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** UCB announces U.S. FDA approval of RYSTIGGO^=C2=AE (rozanolixizumab-noli=
) for the treatment of adults with generalized myasthenia gravis
------------------------------------------------------------
=C2=B7 FDA approval of RYSTIGGO^=C2=AE (rozanolixizumab-noli) has been gran=
ted under the Priority Review designation for the treatment of generalized =
myasthenia gravis (gMG) in adult patients who are anti-acetylcholine recept=
or (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive^=
1
=C2=B7 U.S. FDA approval is based on the pivotal Phase 3 MycarinG study in =
gMG^2, a large phase 3 study which demonstrated treatment with rozanolixizu=
mab-noli resulted in statistically significant improvements in gMG-specific=
outcomes, including everyday activities such as breathing, talking, swallo=
wing, and being able to rise from a chair^3
=C2=B7 Additional treatment option provides opportunity for U.S. clinicians=
to tailor therapeutic approach based on individual patient needs =C2=A0
Brussels (Belgium) 07:00 June 27 2023: (CET) Regulated Information =E2=80=
=93 Inside Information =C2=A0=E2=80=93 UCB (Euronext Brussels: UCB), a glob=
al biopharmaceutical company, today announced RYSTIGGO^=C2=AE (rozanolixizu=
mab-noli)* has been approved by the U.S. Food and Drug Administration (FDA)=
for the treatment of generalized myasthenia gravis (gMG) in adult patients=
who are anti-acetylcholine receptor (AchR) or anti-muscle-specific tyrosin=
e kinase (MuSK) antibody positive.^1
Rozanolixizumab-noli injection for subcutaneous infusion is a humanized IgG=
4 monoclonal antibody that binds to the neonatal Fc receptor (FcRN), result=
ing in the reduction of circulating IgG.^1,4 It is the only FDA-approved tr=
eatment in adults for both anti-AChR and anti-MuSK antibody-positive gMG, t=
he two most common subtypes of gMG.=C2=A0
=E2=80=9CgMG can cause unpredictable fluctuations in severity and frequency=
of symptoms, which are often debilitating and can substantially impact the=
lives of patients. People living with gMG often face treatment options tha=
t are broad-acting, and that have traditionally only offered symptomatic re=
lief=E2=80=9D , explained Professor Vera Bril, Professor of Medicine (Neuro=
logy), University of Toronto, Director of the Neuromuscular Section, Divisi=
on of Neurology, University of Toronto and University Health Network, Toron=
to, and lead investigator of the MycarinG study.=E2=80=9CThere is a signifi=
cant need for new, innovative treatment options to reduce the day-to-day bu=
rden of gMG. Rozanolixizumab-noli is a new treatment option, targeting one =
of the mechanisms of disease to provide symptom improvement in patient-and =
physician reported outcomes at day 43.=E2=80=9D
gMG is a rare, chronic, heterogeneous (phenotypic and pathogenic), unpredic=
table autoimmune disease characterized by dysfunction and damage at the neu=
romuscular junction (NMJ).^4,5,6 Several factors are understood to be drive=
rs of gMG disease pathology, including complement-cascade, immune cells and=
pathogenic IgG autoantibodies. Pathogenic IgG autoantibodies can impair sy=
naptic transmission at the NMJ by targeting specific proteins on the post-s=
ynaptic membrane^4,7=C2=A0This disrupts the ability of the nerves to stimul=
ate the muscle and results in a weaker contraction.^4 gMG has a global prev=
alence of 100=E2=80=93350 cases per every 1 million people.^5
=E2=80=9CNo two people living with gMG experience the disease in the same w=
ay, so we can=E2=80=99t take a =E2=80=98one size fits all=E2=80=99 approach=
to disease management,=E2=80=9D said Iris Loew-Friedrich, Executive Vice-P=
resident and Chief Medical Officer at UCB. =E2=80=9CDisease management shou=
ld be based on the clinical needs and preferences of the individual patient=
, and the aim of treatment is to help restore that patient=E2=80=99s abilit=
y to carry out activities of daily living. The approval of rozanolixizumab-=
noli means doctors have an additional approved treatment option for their g=
MG patients who have not yet found a treatment that meets their needs.=E2=
=80=9D
The FDA approval^1 is supported by safety and efficacy data from the pivota=
l Phase 3 MycarinG study (NCT03971422), published in The Lancet Neurology i=
n May 2023.^1 The primary efficacy endpoint was the comparison of the chang=
e from baseline between treatment groups in the MG-ADL total score at day 4=
3. MG-ADL is a measurement tool which assesses the impact of gMG on daily f=
unctions of 8 signs or symptoms that are typically affected in gMG. These i=
nclude activities such as breathing, talking, swallowing, and being able to=
rise from a chair^3. Each item is assessed on a 4-point scale where a scor=
e of 0 represents normal function and a score of 3 represents loss of abili=
ty to perform that function. A total score ranges from 0 to 24, with the hi=
gher scores indicating more impairment. A statistically significant differe=
nce favoring rozanolixizumab-noli was observed in the MG-ADL total score ch=
ange from baseline [-3.4 points in rozanolixizumab-noli-treated group at ei=
ther dose vs -0.8 points in the placebo-treated group (p<0.001)].=C2=A0
The secondary endpoint was the change between treatment groups from baselin=
e to day 43 in the QMG. The QMG is a 13-item categorical grading system tha=
t assesses muscle weakness. Each item is assessed on a 4-point scale where =
a score of 0 represents no weakness and a score of 3 represents severe weak=
ness. A total possible score ranges from 0 to 39, where higher scores indic=
ate more severe impairment. A statistically significant difference favoring=
rozanolixizumab-noli was observed in the QMG total score change from basel=
ine [-5.4 points and -6.7 points in rozanolixizumab-noli-treated group at =
=E2=89=887mg/kg and =E2=89=8810 mg/kg dose level, respectively, vs -1.9 poi=
nts in the placebo-treated group (p<0.001)].=C2=A0
The most common adverse reactions (reported in at least 10% of patients tre=
ated with in rozanolixizumab-noli) were headache, infections, diarrhea, pyr=
exia, hypersensitivity reactions, and nausea.^1
=E2=80=9CWe want to thank UCB for their continued commitment to the MG comm=
unity to bring a new FDA-approved treatment option for generalized myasthen=
ia gravis to patients and their treating physicians,=E2=80=9D said Samantha=
Masterson, President and Chief Executive Officer of the Myasthenia Gravis =
Foundation of America (MGFA). =E2=80=9CPeople living with generalized myast=
henia gravis continue to experience significant unmet medical needs, this m=
eans expanding the number of FDA-approved treatment options is particularly=
important to treat this chronic, autoimmune, neuromuscular disease.=E2=80=
=9D
Rozanolixizumab-noli will be commercially available in the U.S. during the =
3rd quarter of 2023. =C2=A0
=E2=80=9CBuilding on our decades of experience in neurology and immunology,=
we are proud to support the MG community with solutions to help improve pa=
tient lives, including a new FDA-approved treatment, education and support=
=E2=80=9D, continued Iris Loew-Friedrich. The approval following priority r=
eview of rozanolixizumab-noli is a testament to this medicine=E2=80=99s pot=
ential as a generally well-tolerated treatment option that is targeted to t=
he individual needs of patients. We are so grateful to the patients, care p=
artners, and investigators who participated in the MycarinG study, and to o=
ur employees and collaborators, whose dedication and commitment to the MG c=
ommunity made this important milestone possible.=E2=80=9D
The FDA reviewed rozanolixizumab-noli under Priority Review. Rozanolixizuma=
b is also currently under review by the European Medicines Agency (EMA) and=
the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) for the tre=
atment of adults with gMG. In 2019, the U.S. FDA granted orphan drug design=
ation to rozanolixizumab-noli for the treatment of generalized myasthenia g=
ravis.^8 Orphan designation was granted by the European Commission in April=
2020 to rozanolixizumab for the treatment of generalized myasthenia gravis=
.^9 The PMDA granted similar orphan status to rozanolixizumab in Japan in N=
ovember.^10=C2=A0Responses from regulatory agencies to these submissions ar=
e expected by H1 2024. =C2=A0
About Generalized Myasthenia Gravis (gMG)
gMG is a rare disease with a global prevalence of 100=E2=80=93350 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 life-thr=
eatening weakness of the muscles of respiration, double vision, drooping ey=
elids, and difficulty swallowing, chewing and talking.^11,12
In gMG, pathogenic autoantibodies can impair synaptic transmission at the n=
euromuscular junction (NMJ) by targeting specific proteins on the post-syna=
ptic membrane.^3,4 This disrupts the ability of the nerves to stimulate the=
muscle and results in a weaker contraction. gMG can occur in any race, gen=
der or age.^13=C2=A0=C2=A0
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.
The primary endpoint for the MycarinG study is change in the Myasthenia Gra=
vis-Activities of Daily Living Profile (MG-ADL) score, an eight-item patien=
t-reported scale developed to assess MG symptoms and their effects on daily=
activities. Additional endpoints include response rates, changes in the My=
asthenia Gravis composite (MGC) score, the Quantitative MG (QMG) score, pat=
ient-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 ex=
tensions to this clinical trial. As a result, UCB is exploring the potentia=
l for further extension studies into this treatment.
For more information about the trial, visit https://clinicaltrials.gov/ct2/=
show/NCT03971422.=C2=A0
About rozanolixizumab
Rozanolixizumab is a subcutaneous administered, humanized monoclonal antibo=
dy that specifically binds, with high affinity, to human neonatal Fc recept=
or (FcRn). It has been designed to block the interaction of FcRn and Immuno=
globulin G (IgG), accelerating the catabolism of antibodies and reducing th=
e concentration of pathogenic IgG autoantibodies.^2=C2=A0
Outside of the U.S. rozanolixizumab is not approved for use in any indicati=
on by any other regulatory authority worldwide.
*In the U.S., the International Nonproprietary Name (INN) for rozanolixizum=
ab is =E2=80=98rozanolixizumab-noli=E2=80=99 following the FDA=E2=80=99s =
=E2=80=98Non-proprietary Naming of Biological Products Guidance=E2=80=99. T=
his guidance advises that the nonproprietary name designated for originator=
biological products should be a proper name that is a combination of the c=
ore name and a distinguishing suffix that is devoid of meaning and composed=
of four lowercase letters.=C2=A0
Important Safety Information for RYSTIGGO^=C2=AE
WARNINGS AND PRECAUTIONS=C2=A0
Infections: RYSTIGGO may increase the risk of infection. Delay RYSTIGGO adm=
inistration in patients with an active infection until the infection is res=
olved. During treatment with RYSTIGGO, monitor for clinical signs and sympt=
oms of infection. If serious infection occurs, administer appropriate treat=
ment and consider withholding RYSTIGGO until the infection has resolved.
Immunization
Immunization with vaccines during RYSTIGGO treatment has not been studied. =
The safety of immunization with live or live-attenuated vaccines and the re=
sponse to immunization with any vaccine are unknown. Because RYSTIGGO cause=
s a reduction in IgG levels, vaccination with live-attenuated or live vacci=
nes is not recommended during treatment with RYSTIGGO. Evaluate the need to=
administer age-appropriate vaccines according to immunization guidelines b=
efore initiation of a new treatment cycle with RYSTIGGO.
Aseptic Meningitis: Serious adverse reactions of aseptic meningitis (also c=
alled drug-induced aseptic meningitis) have been reported in patients treat=
ed with RYSTIGGO. If symptoms consistent with aseptic meningitis develop, d=
iagnostic workup and treatment should be initiated according to the standar=
d of care.
Hypersensitivity Reactions: Hypersensitivity reactions, including angioedem=
a and rash, were observed in patients treated with RYSTIGGO. Management of =
hypersensitivity reactions depends on the type and severity of the reaction=
. Monitor patients during treatment with RYSTIGGO and for 15 minutes after =
for clinical signs and symptoms of hypersensitivity reactions. If a reactio=
n occurs, institute appropriate measures if needed.
ADVERSE REACTIONS
In a placebo-controlled study, the most common adverse reactions (reported =
in at least 10% of RYSTIGGO-treated patients) were headache, infections, di=
arrhea, pyrexia, hypersensitivity reactions, and nausea. Serious infections=
were reported in 4% of patients treated with RYSTIGGO. Three fatal cases o=
f pneumonia were identified, caused by COVID-19 infection in two patients a=
nd an unknown pathogen in one patient. Six cases of infections led to disco=
ntinuation of RYSTIGGO.
The full Prescribing Information will be available at https://www.ucb-usa.c=
om/RYSTIGGO-prescribing-information.pdf (https://eur02.safelinks.protection=
.outlook.com/?url=3Dhttps%3A%2F%2Fwww.ucb-usa.com%2FRYSTIGGO-prescribing-in=
formation.pdf&data=3D05%7C01%7CJim.Baxter%40ucb.com%7C12f054d6d36246b0dcf70=
8db769a8ff7%7C237582ad3eab4d44868806ca9f2e613b%7C0%7C0%7C638234178432556862=
%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haW=
wiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=3DE3TjXnLiS9quwi9gMvqi%2Ffn0y5PMh77e=
aJzZoH%2FRaos%3D&reserved=3D0)
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
US Rare Disease Communications
Daphe Teo
Daphne.teo@ucb.com
+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 =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.
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References
1. RYSTIGGO^=C2=AE U.S. Presribing Information
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 G, et al. Myasthenia gravis activities of daily living profile. Ne=
urology. 1992;52(7):1487-9
4. 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.
5. Juel VC, Massey JM. Myasthenia gravis. Orphanet J Rare Dis. 2007;2:44.=
=C2=A0
6. National Institute of Neurological Disorders and Stroke. 2022. Myastheni=
a Gravis Fact Sheet. https://www.ninds.nih.gov/myasthenia-gravis-fact-sheet=
. Accessed June 2023.
7. Punga AR, et al. Epidemiology, diagnostics, and biomarkers of autoimmune=
neuromuscular junction disorders. Lancet Neurol. 2022;21(2):176-88.
8. Myasthenia Gravis Foundation of America. Clinical Overview of MG. https:=
//myasthenia.org/Professionals/Clinical-Overview-of-MG. Accessed June 2023
9. US Food and Drug Administration. https://www.accessdata.fda.gov/scripts/=
opdlisting/oopd/detailedIndex.cfm?cfgridkey=3D669918. Accessed June 2023
10. European Medicines Agency, EU/3/20/2272: Orphan designation for the tre=
atment of myasthenia gravis https://www.ema.europa.eu/en/medicines/human/or=
phan-designations/eu3202272. Accessed June 2023
11. Data on file, UCB Inc
12. Myasthenia Gravis Foundation of America. Clinical Overview of MG. https=
://myasthenia.org/Professionals/Clinical-Overview-of-MG. Accessed June 2023
13. Hansen JS, et al. Mortality in myasthenia gravis: A nationwide populati=
on-based follow-up study in Denmark. Muscle Nerve. 2016;53:73-77.
14. Myasthenia Gravis Foundation of America. MG Quick Facts. https://myasth=
enia.org/MG-Education/MG-Quick-Facts. Accessed June 2023
GenericFile
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