Six Chinese-Developed Drugs Receive FDA Breakthrough Therapy Designation, Four Targeting Cancers

According to publicly available data, among the new drugs granted FDA Breakthrough Therapy Designation throughout 2024, at least six were developed by companies in China.(Incomplete statistics, excluding products for which only China development rights were acquired), four of which are used for cancer treatment.

Breakthrough Therapy Designation is an important review process by the U.S. FDA to expedite the development and review of drugs, primarily applicable to breakthrough innovative drugs for the treatment of serious or life-threatening diseases.。Breakthrough Therapy DesignationEmphasizing the breakthrough in clinical safety and efficacy of existing therapies, demonstrating significant advantages and breakthroughs in clinical practice.，Receiving this designation means that the FDA recognizes its therapeutic potential,Is expected to accelerate the R&D process.

This article will review the research and development progress of these six new drugs based on publicly available information, examining their breakthroughs in clinical settings and the potential hope they may bring to patients with certain diseases.

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Dizal: Sunvozertinib

Mechanism of Action: EGFR-TKI

Indications: Non-Small Cell Lung Cancer

In April 2024, Dizal Pharmaceutical announced that the FDA granted Breakthrough Therapy Designation to its self-developed novel lung cancer targeted drug, Sunvozertinib, forFirst-line treatment with epidermal growth factor receptor (EGFRPatients with advanced non-small cell lung cancer (NSCLC) harboring exon 20 insertion (exon20ins) mutationsEarlier, the product was used for second-line/later-line treatment of E.GFR exon20insThe indication for NSCLC has also been granted Breakthrough Therapy Designation by the FDA.

Dizal's Sunvozertinib is an oral, irreversible, highly selective EGFR-TKI targeting multiple EGFR mutation subtypes, which has been approved in China for previously treated patients.EGFR exon20insMutant advanced NSCLC.Dizal is also advancing the launch process of Sunvozertinib in the United States, and the new drug application for this product has been granted by the FDA in January 2025.Priority Review Status，Used when disease progression occurs during or after previous platinum-based chemotherapy, and confirmed by an FDA-approved kit.exon20insAdult patients with locally advanced or metastatic non-small cell lung cancer。

LEPU BIOPHARMA: Vicotuzumab

Mechanism of Action: EGFR-Targeted Antibody-Drug Conjugate (ADC)

Indications: Nasopharyngeal Cancer

In August 2024, LEPU BIOPHARMA announced that its EGFR-targeted ADC, Vericetuzumab (MRG003), received FDA approval.Breakthrough Therapy Designation, for the treatment ofRecurrent or Metastatic Nasopharyngeal Carcinoma (R/M NPC)。Currently,Vibegron Monoclonal AntibodyAlready in ChinaSubmission for Market Approval, and has been included by NMPA in the priority review and breakthrough therapy category for patients with recurrent/metastatic nasopharyngeal carcinoma who have failed at least two prior lines of systemic chemotherapy and PD-1/PD-L1 inhibitor treatments.

MRG003 is composed of an EGFR-targeted monoclonal antibody conjugated with the potent microtubule inhibitor MMAE molecule through a vc linker. The molecular design of this drug has the potential to expand to a broader range of cancer types with EGFR overexpression and has the potential to overcome various types of resistance caused by common mutations. This productPhase 1/2 Clinical Study Results of Combined PD-1 Inhibitor Putelimab in EGFR-positive Solid Tumor Patients Show9 evaluableAfter receiving first-line treatment with PD-1 plus platinum-based chemotherapyEGFR-positive nasopharyngeal cancer patientsIn China,Objective Response Rate (ORR)The ORR was 77.8%, and the disease control rate (DCR) was 100%.。

Hansoh Pharma, GSK: HS-20093

Mechanism of Action: B7-H3 Targeting ADC

Indications: Small Cell Lung Cancer

In August 2024, Hansoh Pharma and GlaxoSmithKline (GSK) announced that the FDA grantedHS-20093(GSK5764227) Breakthrough Therapy Designation for the treatment ofPatients with recurrent or refractory extensive-stage small cell lung cancer (ES-SCLC) who progressed during or after platinum-based chemotherapy。HS-20093 is a B7-H3-targeted ADC covalently linked by a fully humanized B7-H3 monoclonal antibody and a topoisomerase inhibitor (TOPOi) payload. Hansoh Pharma previously entered into an exclusive licensing agreement with GSK worth over $1.7 billion, granting GSK a global exclusive license (excluding mainland China, Hong Kong, Macao, and Taiwan) for the development, manufacturing, and commercialization of this product.

According to the previous press release from Hansoh Pharma, the FDA's Breakthrough Therapy designation was obtained.Phase 1 ARTEMIS-001 First-in-Human Clinical TrialData support. According to the results published by researchers at the 2024 World Conference on Lung Cancer (WCLC), for the ES-SCLC patient cohort,The DCR of HS-20093 in the 8.0mg/kg and 10.0mg/kg dose groups were 80.6% and 95.5%, respectively.Currently,HS-20093Has entered the international multi-center phase 3 clinical research stage for recurrent small cell lung cancer (ARTEMIS-008).

Simcere, Ningdan New Drug: Edaravone Dextrorphan Sublingual Tablets

Mechanism of Action: Neuroprotective Agent

Indications: Acute Ischemic Stroke (AIS)

In September 2024, Simcere announced that the sublingual tablet of Edaravone and Dextrorphan (Xianbi Xin sublingual tablet), co-developed with Ningdan New Drug, received FDA Breakthrough Therapy Designation.For the treatment of Acute Ischemic Stroke (AIS)。Edaravone Dexborneol Sublingual Tablets are a type of neuroprotective agent containing two active ingredients, edaravone and dexborneol, with antioxidant, anti-inflammatory, and synergistic effects. In December 2024, this therapy was approved in China.Approved for Marketing, used to improve neurological symptoms, activities of daily living, and functional impairment caused by acute ischemic stroke.

According to the press release from Simcere, the breakthrough therapy designation was based on the significant improvement in efficacy indicators demonstrated by Edaravone Dextrorphan Sublingual Tablets in earlier clinical studies. Data from a multi-center Phase 3 clinical study conducted in China for the treatment of AIS showed that, compared to placebo,The sublingual tablet significantly improved neurological recovery and independent living ability in AIS patients after treatment, achieving the expected efficacy endpoint with good safety.。

Huahui Anjian: Libevirtamab

Mechanism of Action: Hepatitis B Virus Surface Large Envelope Protein Pre-S1 (PreS1) Region-Targeting Neutralizing Antibody

Indications: Chronic Hepatitis D Virus (HDV) Infection

In November 2024, Huahui Anjian announced that its Libevirtamab (HH-003) had been granted Breakthrough Therapy Designation by the FDA for the treatment ofChronic HDV Infection。Libevetumab is a neutralizing antibody developed by Huahui Anjian Company that targets the PreS1 region of the large envelope protein on the surface of the hepatitis B virus.It specifically binds to the PreS1 region on the surface of HBV/HDV, blocking the binding of HBV, HDV, and their receptor NTCP, thereby preventing viral entry into hepatocytes and neutralizing viral infection.。The therapy has currently been submitted for marketing approval in China and has been included in the priority review by the NMPA, with the indication for chronic HDV infection.

Huahui Anjian previously announced the Phase 1b clinical trial data of HH-003 in chronic hepatitis B virus (HBV) infected patients for the first time at the 2022 American Association for the Study of Liver Diseases (AASLD) Annual Meeting. The study showed that HH-003 demonstrated good safety and antiviral activity in the research.In the immune clearance group subjects, a clear reduction in HBV DNA and hepatitis B surface antigen was observed after three doses of HH-003 administered intravenously every two weeks.。

Kelun-Biotech,Merck & Co., Inc. (MSD): Lucantusumab

Mechanism of Action: TROP2-Targeted ADC

Indications: Non-squamous Non-small Cell Lung Cancer

Lukang SatuzumabIt is an ADC jointly developed by Merck and Kelun Biotech.Composed of a humanized monoclonal antibody targeting TROP2, an enzymatically cleavable linker, and a novel topoisomerase I inhibitor.. In November 2024, the therapy in ChinaApproved for Marketing, for the treatment of triple-negative breast cancer.

In December 2024, Merck announced that the FDA had granted its investigational ADC drugLukansatuzumab (sacituzumab tirumotecan，sac-TMT) Breakthrough Therapy Designation, for the treatment ofCarryEGFRPatients with advanced or metastatic non-squamous NSCLC harboring mutations, whose diseases progressed after receiving tyrosine kinase inhibitors (TKI) and platinum-based chemotherapy.This designation is based on the Phase 2 expansion cohort of a Phase 1/2 clinical trial.EGFRMutant NSCLC Patient Data。As of the data as of February 9, 2023, the median follow-up time was 11.5 months. InEGFRIn mutated patients, the ORR was 60%.DCR is 100% (20/20), The median duration of response was 9.3 months, and the median progression-free survival was 11.1 months,The overall survival rate at 12 months was 80.7%.。

We hope that the subsequent development of these new drugs in research will proceed smoothly, and bring new treatment options to more patients as soon as possible.