Myastheni=
a Gravis Fact Sheet. https://www.ninds.nih.gov/myasthenia-gravis-fact-sheet=
. Accessed October 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 Jr. Myasthenia gravis: The role of complement at the neuromusc=
ular junction. Ann N Y Acad Sci. 2018;1412(1):113=E2=80=92128.=C2=A0
7. Phillips WD, Vincent A. Pathogenesis of myasthenia gravis: Update on dis=
ease types, models, and mechanisms. F1000Res. 2016;5:F1000 Faculty Rev-1513=
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8. Mantegazza R, et al. Complement inhibition for the treatment of myasthen=
ia gravis. Immunotargets Ther. 2020;9:317=E2=80=93331.
9. Howard JF Jr, et al. Zilucoplan: An Investigational Complement C5 Inhibi=
tor for the Treatment of Acetylcholine Receptor Autoantibody-Positive Gener=
alized Myasthenia Gravis. Expert Opin Investig Drugs. 2021 May;30(5):483-49=
3.=C2=A0
10. Bril V, et al. Safety and efficacy of rozanolixizumab in patients with =
generalised myasthenia gravis (MycarinG): a randomised, double-blind, place=
bo-controlled, adaptive phase 3 study. Lancet Neurol. 2023;22(5):383-94.
11. National Library of Medicine (NIH) Clinical Review Report: Eculizumab (=
Soliris): Alexion Pharma Canada Corporation: Indication: Adult patients wit=
h generalized myasthenia gravis [Internet]. Appendix 5. https://www.ncbi.nl=
m.nih.gov/books/NBK567509/#:~:text=3DGo%20to%3A-,Myasthenia%20Gravis%20Acti=
vities%20of%20Daily%20Living%20Scale%20(MG%2DADL),to%20activities%20of%20da=
ily%20living. Accessed October 2023.
12. Wolfe Gl, et al. Myasthenia gravis activities of daily living profile. =
Neurology. 192;52(7):1487-9
13. CHMP Positive Opinion: Zilbrysq https://www.ema.europa.eu/en/medicines/=
human/summaries-opinion/zilbrysq Accessed October 2023.
14. UCB Data on file: Japan MHLW, 25 September 2023
15. US Food and Drug Administration. https://www.accessdata.fda.gov/scripts=
/opdlisting/oopd/detailedIndex.cfm?cfgridkey=3D699319. Accessed October 202=
3.
16. Myasthenia Gravis Foundation of America. MG Quick Facts. https://myasth=
enia.org/MG-Education/MG-Quick-Facts. Accessed October 2023.
17. Juel VC, Massey JM. Myasthenia gravis. Orphanet J Rare Dis. 2007;2:44.
18. RYSTIGGO^=C2=AE U.S. Prescribing Information.
GenericFile
UCB PR zilucoplan FDA Oct 18 2023 ENG (https://mb.cision.com/Public/18595/3=
855610/8743219b260535d3.pdf)
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