12 Breakthrough Therapies Expected for Approval in China in H2 2024: Innovations from Hengrui, Kelun, Johnson & Johnson, Eli Lilly, and More

Aug 02, 2024 11:53 CST Updated 11:53

Johnson & Johnson

Medical Device R&D and Manufacturer

Legend Biotech

Tumor Cell Immunotherapy Developer

Hengrui Pharma

Innovative and High-Quality Pharmaceutical Developer

▎Edited by the WuXi AppTec content team

Since the implementation of China's National Medical Products Administration (NMPA) "Breakthrough Therapy Drug Work Procedure" in 2019, a number of investigational new drugs have made it onto the "breakthrough" drug list each year, and continue to do so.Bring hope to patients with various severe diseasesBetter efficacy or safetyTreatment options.This article will be based onThe breakthrough therapy designation disclosure information of the NMPA Center for Drug Evaluation (CDE) and the NDA submission/priority review records of these drugs,Disc12 "Breakthrough" New Drugs Expected to Be Approved for Marketing in China Within the Next 5 Months (Second Half of 2024)，to see which disease patients these potential breakthrough therapies are expected to benefit.(This article is an incomplete statistic,Only counting new drugs expected to be approved for the first time, excluding new indications)

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Legend Biotech: Cilta-cel (LCAR-B38M CAR-T Cell Autologous Infusion Preparation)

Mechanism of Action: BCMA-Targeted CAR-T Cell Therapy

Indications: Relapsed or Refractory Multiple Myeloma

Cilta-cel is Johnson & Johnson（Johnson & Johnson）A CAR-T therapy targeting BCMA, co-developed with Legend Biotech.In August 2020, the product was included by the CDE as a breakthrough therapy for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM) who have previously received at least three lines of therapy, including at least one proteasome inhibitor and at least one immunomodulatory agent. In January 2023, the product was approved in China forRelapsed or Refractory Multiple MyelomaThe marketing application has been granted priority review.This product previouslyAlreadyApproved for marketing by the U.S. FDA。

According to Legend BiotechData disclosed in April 2023,Cilta-celCARTITUDE-4 Study First Phase 3 Results Show, which canReduced the risk of disease progression/death by 74% in patients.Conducted in ChinaLEGEND-2In a study with ≥5 years of follow-up, the median overall survival was 55.8 months, and 18% of R/R MM patients were symptom-free, increasing the possibility of cure in heavily pretreated patient populations.。

Hengrui Pharma: Ivarmacitinib Tablets (SHR0302）

Mechanism of Action: JAK1 Inhibitor

Indications: Autoimmune diseases (Atopic Dermatitis)

Ivarmacitinib Tablet (ivarmacitinib tablet) isAn orally administered, highly selective JAK1 inhibitorStudies show that JAK1 inhibitors control the condition by modulating various cytokines associated with the pathogenesis of atopic dermatitis.The product is based onPositive Phase 2 Study Results in Atopic Dermatitis Population，Once included in the CDE's breakthrough therapy category。June 2023,Emma Xitinib Tablets Submit the First Marketing Application for Atopic Dermatitis in ChinaAnd subsequently submitted two new indications for adolescents.Treatment of Atopic Dermatitis and Rheumatoid Arthritis。

2024In March, the Phase 3 study of Emmaclitinib for the treatment of moderate to severe atopic dermatitisResults Disclosure: Moderate to severe AD patients treated with Imasitinib for 16 weeks showed significant improvement in skin lesions and itching symptoms. A 52-week long-term follow-up confirmed the sustained efficacy of the drug, with overall good safety.20In January 2018, Hengrui Pharma licensed Emaxitinib to Johnson & Johnson for a total amount of up to $2.23 billion in the United States, the European Union, and Japan.Exclusive RightsLicensed toArcutis Company。

Johnson & Johnson: JNJ-61186372 Injection (amivantamab，Amivantamab Injection

Mechanism of Action: Bispecific Antibody Targeting EGFR and MET

Indications: Non-Small Cell Lung Cancer

Amivantamab isJohnson & JohnsonA bispecific antibody targeting EGFR and MET developed by the company. It not only blocks ligand binding to EGFR and MET and promotes receptor degradation, but also triggers antibody-dependent cellular cytotoxicity. In September 2020, amivantamab was designated as a breakthrough therapy by the CDE for the treatment of patients who progress during or after platinum-based doublet chemotherapy, or who are intolerant to platinum-based chemotherapy.EGFR20Exon insertion mutations in metastatic or surgically unresectableNon-Small Cell Lung CancerPatient. 2023Since then, multiple submissions have been made in China for the Evolocumab Injection.Marketing Application。Amivantamab for the treatment of locally advanced or metastaticEGFRMultiple clinical studies on mutant NSCLC have yielded positive results.

Innovent Biologics/Gensaic Bio: Fuzelase Tablet

Mechanism of Action: KRAS G12C Inhibitor

Indications: Non-small cell lung cancer, Colorectal cancer

Fuzelase Tablets (IBI351，GFH925 Tablets) is a highly efficient oral new molecular entity compound. Innovent Biologics has reached an exclusive global licensing agreement with GenFleet Therapeutics, obtaining exclusive rights to develop and commercialize the product in China as the exclusive partner.Previously,Fuzelase TabletsIncluded in the CDE's breakthrough therapy designation twice, respectively for the treatment ofKRAS G12CPatients with advanced non-small cell lung cancer harboring mutations,KRAS G12CPatients with mutant-type advanced colorectal cancer.

In October 2023, the marketing application for Fuzelerse tablets was accepted by the CDE and included in the priority review, intended for the treatment ofKRAS G12CMutant-type advanced non-small cell lung cancer.Public information shows that this is alsoChina's first KRAS G12C inhibitor to submit an application for marketing.According to the latest research data published by GenFleet Therapeutics in June this year, Fuzelase tablets combined with CetuximabFirst-line treatment for non-small cell lung cancerPhase 2 clinical study data selected for ASCO oral presentation and breakthrough research abstract. The results showedThe objective response rate (ORR) was 81.8%, and the disease control rate (DCR) was 100%.; The majority of evaluable patients (27/33) achieved tumor relief, with one case achieving complete relief and two cases achieving partial relief, with 100% reduction in target lesions observed.

Lilly: Donanemab Injection

Mechanism of Action: β-Amyloid Antibody

Indications: Early Alzheimer's Disease

Donanemab Injection is a product of Eli Lilly and Company（Eli Lilly and Company）A monoclonal antibody developed to bind with the β-amyloid subtype N3pG has been approved by the FDA in July this year.ApprovalTreatmentAdults with Early Symptoms of Alzheimer's Disease (AD). In China,DonanemabWas publicly announced by the CDE as a breakthrough therapy designation in January 2023.For the treatment of early symptomatic Alzheimer's disease, including mild cognitive impairment due to Alzheimer's disease and mild Alzheimer's disease.

In November 2023, the product's new drug marketing application was accepted by the CDE.Priority Review, intended for the treatment of early Alzheimer's disease. According to Lilly's disclosureTheClinical data, pivotal Phase 3 study shows, donanemabCompared with placeboSlowed cognitive and functional decline by up to 35% and reduced the risk of disease progression by up to 39% over 18 months; compared to the start of the study,Monthly 30-minute infusions reduced amyloid plaques by an average of 84%.

Kelun BioTech: Lukansatuzumab

Mechanism of Action: TROP2-Targeted ADC

Indications: Triple-negative breast cancer, etc.

Lukang Satuzumab ( Injectable SKB264 ) is a TROP2-targeted ADC developed by Kelun Biotech.The product has been included in the CDE's breakthrough therapy category four times., respectively targeting: locally advanced or metastatic triple-negative breast cancer (TNBC), locally advanced or metastaticEGFRMutant non-small cell lung cancer, locally advanced or metastatic HR-positive/HER2-negative breast cancer, PD-L1-negative triple-negative breast cancer.In November 2023, the drug's marketing application was accepted by the CDE.Priority Review, used forAdult patients with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC) who have previously received at least two systemic treatments (at least one of which was for advanced or metastatic stage).。

ConnieMab: CM310 Recombinant Humanized Monoclonal Antibody Injection (Sipuchimab)

Mechanism of Action:IL-4Rα-Targeted Monoclonal Antibody

Indications:Atopic Dermatitis

Sipuleucel-T is a targetedInterleukin-4 Receptor Alpha Subunit（IL-4Rα) is a highly efficient, humanized monoclonal antibody. In June 2022, the productUsed forStudy on the Treatment of Moderate to Severe Atopic Dermatitis Included in the CDE Breakthrough Therapy Designation. In November 2023, the product's marketing application was includedPriority review, for the treatment of conditions not well-controlled by topical medications or unsuitable for topical medication treatmentAdult Moderate to Severe Atopic Dermatitis. In addition, this productTargetingIndications for seasonal allergic rhinitis, as well as chronic rhinosinusitis with nasal polyps (CRSwNP), alsoThe listing application has been submitted.

Tainuomabo: TNM002 Injection

Mechanism of Action: Recombinant Natural Fully Human Monoclonal Antibody Against Tetanus Toxin

Indications: Prevention of tetanus

TNM002 Injection is developed by Tanomab.Recombinant Anti-Tetanus Toxin Fully Human Monoclonal Antibody Drug. The product was included in the breakthrough therapy category by the CDE in February 2022 forPrevent TetanusIn November 2023, the marketing application for TNM002 was granted priority review by the CDE, intended for use inEmergency Prevention of Tetanus in AdultsExisting clinical data shows that, after administration, the level of anti-tetanus antibodies in the body can quickly reach a high protective level against tetanus and maintain this protection for an extended period.。

Yifang Biotech/Zhengda Tianqing: Geshurexi Tablets

Mechanism of Action:KRAS G12CSelective Inhibitor

Indications: Non-small cell lung cancer, Colorectal cancer, Pancreatic cancer

Garsorasib (garsorasib, D-1553 tablets) is a novel and highly effective drug developed by Innovent Biologics.KRAS G12CSelective Inhibitor. Yifang Biotech has reached a collaboration with Zhengda Tianqing, granting the latter exclusive rights to develop, register, manufacture, and commercialize the product in mainland China during the agreement period. The product has been designated as a breakthrough therapy by the CDE three times, targeting:KRAS G12CMutant Non-Small Cell Lung Cancer, KRAS G12C Colorectal Cancer,KRAS G12CMutant Pancreatic Ductal Adenocarcinoma.

December 2023,Geshurui Xi'sMarketing application included by CDEPriority Review, applicable toKRAS G12CTreatment of locally advanced or metastatic non-small cell lung cancer with mutations.Geshurexi TreatmentKRAS G12CEarly Clinical Results of Mutated Non-Small Cell Lung Cancer Show That,The objective response rate (ORR) was 40.5%, the disease control rate (DCR) was 91.9%, and the median progression-free survival (mPFS) was 8.2 months.。

BeiGene：Zenidatamab for Injection

Mechanism of Action:HER2 Bispecific Antibody

Indications:HER2-highly expressed unresectable locally advanced or metastatic biliary tract cancer

Zanidatamab (zanidatamab, ZW25) isA HER2-targeted bispecific antibody that can simultaneously bind to two non-overlapping HER2 epitopes。This drug originated from a 2018 collaboration between BeiGene and Zymeworks.Authorization cooperation of up to approximately 430 million US dollars,BeiGene has exclusive rights in Asia (excluding Japan), Australia, and New Zealand. In February 2022, the product was included in the breakthrough therapy designation by the CDE.Monotherapy for HER2-positive locally advanced unresectable or metastatic biliary tract cancer (BTC) that has failed prior systemic chemotherapy。

2023November,Zenidatamab TreatmentHER2-Overexpressing Biliary Tract CancerThe patient's marketing application was submitted to the CDEIncluded in Priority ReviewIn a global Phase 2b study for this indication, the confirmed objective response rate (cORR) was 41%, with a median duration of response (DOR) of 12.9 months; among 33 responders, 49% had sustained responses, and 82% had a DOR ≥ 16 weeks.

Innovent Biologics/Baoyuan Pharma: Tarextumab Capsules

Mechanism of Action: A New Generation of ROS1 Tyrosine Kinase Inhibitor (TKI)

Indications: Non-Small Cell Lung Cancer

Telisotuzumab (AB-106）Is aNew Generation OralSelective ROS1 Inhibitor, Innovent Biologics andBaoyuan Pharma (Acquired by Nuvation Bio)Jointly responsible for the development and commercialization of the product in the Greater China region. Previously, two indications of this product have been included by the CDE as breakthrough therapies, respectively for: carryingROS1Fusion gene andAfter ROS1-TKI treatment failureNon-small cell lung cancer, carryingROS1Fusion Gene andUntreated with ROS1-TKINon-small cell lung cancer.

Currently, the marketing applications for these two indications of Taletrectinib have both been included in the priority review by the CDE.According to previously published news reports, in the Phase 2 TRUST-I clinical study,TKI-naive patientsAndCrizotinib Treatment FailureROS1-positive NSCLC patientsObjective Response Rate (ORR) was91%And 52%。In addition, heRetini in patients with measurable brain metastases and acquired resistance mutations (includingG2032R) in patients, continued to demonstrate potent antitumor activity.

Hengrui Pharma: Famitinib

Mechanism of Action: Multi-target Tyrosine Kinase Inhibitor

Indications: Cervical cancer, Non-small cell lung cancer

Famitinib malate capsules are a small-molecule multi-target tyrosine kinase inhibitor independently developed by Hengrui Pharma. It exhibits inhibitory activity against various receptor tyrosine kinases and belongs to the category of multi-target anti-angiogenic targeted drugs. The drug has been included in the CDE's breakthrough therapy designation twice, with the indication being its combination with Camrelizumab for treating patients who have failed first-line or higher-level treatments.Recurrent and Metastatic Cervical Cancer, First-line treatment for tumors with PD-L1 expression positive (TPS≥1%) and withoutEGFR/ALKGenetic AbnormalitiesRecurrent or Metastatic Non-Small Cell Lung Cancer。

In December 2023, the new drug application for Camrelizumab combined with Famitinib capsules for the treatment of patients with recurrent or metastatic cervical cancer who failed prior platinum-based chemotherapy was accepted by the CDE. In the pivotal Phase 2 clinical study, compared to Camrelizumab monotherapy, this combination therapy significantly improved the objective response rate (ORR), which reached 41%, with rapid onset and prolonged duration of response.

References:

[1] Website of the Center for Drug Evaluation, National Medical Products Administration of China. From https://www.cde.org.cn/main/xxgk/listpage/da6efd086c099b7fc949121166f0130c

[2] Official websites of various companies and public materials

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