RegulationOrphan Drug (US), Breakthrough Therapy (CN), Promising Innovative Medicine (GB), Priority Review (US), Paediatric investigation plan (EU), Fast Track (US), Orphan Drug (EU)

Gene Sequence

Sequence Code 18457995

Source: *****

Clinical Trials associated with Efgartigimod Alfa

NCT06684847 / Not yet recruitingPhase 3

A Phase 3 Randomized, Double-Blinded, Placebo-Controlled Multicenter Trial with Open-Label Extension to Evaluate the Efficacy, Safety, and Tolerability of Efgartigimod PH20 Subcutaneous Administered by Prefilled Syringe in Adult Patients with Primary Sjögren's Disease

The main purpose of the proposed study is to evaluate the efficacy of efgartigimod PH20 SC in patients with moderate-to-severe Primary Sjögren's Disease (pSjD). The study consists of a double-blinded placebo-controlled treatment period and an open-label treatment period. The maximum study duration for participants in both study parts is approximately 105 weeks.

Start Date01 Dec 2024

Sponsor / Collaborator

argenx SE

NCT06655155 / Not yet recruitingPhase 2

A Randomized, Double-Blinded, Placebo-Controlled, Phase 2, Parallel-Group Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Adult Participants with Systemic Sclerosis

The main purpose of this study is to evaluate the effect and safety of efgartigimod PH20 SC compared to placebo in adults with systemic sclerosis. The study consists of a screening period, a treatment period of up to 48 weeks and a safety follow-up period. After the screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod PH20 SC or placebo. The total study duration can be up to approximately 15 months.

Start Date18 Nov 2024

Sponsor / Collaborator

argenx SE

NCT06688253 / Not yet recruitingPhase 4IIT

Intravenous Human IgG1 Fc Fragment (Efgartigimod) in Myasthenic Crisis

Efgartigimod in Myasthenic Crisis Background Myasthenia gravis (MG) is a prevalent autoimmune disorder affecting neuromuscular junctions, characterized by weakness in skeletal muscles. It is associated with the production of autoantibodies, primarily targeting acetylcholine receptors (AchR), and is often complicated by myasthenic crisis, which can lead to severe respiratory failure. Current treatments primarily involve non-specific immunosuppression, which may not provide rapid relief.
Aim This study investigates the therapeutic impact of efgartigimod, an FcRn-targeting Fc fragment, on patients experiencing a myasthenic crisis. We hypothesize that efgartigimod is non-inferior to conventional treatments like intravenous immunoglobulin (IVIG) and plasma exchange (PLEX) in terms of clinical efficacy and safety.
Study Rationale Efgartigimod aims to reduce pathogenic IgG autoantibodies implicated in MG by accelerating their degradation. This targeted approach could provide faster symptom relief during acute exacerbations compared to existing therapies.
Objectives Primary Objective: To assess the non-inferiority of efgartigimod compared to PLEX and IVIG based on MG-ADL improvements.
Secondary Objectives: Evaluate safety, tolerability, length of hospital stay, respiratory parameters, need for additional therapies, and one-year outcomes.
Primary Endpoint MG-ADL Improvement: Defined as a ≥3-point improvement post-treatment. The comparison will be made using one-month post-treatment assessments, with follow-ups every three months.
Secondary Endpoints Safety and tolerability Length of hospital stay Changes in respiratory function Need for rescue therapy in case of clinical deterioration Sample Size The study will recruit 32 patients (16 historical group and 16 interventional group), calculated to detect significant differences in MG-ADL improvements with a significance level of 0.05 and power of 0.80.
Patient Recruitment Patients with a confirmed diagnosis of MG who present to the neurology department will be recruited and randomly assigned to either the efgartigimod treatment group or the historical control group receiving standard care (IVIG/PLEX).
Inclusion Criteria Adults > 18 years Confirmed MG diagnosis with generalized weakness (MGFA class II-V) Positive AchR or MuSK antibodies Evidence of myasthenic crisis Informed consent Exclusion Criteria Contraindications to efgartigimod Significant comorbidities affecting study participation Prior exposure to efgartigimod Ongoing infections or conditions exacerbating MG symptoms Recent major surgery or significant renal/hepatic dysfunction Planned Protocol Administer efgartigimod intravenously at 10 mg/kg weekly for four weeks. Total trial duration: 12 months for enrollment and treatment, followed by a 14-month follow-up.

Start Date15 Nov 2024

Sponsor / Collaborator

Rambam Health Care Campus

[+1]

100 Clinical Results associated with Efgartigimod Alfa

100 Translational Medicine associated with Efgartigimod Alfa

100 Patents (Medical) associated with Efgartigimod Alfa

116

Literatures (Medical) associated with Efgartigimod Alfa

01 Dec 2024·ADVANCES IN THERAPY

Risk–Benefit Analysis of Novel Treatments for Patients with Generalized Myasthenia Gravis

Article

Author: Qi, Cynthia Z. ; Brauer, Edward ; Smith, A. Gordon ; Yang, Hongbo ; Wolfe, Gil I ; Gelinas, Deborah ; Saccà, Francesco ; Smith, A Gordon ; Chen, Xin ; Phillips, Glenn ; Qi, Cynthia Z ; Habib, Ali A ; Wolfe, Gil I. ; Habib, Ali A. ; Du, Mandy

INTRODUCTION:

This study used network meta-analysis (NMA) to inform and compare the number needed to treat (NNT), number needed to harm (NNH), and cost per improved outcome (CPIO) associated with more recently approved treatments for anti-acetylcholine receptor antibody-positive (anti-AChR Ab+) generalized myasthenia gravis (gMG).

METHODS:

Clinical trials of neonatal Fc receptor (FcRn) inhibitors, efgartigimod intravenous (IV) and rozanolixizumab, and complement inhibitors, ravulizumab and zilucoplan, versus placebo (with background conventional treatment) were included in the primary NMA to compare efficacy and safety outcomes. The outputs from the NMAs were used to estimate NNT and NNH of each treatment versus placebo. CPIO (2024 USD) was estimated for a ≥ 3- or ≥ 5-point reduction from baseline in Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores. Sensitivity analyses were performed adding efgartigimod PH20 subcutaneous (SC) and eculizumab to the NMA.

RESULTS:

Efgartigimod IV had the lowest NNT versus placebo for achieving a ≥ 3- and ≥ 5-point reduction in QMG, as well as a ≥ 5-point reduction in MG-ADL, whereas rozanolixizumab had the lowest NNT for a ≥ 3-point reduction in MG-ADL. The NNH versus placebo was similar across comparator treatments. Efgartigimod IV had the lowest CPIO among all treatments for all assessed efficacy outcomes. Sensitivity analyses yielded results consistent with primary analysis and indicated that efgartigimod PH20 SC had comparable NNT and CPIO values to efgartigimod IV, whereas eculizumab had comparable NNT and higher CPIO values compared to other complement inhibitors.

CONCLUSIONS:

FcRn inhibitors and complement inhibitors assessed in this study all demonstrated clinical benefit in terms of NNT as well as an acceptable safety profile in terms of NNH. Within the limitations of this meta-analysis, efgartigimod was associated with a favorable benefit-risk profile as well as a better economic value compared to ravulizumab, rozanolixizumab, and zilucoplan as treatments for anti-AChR Ab+ gMG.

01 Nov 2024·NEUROLOGICAL SCIENCES

Successful treatment with efgartigimod as an add-on therapy in acute attack of anti-AQP4 antibody-positive NMOSD: a case report

Article

Author: Zhao, Hong-Dong ; Jiang, Teng ; Shi, Jian-Quan ; Huang, Shi-Qi ; Yuan, Zhen-Hua ; Hong, Ye

BACKGROUND:

Neuromyelitis Optica Spectrum Disorder (NMOSD) is an autoimmune demyelinating disease characterized by recurrent myelitis and optic neuritis. It is associated with high rates of relapse and disability. The main treatment strategies for acute attacks include intravenous methylprednisolone pulse (IVMP) treatment and rescue treatment with plasma exchange (PLEX). Recently, the blockade of neonatal Fc receptor (FcRn)-IgG interaction has gained momentum as a therapeutic strategy. Efgartigimod, the first approved FcRn inhibitor for treating generalized myasthenia gravis, has shown impressive safety, efficacy, and tolerability, and is being regarded as "PLEX in a bottle".

CASE DESCRIPTION:

We report a 65-year-old female patient who was diagnosed with anti-AQP4 antibody positive NMOSD. Add-on treatment with efgartigimod to IVMP and intravenous immunoglobulin (IVIG) at the second acute relapse showed favorable results.

CONCLUSION:

This case suggests that efgartigimod is a potentially effective add-on therapy in acute attacks of AQP4-IgG-positive NMOSD.

01 Nov 2024·JOURNAL OF THE NEUROLOGICAL SCIENCES

Post-hoc analyses from the ADAPT clinical study demonstrate aggregate sustained benefit of Efgartigimod in generalized myasthenia gravis

Article

Author: Cynthia, Qi Z. ; Mantegazza, Renato ; Brauer, Edward ; Edward, Brauer ; Gelinas, Deborah ; Qi, Cynthia Z. ; Renato, Mantegazza ; Howard James, F. ; Deborah, Gelinas ; Sarah, Dewilde ; Alison, Griffiths ; Griffiths, Alison ; Dewilde, Sarah ; Phillips, Glenn ; Glenn, Phillips ; Qi, Cynthia Z ; Howard, James F ; Howard, James F.

OBJECTIVES:

This post-hoc analysis evaluates the long-term efficacy of efgartigimod versus placebo in adult patients with generalized myasthenia gravis (gMG) with acetylcholine-receptor autoantibodies (AChR-Ab+), based on data from the ADAPT RCT and its open-label extension ADAPT+.

METHODS:

Changes from baseline in Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores were assessed by treatment group over the ADAPT (up to 20 weeks) and ADAPT+ time horizon (extended to 64 weeks for efgartigimod group patients). Response to treatment was defined as (Meriggioli, 2009 [3]⁾5-point reduction in QMG or (Meriggioli, 2009 [3]⁾3-point reduction in MG-ADL vs. baseline values.

RESULTS:

AChR-Ab+ patients treated with efgartigimod spent a substantially greater percentage of time in response in ADAPT based on at least a 5-point change in QMG compared to the placebo group (44 % versus 13 % respectively, p = 0.0034). Analyses based on a 3-point change in MG-ADL in ADAPT showed the percentage of time in response was nearly double for efgartigimod versus placebo (59 % versus 30 % respectively, p = 0.010). These trends were also maintained using different response definitions, as well as in patients with and without prior immune therapy exposure and by time from diagnosis (<7 years versus ≥7 years).

CONCLUSIONS:

The clinical benefit of efgartigimod was sustained over repeat treatment cycles and maintained over the long term. Response to treatment was consistent regardless of response definition and was repeated in different patient subgroups. Overall, the results of this analysis indicate that efgartigimod is an effective therapeutic option, demonstrating a robust benefit among AChR-Ab+ patients with gMG.

249

News (Medical) associated with Efgartigimod Alfa

12 Nov 2024

·www.businesswire.com

Viridian Therapeutics Reports Third Quarter 2024 Financial Results and Recent Progress Including New FcRn Data

WALTHAM, Mass.--( BUSINESS WIRE )--Viridian Therapeutics, Inc. (NASDAQ: VRDN), a biotechnology company focused on discovering and developing potential best-in-class medicines for serious and rare diseases, today reported recent business highlights and financial results for the third quarter ending September 30, 2024. “ We had another exceedingly strong quarter of execution across our TED and FcRn portfolios,” said Steve Mahoney, Viridian’s President and Chief Executive Officer. “ In TED, we reported highly-compelling and positive phase 3 data for veligrotug and eagerly anticipate the readout of THRIVE-2 in December. We also initiated both pivotal phase 3 clinical trials for VRDN-003, our next-generation subcutaneous IGF-1R inhibitor. For our FcRn inhibitor portfolio, we are thrilled today to share VRDN-008 NHP data for the first time, demonstrating a potential best-in-class pharmacokinetic and pharmacodynamic profile for this half-life extended bispecific FcRn inhibitor. Together with our Fc fragment approach with VRDN-006, we believe these programs in our FcRn portfolio will bring differentiated options for patients and each contribute to an exciting pipeline beyond TED. This quarter, we also further strengthened our cash position with a financing that allows us to accelerate our R&D pipeline and extend our cash runway into the second half of 2027.” RECENT TED PORTFOLIO PROGRESS Veligrotug is an intravenously delivered anti-insulin-like growth factor-1 receptor (IGF-1R) antibody in phase 3 development for thyroid eye disease. Achieved All Primary and Secondary Endpoints in THRIVE: In September, Viridian announced positive topline results for veligrotug in THRIVE, a global phase 3 clinical trial in patients with active TED. Veligrotug met all of its primary and secondary endpoints after five infusions with high statistical significance (p < 0.0001) and was generally well-tolerated. THRIVE-2 Topline Data Readout on Track for December 2024: THRIVE-2 is a global phase 3 clinical trial assessing the efficacy and safety of veligrotug after five infusions in patients with chronic TED. THRIVE-2 completed enrollment in July 2024 with a total of 188 patients, exceeding the enrollment target of 159 patients due to patient demand, and is on track for topline readout of the 15-week primary efficacy analysis in December. Veligrotug BLA on Track for Second Half 2025: Viridian anticipates submitting a Biologics License Application (BLA) in the second half of 2025, pending data, and expects that its data package will support a marketing authorization application in Europe. VRDN-003 is a potential best-in-class, subcutaneous, half-life extended anti-IGF-1R antibody with the same binding domain as veligrotug. REVEAL-1 and REVEAL-2 Initiated in August 2024: REVEAL-1 and REVEAL-2 are designed to evaluate VRDN-003 in patients with active and chronic TED, respectively, via a subcutaneous administration every four weeks or every eight weeks. Viridian believes the THRIVE topline results provide strong evidence to support the profile of VRDN-003 as a potential best-in-class subcutaneous anti-IGF-1R antibody in a low-volume, infrequent, self-administered, subcutaneous injection designed for patients to use at home. Topline Data and BLA in 2026: Viridian anticipates topline data from both REVEAL-1 and REVEAL-2 in the first half of 2026, with a BLA submission planned by year-end 2026. The company plans to launch VRDN-003 with a commercially available low-volume autoinjector for at-home administration. FCRN PORTFOLIO UPDATE WITH NEW VRDN-008 NHP DATA VRDN-006 is a highly selective Fc fragment which inhibits FcRn, and is designed to be a convenient subcutaneous and self-administered option for patients. IND on Track for Year-End 2024: Viridian is on track to submit an IND application for VRDN-006 by year-end 2024. Viridian anticipates starting a phase 1 clinical trial for VRDN-006 in healthy volunteers in early 2025 and proof-of-concept IgG reductions from that study in the second half of 2025. VRDN-008 is a half-life extended bispecific FcRn inhibitor comprising an Fc fragment and an albumin-binding domain designed to prolong IgG suppression and provide a potentially best-in-class subcutaneous option for patients. Positive New NHP Data Confirms Half-Life Extension: In a head-to-head study, single doses of VRDN-008 demonstrated 3x the half-life of efgartigimod and showed a deeper and more sustained IgG reduction in NHPs with peak IgG reductions that were 20% deeper than efgartigimod. IgG levels returned to baseline 35 days after VRDN-008 dosing, more than twice as long as efgartigimod, which returned to baseline 14 days after dosing. VRDN-008 spared albumin and low-density lipoprotein (LDL), consistent with efgartigimod. Given that NHP data for FcRn inhibitors have been highly translatable to humans, Viridian believes this data shows the potential for VRDN-008 to be a best-in-class, extended half-life FcRn inhibitor for patients. IND Submission Expected Year-End 2025: With these positive proof-of-concept data for VRDN-008, Viridian plans to further advance IND-enabling activities and submit an IND by year-end 2025. Viridian anticipates proof-of-concept IgG reduction data in healthy volunteers in the second half of 2026. FINANCIAL RESULTS Cash Position : Cash, cash equivalents, and short-term investments were $753.2 million as of September 30, 2024, compared with $571.4 million as of June 30, 2024. The company completed a public offering in September 2024 with net proceeds of $243.2 million and believes that its current cash, cash equivalents, and short-term investments will be sufficient to fund its operations into the second half of 2027. R&D Expenses : Research and development expenses were $69.2 million during the quarter ended September 30, 2024, compared to $30.4 million during the quarter ended September 30, 2023. The increase in research and development expenses was driven by increased clinical trials costs associated with our ongoing THRIVE and THRIVE-2 pivotal clinical trials, as well as increased personnel costs to support our pipeline development. G&A Expenses : General and administrative expenses were $14.4 million during the quarter ended September 30, 2024, compared to $20.9 million during the quarter ended September 30, 2023. The decrease in general and administrative expenses was driven by a decrease in personnel-related costs, including share-based compensation expenses. Net Loss : The company’s net loss was $76.7 million for the third quarter ended September 30, 2024, compared with $47.7 million for the same period last year. Shares Outstanding: As of September 30, 2024, Viridian had 97,850,645 shares of common stock outstanding on an as-converted basis, which included 79,181,445 shares of common stock and an aggregate 18,669,200 shares of common stock issuable upon the conversion of 134,864 and 145,160 shares of Series A and Series B preferred stock, respectively. About Viridian Therapeutics Viridian is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases. Viridian’s expertise in antibody discovery and protein engineering enables the development of differentiated therapeutic candidates for previously validated drug targets in commercially established disease areas. Viridian is advancing multiple candidates in the clinic for the treatment of patients with thyroid eye disease (TED). The company is conducting a pivotal program for veligrotug (VRDN-001), including two global phase 3 clinical trials (THRIVE and THRIVE-2), to evaluate its efficacy and safety in patients with active and chronic TED. Viridian is also advancing VRDN-003 as a potential best-in-class subcutaneous therapy for the treatment of TED, including two ongoing global phase 3 pivotal clinical trials, REVEAL-1 and REVEAL-2, to evaluate the efficacy and safety of VRDN-003 in patients with active and chronic TED. In addition to its TED portfolio, Viridian is advancing a novel portfolio of neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008, which has the potential to be developed in multiple autoimmune diseases. Viridian is based in Waltham, Massachusetts. For more information, please visit www.viridiantherapeutics.com . Follow Viridian on LinkedIn and X . Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as, but not limited to, “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “on track,” “plan,” “potential,” “predict,” “project,” “design,” “should,” “target,” “will,” or “would” or other similar terms or expressions that concern our expectations, plans and intentions. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations, and assumptions. Forward-looking statements include, without limitation, statements regarding: preclinical and clinical development of Viridian’s product candidates veligrotug (formerly VRDN-001), VRDN-003, VRDN-006 and VRDN-008; anticipated start dates of studies, including the initiation date of the phase 1 clinical trial for VRDN-006; milestones; timelines; anticipated data results and timing of their disclosure, including topline results; regulatory interactions and anticipated timing of regulatory submissions, including the anticipated IND submissions for VRDN-006 and VRDN-008 and the anticipated BLA submissions for veligrotug and VRDN-003; Viridian’s expectation that its data package will support a BLA submission for veligrotug in the second half of 2025, pending data; Viridian’s expectation that its data package will support a marketing authorization application in Europe for veligrotug; clinical trial designs, including the REVEAL-1 and REVEAL-2, global phase 3 clinical trials for VRDN-003; Viridian’s plans to launch VRDN-003 with a commercially available auto-injector pen, if approved; the potential utility, efficacy, potency, safety, clinical benefits, clinical response, convenience and number of indications of veligrotug, VRDN-003, VRDN-006 and VRDN-008; potential dosing regimen for VRDN-008; Viridian’s product candidates potentially being best-in-class; and that Viridian’s cash, cash equivalents and short-term investments will be sufficient to fund its operations into the second half of 2027. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to: potential utility, efficacy, potency, safety, clinical benefits, clinical response and convenience of Viridian’s product candidates; that results or data from completed or ongoing clinical trials may not be representative of the results of ongoing or future clinical trials; that preliminary data may not be representative of final data; the timing, progress and plans for our ongoing or future research, preclinical and clinical development programs; changes to trial protocols for ongoing or new clinical trials; expectations and changes regarding the timing for regulatory filings; regulatory interactions expectations and changes regarding the timing for enrollment and data; uncertainty and potential delays related to clinical drug development; the duration and impact of regulatory delays in our clinical programs; the timing of and our ability to obtain and maintain regulatory approvals for our therapeutic candidates; manufacturing risks; competition from other therapies or products; estimates of market size; other matters that could affect the sufficiency of existing cash, cash equivalents and short-term investments to fund operations; our financial position and projected cash runway; our future operating results and financial performance; Viridian’s intellectual property position; the timing of preclinical and clinical trial activities and reporting results from same; and those risks set forth under the caption “Risk Factors” in our most recent quarterly report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on August 8, 2024 and other subsequent disclosure documents filed with the SEC. Any forward-looking statement speaks only as of the date on which it was made. Neither the company, nor its affiliates, advisors, or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing the company’s views as of any date subsequent to the date hereof. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (amounts in thousands, except share and per share data) (unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Revenue: Collaboration Revenue - related party $ 86 $ 72 $ 230 $ 242 Total revenue 86 72 230 242 Operating Expenses: Research and development 69,158 30,385 166,294 121,208 General and administrative 14,408 20,911 45,499 62,006 Total operating expenses 83,566 51,296 211,793 183,214 Loss from operations (83,480 ) (51,224 ) (211,563 ) (182,972 ) Other income (expense) Interest and other income 7,795 4,164 23,527 13,029 Interest and other expense (1,004 ) (600 ) (2,188 ) (931 ) Net loss (76,689 ) (47,660 ) (190,224 ) (170,874 ) Change in unrealized gain on investments 1,475 109 594 326 Comprehensive loss $ (75,214 ) $ (47,551 ) $ (189,630 ) $ (170,548 ) Net loss $ (76,689 ) $ (47,660 ) $ (190,224 ) $ (170,874 ) Net loss per share, basic and diluted $ (1.15 ) $ (1.09 ) $ (2.98 ) $ (3.97 ) Weighted-average shares used to compute basic and diluted loss per share 66,420,063 43,654,577 63,800,798 43,057,658 Viridian Therapeutics, Inc. Selected Financial Information Condensed Consolidated Balance Sheets (amounts in thousands) (unaudited) September 30, December 31, 2024 2023 Cash, cash equivalents and short-term investments $ 753,240 $ 477,370 Other assets 18,660 13,054 Total assets $ 771,900 $ 490,424 Total liabilities 64,404 48,402 Total stockholders’ equity 707,496 442,022 Total liabilities and stockholders’ equity $ 771,900 $ 490,424

Phase 3Financial StatementClinical ResultPhase 1Phase 2

12 Nov 2024

·www.pharmaceutical-technology.com

Zai Lab and argenx announce NMPA approval for sBLA for VYVGART Hytrulo

The therapy is a once-weekly SC injection. Credit: Chinnapong via Shutterstock. Zai Lab and Argenx have announced receipt of approval from the National Medical Products Administration (NMPA) of China for the supplemental biologics license application (sBLA) for VYVGART Hytrulo 1,000mg vial for treating chronic inflammatory demyelinating polyneuropathy (CIDP) in adults. This once-weekly 30-90-second subcutaneous (SC) injection reportedly marks the first and only approval in China for a therapy to treat this immune-mediated neuromuscular disorder of the peripheral nervous system. The approval is based on the positive outcome of the randomised, double-blind, placebo-controlled multicentre ADHERE study. The trial comprised an open-label period to detect responders, followed by a randomised-withdrawal, double-blinded period to further evaluate the therapy. Patients from the Greater China region were enrolled in the ADHERE trial by Zai Lab, and the treatment response observed was in line with global study results. A 69% reduction in the risk of relapse with VYVGART Hytrulo compared to placebo was shown in a subgroup analysis of Chinese individuals. Furthermore, 78% of Chinese individuals showed clinical improvement during the open-label period of the study. The safety and tolerability profile of VYVGART Hytrulo was also favourable among the Chinese participant group, consistent with global trial participants. Zai Lab holds an exclusive license agreement with argenx to develop and commercialise VYVGART Hytrulo in Greater China. Zai Lab Global Research and Development head Rafael G Amado said: “We are pleased to receive NMPA approval for VYVGART Hytrulo, marking a groundbreaking milestone for CIDP patients in China. “This approval brings a much-needed treatment option to patients who have been suffering from CIDP for far too long. We will continue to work with argenx to explore the potential in other immunoglobulin G (IgG)-mediated autoimmune indications.” argenx CEO Tim Van Hauwermeiren said: “VYVGART Hytrulo is a precision therapy for patients living with CIDP, many of whom have been waiting for a new treatment innovation. “We are grateful to our partners at Zai Lab for collaborating with argenx to reach CIDP patients in China, and to the NMPA for approving VYVGART Hytrulo for CIDP.” In May this year, China’s Centre for Drug Evaluation (CDE) accepted the sBLA with a priority review designation for VYVGART Hytrulo for CIDP. Last month, the CDE granted breakthrough status for the therapy. In July this year, the NMPA approved efgartigimod for treating generalised myasthenia gravis.

Drug ApprovalLicense out/inPriority ReviewClinical ResultBreakthrough Therapy

11 Nov 2024

·www.businesswire.com

Zai Lab and argenx Announce Approval of VYVGART Hytrulo for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) in China

SHANGHAI & CAMBRIDGE, Mass.--( BUSINESS WIRE )--Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) and argenx (Euronext & Nasdaq: ARGX) today announced that China’s National Medical Products Administration (NMPA) approved the supplemental Biologics License Application (sBLA) for VYVGART Hytrulo 1,000mg (5.6ml)/vial [Efgartigimod Alfa Injection (Subcutaneous Injection)] for the treatment of adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP). VYVGART Hytrulo is approved for CIDP as a once weekly 30-to-90 second subcutaneous injection. It is the first and only therapy approved in China for the treatment of CIDP, a debilitating, often progressive, immune-mediated neuromuscular disorder of the peripheral nervous system. “We are pleased to receive NMPA approval for VYVGART Hytrulo, marking a groundbreaking milestone for CIDP patients in China,” said Rafael G. Amado, M.D., President, Head of Global Research and Development at Zai Lab. “This approval brings a much-needed treatment option to patients who have been suffering from CIDP for far too long. We appreciate the NMPA for their thorough assessment and recognition of the therapy’s differentiated profile and the large unmet patient medical need in China. We will continue to work with argenx to explore the potential in other Immunoglobulin G (IgG)-mediated autoimmune indications.” “VYVGART Hytrulo is a precision therapy for patients living with CIDP, many of whom have been waiting for a new treatment innovation,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx. “We are grateful to our partners at Zai Lab for collaborating with argenx to reach CIDP patients in China, and to the NMPA for approving VYVGART Hytrulo for CIDP. Zai has a strong record of impeccable execution and a shared value of doing all that we can, together, for patients in need. We look forward to continuing our partnership with Zai as argenx continues to reach more patients in one of the world’s fastest growing markets.” “CIDP is a serious and debilitating disease with approximately 50,000 diagnosed patients in China 1 , with only a small fraction of patients able to achieve remission on corticosteroids and plasma-derived therapies, the current standard of care.” said Prof. Ting Chang, M.D., Deputy Chief Physician and Associate Professor, Department of Neurology, Tangdu Hospital. “In addition, existing treatment options are problematic and challenging for some patients. VYVGART Hytrulo provides a new, safe and effective treatment option that can meaningfully improve and stabilize disease symptoms and potentially lessen the burden of treatment for these patients. This is an important advancement for the patient community, and we are grateful to Zai Lab for their work supporting patients who have been devastated by this disease for so long.” The NMPA approval is supported by the positive results from the ADHERE (NCT04281472) study, a multicenter, randomized, double-blind, placebo-controlled trial evaluating VYVGART Hytrulo for the treatment of CIDP. The ADHERE study included an open-label period to identify responders who then entered a randomized-withdrawal, double-blinded period. Zai Lab enrolled patients into the ADHERE trial in Greater China and treatment response in these participants was consistent with global study outcomes. Subgroup analysis of Chinese participants demonstrated a 69% reduction in the risk of relapse with VYVGART Hytrulo compared to placebo. In addition, 78% of Chinese participants treated in the open-label period of the study demonstrated evidence of clinical improvement, further confirming the role IgG autoantibodies play in the underlying biology of CIDP. The favorable safety and tolerability profile of VYVGART Hytrulo dosed weekly in the Chinese patient cohort was consistent with what was shown in global trial participants. In May 2024, Zai Lab announced that the Centre for Drug Evaluation (CDE) accepted the sBLA with priority review designation for VYVGART Hytrulo for CIDP in China. The CDE granted the Breakthrough Therapy Designation for the treatment of patients with CIDP in September 2023. About VYVGART Hytrulo VYVGART Hytrulo is a subcutaneous product that consists of efgartigimod alfa, a human IgG1 antibody fragment, and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE ® drug delivery technology to facilitate subcutaneous delivery of biologics. The product is a single subcutaneous injection (1,000 mg fixed dose) delivered over 30-to-90 seconds and given weekly. VYVGART Hytrulo can be administered by a healthcare professional or at home by the patient or caregiver after adequate training in the subcutaneous injection technique. It is approved in the United States (marketed as VYVGART ® Hytrulo for generalized myasthenia gravis (gMG) and CIDP), EU (marketed as VYVGART ® SC for gMG), Japan (marketed as VYVDURA ® for gMG) and China (marketed as VYVGART Hytrulo ® for gMG and CIDP). Zai Lab has an exclusive license agreement with argenx to develop and commercialize efgartigimod in mainland China, Hong Kong, Macau, and Taiwan (collectively, Greater China). About CIDP in China There are an estimated 50,000 patients diagnosed with CIDP in mainland China. 1 Current treatment options are primarily corticosteroids and intravenous immunoglobulin (IVIg), with plasma exchange (PLEX) generally reserved for refractory patients. There is limited access to PLEX or IVIg in many parts of the world, including China. Because most patients require treatment for an extended period, there remains a significant unmet need for alternative treatment options that are effective, well-tolerated, and convenient for patients with CIDP in China. 1 Chronic inflammatory demyelinating polyneuropathy and diabetes, 2020. About Zai Lab Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) is an innovative, research-based, commercial-stage biopharmaceutical company based in China and the United States. We are focused on discovering, developing, and commercializing innovative products that address medical conditions with significant unmet needs in the areas of oncology, immunology, neuroscience, and infectious disease. Our goal is to leverage our competencies and resources to positively impact human health in China and worldwide. For additional information about Zai Lab, please visit www.zailaboratory.com or follow us at www.twitter.com/ZaiLab_Global . About argenx argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker in the U.S., Japan, Israel, the EU, the UK, Canada and China. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit www.argenx.com and follow us on LinkedIn , X/Twitter , Instagram , Facebook , and YouTube . Zai Lab Forward-Looking Statements This press release contains forward-looking statements about future expectations, plans, and prospects for Zai Lab, including, without limitation, statements regarding the prospects of and plans for development and commercialization of efgartigimod in Greater China, the safety and efficacy of efgartigimod, and the potential treatment of patients with CIDP and other autoimmune disorders in Greater China. These forward-looking statements may contain words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “plan,” “possible,” “potential,” “will,” “would,” and other similar expressions. Such statements constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are not statements of historical fact or guarantees or assurances of future performance. Forward-looking statements are based on our expectations and assumptions as of the date of this press release and are subject to inherent uncertainties, risks, and changes in circumstances that may differ materially from those contemplated by the forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including but not limited to (1) our ability to successfully commercialize and generate revenue from our approved products, (2) our ability to obtain funding for our operations and business initiatives, (3) the results of clinical and pre-clinical development of our product candidates, (4) the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approvals of our product candidates, (5) risks related to doing business in China, and (6) other factors identified in our most recent annual and quarterly reports and in other reports we have filed with the U.S. Securities and Exchange Commission (SEC). We anticipate that subsequent events and developments will cause our expectations and assumptions to change, and we undertake no obligation to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise, except as may be required by law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release. Our SEC filings can be found on our website at www.zailaboratory.com and the SEC’s website at www.sec.gov .

Drug ApprovalLicense out/inBreakthrough TherapyClinical ResultPriority Review

100 Deals associated with Efgartigimod Alfa

External Link

KEGG	Wiki	ATC	Drug Bank
-	Efgartigimod Alfa	-	DB15270

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Purpura, Thrombocytopenic, Idiopathic	JP	argenx SE	26 Mar 2024
Myasthenia Gravis	US	arGEN-X BV	17 Dec 2021

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating	NDA/BLA	CN	Zai Lab (Shanghai) Co., Ltd.	24 Apr 2024
Primary Sjögren's syndrome	Phase 3	-	argenx SE	01 Dec 2024
Graves Ophthalmopathy	Phase 3	US	argenx SE	27 Mar 2024
Graves Ophthalmopathy	Phase 3	CN	argenx SE	27 Mar 2024
Graves Ophthalmopathy	Phase 3	BG	argenx SE	27 Mar 2024
Graves Ophthalmopathy	Phase 3	EE	argenx SE	27 Mar 2024
Graves Ophthalmopathy	Phase 3	HU	argenx SE	27 Mar 2024
Graves Ophthalmopathy	Phase 3	ES	argenx SE	27 Mar 2024
Pemphigoid, Bullous	Phase 3	US	Zai Lab (Shanghai) Co., Ltd.	22 Mar 2023
Pemphigoid, Bullous	Phase 3	US	Argenx BV	22 Mar 2023

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04687072 (ADVANCE SC, CTgov)	Phase 3	Purpura, Thrombocytopenic, Idiopathic	207	Efgartigimod PH20 SC (Efgartigimod PH20 SC)	hggkqlifvo(jrmqelqjvw) = jxreyxhrrh qboonhkhqe (isvawbvbai, idwmqqrfiq - dwrtguoggx) View more	-	31 Oct 2024
				Placebo PH20 SC (Placebo PH20 SC)	hggkqlifvo(jrmqelqjvw) = jachtcldug qboonhkhqe (isvawbvbai, bweveyrkgw - jtwmpzfuwb) View more
NCT04735432 (ADAPT-SC, NEWS) Manual	Phase 3	Myasthenia Gravis 乙酰胆碱受体（AChR）抗体阳性	-	艾加莫德	nuwzkzskea(trioeodeol) = hcymryzrgq qtcvbhiybr (zwjqfsceij ) Met	Non-inferior	17 Jul 2024
				efgartigimod IV	nuwzkzskea(trioeodeol) = ouvgqixkyp qtcvbhiybr (zwjqfsceij ) Met
EAN	Not Applicable	-	-	Efgartigimod IV	kikmtmnwcu(uyhvvjqtev) = xlyxrtbfxx kifxjmyspz (vhdgwywpuh )	-	28 Jun 2024
				Efgartigimod SC	kikmtmnwcu(uyhvvjqtev) = dlgctwrqoa kifxjmyspz (vhdgwywpuh )
ADHERE (EAN)	Not Applicable	-	322	Efgartigimod PH20 SC	mfacdfhieh(duphmbiiin) = ymdpzsuhnv jcwnlljqrw (crebdvpjrz )	Positive	28 Jun 2024
				Placebo	-
EAN	Not Applicable	-	-	Efgartigimod IV	ziwldtwmnr(wjvtjtwzej) = icwnlaflty pathtweadm (nbwojoktsh, 2.2)	-	28 Jun 2024
EAN	Not Applicable	-	-	Efgartigimod	wltvxbhztr(ataowdzmbm) = gecmvefzfc kxtcbyabve (cmbivmvbou ) View more	-	28 Jun 2024
ADAPT/ADAPT+ (AAN2024)	Phase 3	Myasthenia Gravis acetylcholine receptor antibody-positive	-	Efgartigimod	aavkriabna(rzxnpirkgt) = 0.3-point increase elsjfhbyaj (bwvurrxfiz ) View more	Positive	09 Apr 2024
				Placebo
P4-14.016 (AAN2024)	Phase 2/3	Autoimmune Diseases IgG-mediated autoimmune disorders	-	Efgartigimod	hlojyvtmio(ikhwvwezuz) = ovtcuwwwep bmweeqnssd (pxdyvhulvt )	Positive	09 Apr 2024
				Placebo	hlojyvtmio(ikhwvwezuz) = cnaxctjtgm bmweeqnssd (pxdyvhulvt )
ADPT trial (MDA2024) Manual	Not Applicable	Myasthenia Gravis	17	Efgartigimod	scfttqdvwt(dxeataaknl) = Most common side effects reported were mild infections, headache, and nausea. nwmxbdkkat (tewgdkdmcr )	Positive	03 Mar 2024
MDA2024	Not Applicable	Myasthenia Gravis	-	Efgartigimod 10 mg/kg IV	dzqgmlslvj(kiiqzbkdhw) = 16% nsccqyzmjj (hqtivglrgv ) View more	-	03 Mar 2024

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