Multiple Breakthrough Oncology and Rare Disease Drugs Approved in China Q1 2024

　　【Pharmaceutical Network Industry DynamicsAccording to the information on the website of the National Medical Products Administration, in the first quarter of 2024, the National Medical Products Administration approved 20 new drugs, including several significant cancer drugs and new rare disease medications.
 

For example, in the field of oncology, on March 15, the website of the National Medical Products Administration (NMPA) disclosed that through the priority review and approval process, the conditional approval was granted for the marketing of Keluo Ping (Tolametinib Capsules), a Class 1 innovative drug submitted by Keyo Pharmaceutical. This medicine is suitable for patients with advanced melanoma who have NRAS mutations and have failed anti-PD-1/PD-L1 treatment. It is reported that Tolametinib is a selective Mitogen-Activated Protein Kinase 1 and 2 (MEK1/2) inhibitor jointly developed by Keyo Pharmaceutical and Tianjin Binjiang Drug Research and Development Co., Ltd. This drug is China's first MEK inhibitor and also the world’s first targeted drug approved for the indication of NRAS-mutated advanced melanoma.
 

CARsgen Therapeutics' Zevor-cel Injection, which was approved for marketing on March 1st, is also noteworthy. It is an autologous CAR-T product targeting B-cell maturation antigen (BCMA) for the treatment of relapsed or refractory multiple myeloma, offering a new treatment option for patients. Reportedly, Zevor-cel Injection received orphan drug designation from the U.S. FDA in 2019 and was granted PRIME and orphan drug designations by the European Medicines Agency in 2019 and 2020 respectively.
 

In the field of rare disease treatment, three new rare disease drugs—canfolimab, zinc acetate tablets, and trientine hydrochloride tablets—were approved for marketing by the National Medical Products Administration in the first quarter, all through the priority review process.
 

Among them, Roche's Crovalimab is a humanized anti-complement protein C5 monoclonal antibody, used for adult and adolescent patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not received complement inhibitor treatment. Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired disease of hematopoietic stem cells caused by somatic gene mutations in the human body, mainly characterized by chronic intravascular hemolysis, thrombosis, bone marrow failure, etc., and belongs to a rare blood system disease.
 

Jiangsu Nobelpharma Co., Ltd.'s zinc acetate tablets were approved for marketing on February 19 and will be used to treat the rare disease Wilson's disease. Hepatolenticular degeneration is a rare autosomal recessive genetic disorder caused by abnormal copper deposition in various tissues of the body. Its characteristic clinical manifestations include acute or chronic liver disease symptoms, progressively worsening extrapyramidal symptoms, psychiatric symptoms, and corneal pigmentation rings. Other common symptoms include hemolytic anemia, osteoarticular symptoms, and hematuria. This disease has been included in the first batch of the rare disease catalog.
 

On January 5, the targeted treatment drug for Wilson's disease, trientine hydrochloride tablets, was approved for marketing. Wilson’s disease is a rare condition that may lead to liver damage, neurological involvement, as well as ocular lesions, kidney damage, osteoarthritis, and various other clinical manifestations. This disease was included in the list of rare diseases published by China's National Health Commission as early as 2018.
 

With the support of favorable policies, rare disease drugs in China are accelerating their market entry to meet the treatment needs of more patients with rare diseases. Statistics show that in 2023, a total of 27 rare disease drugs were approved for marketing in China, including Ozanimod Hydrochloride Capsules, Nitisinone Capsules/Oral Suspension, and Selumetinib Sulfate Capsules, among others. These drugs cover the treatment of 22 rare diseases, such as multiple sclerosis, type 1 hereditary tyrosinemia, and type 1 neurofibromatosis.
 

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