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** UCB announces rozanolixizumab BLA for the treatment of generalized myast=
henia gravis filed with U.S. FDA and designated for Priority Review
------------------------------------------------------------
=C2=B7 Biologic License Application (BLA) designated Priority Review by FDA=
and seeks approval for rozanolixizumab for the treatment of adults with ge=
neralized myasthenia gravis (gMG) who are anti-acetycholine receptor (AChR)=
or anti-muscle-specific tyrosine kinase (MuSK) antibody positive=C2=A0
=C2=B7 Rozanolixizumab FDA Priority Review follows recent European Medicine=
s Agency (EMA) validation of the Marketing Authorization Application (MAA) =
for rozanolixizumab in adults with gMG=C2=A0
=C2=B7 FDA and EMA submissions based on pivotal Phase 3 MycarinG study in g=
MG which demonstrated treatment with rozanolixizumab resulted in clinically=
meaningful and statistically significant improvements in MG specific outco=
mes
=C2=B7 UCB expects to receive feedback from the agencies in Q2 of 2023=C2=
=A0
Brussels (Belgium) 6 Jan 2023 =E2=80=93 7:00AM (CET) =E2=80=93 UCB, a globa=
l biopharmaceutical company, today announced that the U.S. Food and Drug Ad=
ministration (FDA) has accepted the company=E2=80=99s filing to review a Bi=
ologic License Application (BLA) for its investigational treatment rozanoli=
xizumab, and that the Agency has granted Priority Review.^1 Rozanolixizumab=
is a subcutaneous (SC) monoclonal antibody targeting the neonatal Fc recep=
tor (FcRn) for the treatment of adults with generalized myasthenia gravis (=
gMG) who are anti-acetycholine receptor (AChR) or anti-muscle-specific tyro=
sine kinase (MuSK) antibody positive^2.=C2=A0
A FDA Priority Review designation is typically granted by the Agency to a m=
edicine which, if approved, could deliver significant improvements in the s=
afety or effectiveness of the treatment, diagnosis, or prevention of seriou=
s conditions when compared to standard applications3. Priority Review desig=
nation means the FDA=E2=80=99s goal is to take action on an application wit=
hin 6 months, compared to 10 months under standard review^3. In 2019, the U=
.S. FDA granted orphan drug designation to rozanolixizumab for the treatmen=
t of MG.^4
The safety and efficacy of rozanolixizumab have not been established and th=
ey are not currently approved for use in any indication by any regulatory a=
uthority worldwide. =C2=A0=C2=A0
The FDA Priority Review designation follows the recent European Medicines A=
gency (EMA) validation of the Marketing Authorization Application (MAA) for=
rozanolixizumab for the treatment of adults with AChR or MuSK antibody pos=
itive gMG who require treatment in addition to steroids or non-steroidal im=
munosuppressants. Validation confirms that the application is complete and =
the formal review process by the EMA=E2=80=99s Committee for Medicinal Prod=
ucts for Human Use (CHMP) can begin. Orphan designation was granted by the =
European Commission in April 2020 to rozanolixizumab for the treatment of m=
yasthenia gravis.^5
UCB expects to receive feedback from both the FDA and EMA during the second=
quarter of 2023.=C2=A0
=E2=80=9CPeople living with MG suffer from unpredictable, fluctuating, and =
debilitating symptoms that have a huge impact on their lives, and there is =
a clear need for additional targeted treatments. We are firmly committed to=
supporting the gMG community by providing solutions to help improve outcom=
es for patients and reduce the day-to-day burden of the disease,=E2=80=9D s=
aid Charl van Zyl, =C2=A0Executive Vice President Neurology Solutions & Hea=
d of EU/International Markets, UCB. =E2=80=9CThe FDA=E2=80=99s decision to =
assess rozanolixizumab via their priority review process, as well as the re=
cent filing of the MAA in Europe, brings us important steps further on our =
journey towards approvals for rozanolixizumab. We look forward to working w=
ith the FDA and EMA to help bring this new treatment option to patients.=E2=
=80=9D
gMG is a chronic and unpredictable auto-immune disease in which pathogenic =
autoantibodies can impair synaptic transmission at the neuromuscular juncti=
on by targeting specific proteins on the post-synaptic membrane. This disru=
pts the ability of the nerves to stimulate the muscle and results in a weak=
er contraction.^6 People living with gMG can experience a variety of sympto=
ms, including drooping eyelids, double vision, and difficulty in swallowing=
, chewing and talking, as well as severe muscle weakness that can result in=
life-threatening weakness of the muscles of respiration.^4,7=C2=A0In the U=
.S. the prevalence of MG is estimated at 14 to 20 per 100,000 population; a=
pproximately 36,000 to 60,000 cases.^4 In Europe, the prevalence is estimat=
ed at 10 per 100,000 population.^8=C2=A0
Data from the MycarinG study
The Priority Review BLA and the MAA are based on data from the pivotal Phas=
e 3 MycarinG study (NCT03971422), in which rozanolixizumab demonstrated sta=
tistically significant and clinically meaningful improvements in MG-specifi=
c outcomes in patients with AChR MuSK antibody positive MG. In the primary =
endpoint, rozanolixizumab significantly reduced MG-ADL from baseline to Day=
43. Rozanolixizumab showed an LS mean difference vs placebo (95% CI) of -2=
.59 points at the 7mg/kg dose and -2.62 points at the 10mg/kg dose.^9
Furthermore, a greater percentage of patients in the rozanolixizumab 7mg/kg=
and 10mg/kg arms than the placebo arm achieved a 2.0-point or greater impr=
ovement (p=EF=80=BC0.001) in MG-ADL, a 3.0-point or greater improvement in =
Quantitative Myasthenia Gravis (QMG) scores and a 3.0-point or greater impr=
ovement in Myasthenia Gravis Composite (MGC) scores^7, demonstrating clinic=
ally meaningful reductions in these assessments.
Rozanolixizumab demonstrated an acceptable safety and tolerability profile =
with similar occurrences of TEAEs between both doses. A higher proportion o=
f TEAEs occurred in the active treatment arms versus placebo (81.3% for 7 m=
g/kg, 82.6% for 10 mg/kg and 67.2% for placebo) and were comparable between=
the rozanolixizumab groups. The most frequently reported TEAEs were headac=
he, diarrhea, pyrexia, and nausea. A higher incidence of headache was repor=
ted in the rozanolixizumab groups versus placebo, with most cases mild to m=
oderate and severe cases generally managed with non-opioid analgesics.Treat=
ment discontinuation rates due to TEAEs were low.^7
In the MycarinG study, 200 patients were randomised 1:1:1 to receive weekly=
rozanolixizumab 7 mg/kg (N=3D66), 10 mg/kg (N=3D67) or placebo (N=3D67) fo=
r 6 weeks, which was followed by an 8-week observation period.^6
=E2=80=9CPatients living with MG may experience high disease and treatment =
burden resulting in a significant impact on their daily lives. If approved,=
rozanolixizumab has the potential to address unmet needs of gMG patients,=
=E2=80=9D said Iris Loew-Friedrich, Executive Vice-President and Chief Medi=
cal Officer at UCB. =E2=80=9CThrough rozanolixizumab and zilucoplan, we int=
end to bring two medicines with different mechanisms of action that have th=
e potential to provide targeted treatment options to patients. With our gMG=
pipeline, we hope to address both drivers of disease pathology and which a=
ccount for approximately 95% of people living with gMG. Priority Review Des=
ignation by the FDA for rozanolixizumab reflects the extent to which our sc=
ience speaks for itself in potentially addressing the significant unmet nee=
ds still faced by the gMG community.=E2=80=9D
UCB is currently investigating two potential therapies with different modes=
of action for the treatment of gMG. Alongside rozanolixizumab, a NDA for z=
ilucoplan =E2=80=93 a subcutaneous self-administered peptide inhibitor of c=
omplement component 5 (C5 inhibitor) has recently been filed with the U.S. =
FDA for the treatment of adults with AChR antibody positive gMG. Additional=
ly, zilucoplan received MAA validation from the EMA for the treatment of ad=
ults with AChR antibody positive gMG and who require treatment in addition =
to steroids or non-steroidal immunosuppressants.^10,11,12
For further information, contact UCB:=C2=A0
Brand Communications, Rare Diseases
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 =C2=A0=C2=A0
T +32.2.559.94.14=C2=A0
antje.witte@ucb.com
About Generalized Myasthenia Gravis (gMG)
Myasthenia gravis is a rare disease impacting more than 700,000 people worl=
dwide.^13 People living with gMG can experience a variety of symptoms, incl=
uding drooping eyelids, double vision, difficulty swallowing, chewing and t=
alking, as well as severe muscular weakness that can result in life threate=
ning weakness of the muscles of respiration.^4, 4
gMG is a chronic and unpredictable auto-immune disease in which pathogenic =
autoantibodies can impair synaptic transmission at the neuromuscular juncti=
on (NMJ) by targeting specific proteins on the post-synaptic membrane. This=
disrupts the ability of the nerves to stimulate the muscle and results in =
a weaker contraction.^4,14=C2=A0gMG can occur in any race, although previou=
s studies have shown that women are more often impacted than men.^15,16 Mos=
t patients with gMG have pathogenic IgG antibodies that disrupt the transmi=
ssion of nerve impulses to muscles in the NMJ and some activate the complem=
ent cascade.^1 Complement-mediated destruction via MAC formation is a key m=
echanism causing damage at the NMJ and is the key driver of disease in AChR=
antibody positive gMG gMG.
About the rozanolixizumab MycarinG study^17=C2=A0 =C2=A0
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. T=
he primary endpoint for the MycarinG study is change from baseline to day 4=
3 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score, an ei=
ght-item patient-reported scale developed to assess MG symptoms and their e=
ffects on daily activities. Additional endpoints include response rates, ch=
anges in the Myasthenia Gravis composite (MGC) score, the Quantitative MG (=
QMG) score, patient-reported outcomes and adverse events (AEs). The majorit=
y of patients taking part in the MycarinG study opted to enroll in the open=
label extensions to this clinical trial. As a result, UCB is exploring the=
potential for further extension studies into this treatment.=C2=A0
For more information about the trial, visit https://clinicaltrials.gov/ct2/=
show/NCT03971422.
About rozanolixizumab
Rozanolixizumab is a SC administered, humanized monoclonal antibody that sp=
ecifically binds, with high affinity, to human neonatal Fc receptor (FcRn).=
It has been designed to block the interaction of FcRn and Immunoglobulin G=
(IgG), accelerating the catabolism of antibodies and reducing the concentr=
ation of pathogenic IgG autoantibodies.^1,18
Rozanolixizumab is under clinical development with the aim of improving the=
lives of people with pathogenic IgG-autoantibody-driven autoimmune disease=
s. In 2019, the US FDA granted orphan drug designation to rozanolixizumab f=
or the treatment of myasthenia gravis.^2 Orphan designation was granted in =
2020 by the European Commission for rozanolixizumab to the treatment of mya=
sthenia gravis.^3
The safety and efficacy of rozanolixizumab have not been established and it=
is not approved for use in any indication by any regulatory authority worl=
dwide.
About zilucoplan
Zilucoplan is a once-daily SC, self-administered peptide inhibitor of compl=
ement component 5 (C5 inhibitor) under clinical development by UCB in gMG. =
As a C5 inhibitor, zilucoplan inhibits complement-mediated damage to the ne=
uromuscular junction through its targeted dual mechanism of action.^8 In 20=
19, the US FDA granted orphan drug designation to zilucoplan for the treatm=
ent of myasthenia gravis.^9 Orphan designation was granted in 2022 by the E=
uropean Commission to zilucoplan for the treatment of myasthenia gravis.^10
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 UCB in Rare Diseases=C2=A0
At UCB, we don=E2=80=99t just see patients or population sizes, we see peop=
le in need. Through decades of serving the neurology and immunology communi=
ties, we have improved lives with impactful medicines and by enhancing the =
social and emotional well-being of patients. As a continuation of our herit=
age, we are now expanding our efforts to tackle rare neurological and immun=
ological diseases where current options offer little hope, including invest=
igational treatments for gMG, myelin oligodendrocyte glycoprotein antibody-=
associated disease (MOG-AD) and autoimmune encephalitis (AIE).
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=
5.8 billion in 2021. 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=
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oved. Movement from concept to commercial product is uncertain; preclinical=
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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=
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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=
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essful as UCB may have believed at the moment of acquisition. Also, UCB or =
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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=
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UCB is following the worldwide developments diligently to assess the financ=
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Additionally, information contained in this document shall not constitute a=
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References:
1. Data on file.
2. Smith B, et al. Generation and characterization of a high affinity anti-=
human FcRn antibody, rozanolixizumab, and the effects of different molecula=
r formats on the reduction of plasma IgG concentration. MAbs. 2018;10:1111-=
30.
3. US Food and Drug Administration, https://www.fda.gov/patients/fast-track=
-breakthrough-therapy-accelerated-approval-priority-review/priority-review,=
Accessed January 2023
4. US Food and Drug Administration https://www.accessdata.fda.gov/scripts/o=
pdlisting/oopd/detailedIndex.cfm?cfgridkey=3D669918 Accessed January 2023
5. European Medicines Agency, EU/3/20/2272: Orphan designation for the trea=
tment of myasthenia gravis https://www.ema.europa.eu/en/medicines/human/orp=
han-designations/eu3202272 Accessed January 2023
6. Myasthenia Gravis Foundation of America. Clinical Overview of MG. https:=
//myasthenia.org/Professionals/Clinical-Overview-of-MG. Accessed January 20=
23
7. Hansen JS, et al. Mortality in myasthenia gravis: A nationwide populatio=
n-based follow-up study in Denmark. Muscle Nerve. 2016;53:73-77.
8. Salari N, et al. Global prevalence of myasthenia gravis and the effectiv=
eness of common drugs in its treatment: a systematic review and meta-analys=
is. J Transl Med 19, 516 (2021). https://doi.org/10.1186/s12967-021-03185-7=
. Accessed January 2023.
9. Bril V, et al. Rozanolixizumab in generalized myasthenia gravis: Respond=
er analyses from the Phase 3 MycarinG study. Poster 204, AANEM 2022.
10. Howard J, et al. Clinical Effects of the Self-administered Subcutaneous=
Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Genera=
lized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Pla=
cebo-Controlled, Multicenter Clinical Trial. JAMA Neurol 2022 1;77(5)
11. US Food and Drug Administration https://www.accessdata.fda.gov/scripts/=
opdlisting/oopd/detailedIndex.cfm?cfgridkey=3D699319. Accessed January 2023
12. Data on file.
13. Chen J, et al. Incidence, mortality, and economic burden of myasthenia =
gravis in China: A nationwide population-based study. Lancet Reg Health Wes=
t Pac: 2020;5:100063.
14. National institute of Neurological Disorders and Stroke. 2022. Myasthen=
ia Gravis Fact Sheet. https://www.ninds.nih.gov/myasthenia-gravis-fact-shee=
t. Accessed January 2023.
15. Dong D, et al. Gender differences in quality of life among patients wit=
h myasthenia gravis in China. Health and Quality of Life Outcomes 2020 18;2=
96
16. Myasthenia Gravis Foundation of America. MG Quick Facts. https://myasth=
enia.org/MG-Education/MG-Quick-Facts Accessed January 2023
17. ClinicalTrials.gov =E2=80=98A Study to Test Efficacy and Safety of Roza=
nolixizumab in Adult Patients With Generalized Myasthenia Gravis=E2=80=99: =
=C2=A0https://clinicaltrials.gov/ct2/show/NCT03971422. Accessed January 202=
3.
18. Kiessling P, et al. The FcRn inhibitor rozanolixizumab reduces human se=
rum IgG concentration: A randomized phase 1 study. Sci Transl Med. 2017;9(4=
14:eaan1208).
GenericFile
UCB FDA BLA Rozanolixizumab Press Release 06 January ENG (https://mb.cision=
.com/Public/18595/3693925/83418e0d34a70dd2.pdf)
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