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UCB (EBR:UCB) UCB Media Room: CHMP positive opinion for rozanolixizumab for treatment of adults with generalized myasthenia gravis in Europe

Directive transparence : information réglementée

10/11/2023 18:00
https://mb.cision.com/Public/18595/3873881/a9f1eccc0d70ff8d_800x800ar.png ** 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 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. 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. GenericFile UCB PR Rozi CHMP Nov 10 2023 ENG (https://mb.cision.com/Public/18595/387388= 1/b51736c7ba8a5758.pdf) ______________________ If you would rather not receive future communications from UCB SA, please g= o to https://eu.vocuspr.com/OptOut.aspx?2973226x20421x148347x1x6868579x2400= 0x6&Email=3Dregnews%40symexglobal.com. UCB SA, All=C3=A9e de la Recherche, 60 ., Brussels, . B - 1070 Belgium