Two New Chinese Innovative Drugs Receive FDA Orphan Drug Designation

　　【Pharmaceutical Network Product InformationSince November, several Chinese pharmaceutical companies, including Ractigen and Leads Biolabs, have announced that their related innovative drugs have been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA).
 

Ractigen announced on November 19, 2024, that its self-developed FUS gene-targeted small interfering RNA (siRNA) therapy, RAG-21, has successfully received orphan drug designation from the FDA for the treatment of Amyotrophic Lateral Sclerosis (ALS). This marks the company’s second FDA orphan drug certification in the ALS field, following the previous certification of RAG-17 for SOD1-ALS.
 

Data show that ALS is a severe neurodegenerative disease without a cure, selectively damaging motor neurons in the cortex and spinal cord. RAG-21 is an siRNA therapy specifically targeting FUS gene mutations, effectively reducing FUS protein levels and addressing the root cause of motor neuron degeneration. The drug functions through the RNA interference (RNAi) mechanism, alleviating FUS protein mislocalization and abnormal aggregation by lowering FUS protein levels.
 

Preclinical studies show that RAG-21 has a significant knockdown effect and good safety. As a novel RNA therapy, RAG-21 brings new hope for improving the prognosis of FUS-ALS patients, who currently have no disease-modifying treatments available.
 

On November 1, Leads Biolabs announced that its self-developed, globally intellectual property-owned Class 1 new drug LBL-034 has been granted Orphan Drug Designation by the FDA for the treatment of multiple myeloma (MM).
 

Data show that multiple myeloma (MM) is a malignant plasma cell disorder caused by the abnormal proliferation of clonal plasma cells, accounting for approximately 10% of hematologic malignancies and about 1% of all neoplastic diseases. LBL-034 is a next-generation humanized bispecific T-cell engager antibody targeting GPRC5D and CD3, developed using Leads Biolabs' proprietary CD3 bispecific antibody technology platform "LeadsBodyTM." LBL-034 selectively binds to T cells only in the presence of GPRC5D+ cells, thereby conditionally activating T cells within the tumor microenvironment where GPRC5D is expressed. In cells with high, medium, and low GPRC5D expression, LBL-034 consistently demonstrates excellent targeted tumor cell killing effects and robust dose-dependent antitumor activity, showing potential as a best-in-class anticancer therapeutic option.
 

Leads Biolabs stated that the Orphan Drug Designation is a high recognition by the FDA of the significant potential of Leads Biolabs' LBL-034 in the treatment of MM. This will help accelerate the subsequent clinical development and market launch of LBL-034 globally.
 

It is reported that the Orphan Drug Designation is established by the FDA to encourage the development of drugs for rare disease treatments. It provides a series of incentives for new drug development, including but not limited to: tax credits for clinical trial expenses; special guidance from the FDA on all phases of clinical research; exemption from new drug application fees; and seven years of market exclusivity after approval. The Orphan Drug Designation granted by the FDA applies to drugs for rare diseases with fewer than 200,000 patients in the United States.
 

According to the review, since this year, several innovative drugs produced in China have received this qualification. For instance, in September, NineSky Biopharma announced that its self-developed adeno-associated virus (AAV) gene therapy drug SKG1108, a breakthrough gene therapy for treating retinitis pigmentosa (RP), has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). In May, Mabwell announced that its self-developed Nectin-4-targeting ADC innovative drug (research code: 9MW2821) was granted Orphan Drug Designation by the FDA for the treatment of esophageal cancer. In March, Huahao Zhongtian announced that its core product, Utidelone Injection, received Orphan Drug Designation from the U.S. FDA for the treatment of brain metastases in breast cancer. In January, Boan Biotech announced that two of its self-developed Claudin18.2-targeting research drugs, BA1105 and BA1301, were respectively granted Orphan Drug Designation by the FDA for the treatment of gastric cancer (gastroesophageal junction cancer).
 

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