▎WuXi AppTec Content Team Report

According to publicly available data, in 2024, the U.S. FDA approved at least 33 new drugs for rare diseases. In China, at least 17 new drugs for rare diseases were approved in 2024.Approved for Marketingcovering more than ten types of rare diseases, including Wilson's disease, paroxysmal nocturnal hemoglobinuria, hemophilia, "ALS", and Duchenne muscular dystrophy. These new drugs have brought new hope to countless patients and families.

The R&D of rare disease drugs is accelerating globally.In 2024, the U.S. FDA granted orphan drug designation to more than 88 new drug projects. These drugs cover a variety of rare disease types, including Duchenne muscular dystrophy, primary biliary cholangitis, familial Mediterranean fever, and Wilson's disease. Among them,At least 35 new drug projects from Chinese companies have received this qualification, hitting a record high in recent years.。

According to statistics from the World Health Organization, there are approximately 6,000 rare diseases globally, with a total patient population exceeding 300 million. However, currently, less than 5% of rare diseases have available treatments. With advancements in science and technology and increased industry investment, the development of new drugs for rare diseases has entered an unprecedented period of activity. Data shows that in the past five years, over 50% of the new drugs approved by the FDA are for treating rare diseases and rare cancers.

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The development of orphan drugs cannot be separated from the promotion of innovative technologies.In terms of drug types, new drugs granted FDA orphan drug designation in 2024 encompass a variety of categories, including small molecules, antibodies, peptides, AAV gene therapies, CAR-T cell therapies, antisense oligonucleotide therapies, antibody-drug conjugates (ADCs), stem cell therapies, oncolytic viruses, and more.

Among them, there are 39 small-molecule drugs, accounting for more than 40%. Among these, there are some well-known products under research familiar to people in China's industry. For example, BeOne Medicines.BTK Inhibitor Zanubrutinib Proposed for TreatmentMembranous Nephropathy; and Hesmile PharmaceuticalsSmall-molecule FGFR4 inhibitor Epagortinib proposed for treatmentHepatocellular Carcinoma; JACOB'sKRAS G12C Inhibitor Golerecept for TreatmentPancreatic Cancer; Huahao Zhongtian'sUtidelone Injection for TreatmentBrain Metastasis of Breast CancerThese drugs not only demonstrate the advancement of science and technology but also provide more potential treatment options for patients with rare diseases worldwide.

New molecular drugs represented by peptides, oligonucleotides, cell and gene therapies are becoming a significant direction in the treatment of rare diseases. For example, Lisata Therapeutics'Cyclic peptide therapy certepetide proposed for the treatment of cholangiocarcinoma; RactigenSmall Activating RNA Drug RAG-18 for the Treatment of Duchenne Muscular Dystrophy; Ionis Pharmaceuticals'Antisense Oligonucleotide Therapy Tryngolza Proposed for Reducing Triglycerides in Patients with Familial Chylomicronemia Syndrome. The development of these new drugs marks that the research and development of new drugs for rare diseases has entered a brand-new era of new molecules.

However, the development of drugs for rare diseases is not an easy task. Small patient populations, high research and development costs, and significant technical challenges have always been major obstacles faced by the industry. It requires the joint efforts of an innovative biopharmaceutical ecosystem to address these challenges. For example, contract research and manufacturing organizations (CXOs) are also important forces driving innovation in drugs for rare diseases.

As an innovator, a trusted partner of customers, and a significant contributor to the global health industry, WuXi AppTec has provided strong support for the development of new drugs for rare diseases through its integrated, end-to-end CRDMO platform for new drug research and development over the years. By leveraging technological innovation, efficient collaboration, and full-chain services, WuXi AppTec has empowered its partners to bring more treatment options to rare disease patients worldwide. According to publicly available data,Among the 33 new orphan drugs approved by the FDA in 2024, at least five received research and development support from WuXi AppTec. Among the 88 new drug projects granted FDA orphan drug designation, WuXi AppTec supported 19, accounting for 21.6%.。This is equivalent to one out of every five orphan drug projects receiving support from WuXi AppTec.

The development of drugs for rare diseases is a long and complex journey, from early discoveries in the laboratory to ultimately benefiting patients, requiring overcoming countless technical challenges. Throughout this process, WuXi AppTec, with its integrated technology platform and full-chain services—from drug discovery, preclinical research to process development, manufacturing—has provided comprehensive support to its partners.

The research and development of drugs for rare diseases often begins with an in-depth understanding of the disease mechanism. Public information shows that WuXi AppTec's biology business launched a rare disease platform as early as 2016, focusing on the early stages of rare disease drug development. The platform covers the entire route of rare disease drug research and development, providing one-stop services including the construction of rare disease animal models, animal breeding, and in vivo and in vitro pharmacological efficacy studies.The platform has rapidly developed animal models for dozens of rare diseases, including hemophilia A, sickle cell anemia, spinal muscular atrophy, and Duchenne muscular dystrophy. These models cover research hotspots such as blood disorders, metabolic diseases, immune-related diseases, and psychiatric conditions, supporting the pharmacological evaluation of rare disease drugs.。

The development of orphan drugs not only requires scientific breakthroughs but also efficient process development capabilities to address the complex challenges of clinical stages. WuXi AppTec's subsidiary, STA Pharmaceutical, has demonstrated strong technical expertise in this field. Based on the concept of Quality by Design (QbD), STA Pharmaceutical's R&D team can rapidly advance drug process development and ensure safe scale-up, significantly reducing costs while guaranteeing product quality.

Moreover, as new molecular drugs represented by oligonucleotides and peptides become a significant direction in the treatment of rare diseases, WuXi AppTec has long been strategically positioned in this area, with its subsidiary...WuXi TIDES: A New Molecular Business Platform Focused on Oligonucleotides, Peptides, and Related Chemical Conjugate Drugs, demonstrating powerful empowerment capabilities and providing significant support for the development of drugs for rare diseases.

Taking oligonucleotide drugs as an example, siRNA drugs have high development difficulty due to issues such as poor targeting and low stability.WuXi TIDES Platform Effectively Solves the siRNA Delivery Challenge Through N-Acetylgalactosamine (GalNAc) Covalent Conjugation Technology, Becoming the Industry’s Mainstream Solution.The platform also has comprehensive oligonucleotide conjugation capabilities, customized GalNAc molecule synthesis capabilities, and integrated CMC services, which can advance candidate compounds to the IND stage in a short period of time.

For example, the WuXi TIDES platform once advanced a siRNA-GalNAc candidate drug for the treatment of a rare disease to the IND stage in just 10 months, demonstrating its highly efficient technical execution and collaboration capabilities. This ability to rapidly advance has saved precious time for the development of drugs targeting rare diseases.Currently, WuXi TIDES is empowering the development of more than 20 new drugs for rare disease treatments, including complex conjugated drugs such as oligonucleotide-peptide conjugates, PDCs, and RDCs, allowing more patients to potentially benefit from new molecular therapies.。

In addition, WuXi AppTec's Testing Division continues to empower customers in conducting IND and non-clinical research for rare disease drug development.

These platforms jointly form a powerful engine for WuXi AppTec in the field of rare disease drug research and development, helping partners bring more treatment options to rare disease patients worldwide. Currently, WuXi AppTec is actively supporting the advancement of more partners' rare disease drug pipelines globally.

WuXi AppTec's empowerment extends beyond technical support to its global collaborative network. Through close cooperation with global pharmaceutical companies, biotech firms, and research institutions, WuXi AppTec continuously facilitates the global reach of new orphan drugs. For instance, among the 35 new drug projects from Chinese companies that received FDA Orphan Drug Designation in 2024, WuXi AppTec supported five of them.

The Race Against Time: Development of Rare Disease Drugs