Nine Rare Disease Drugs Expected to Gain Approval in 2024, with Huadong Medicine, Tenacia, and Ipsen Poised for Market Entry

　　【Pharmaceutical Network Industry DynamicsAccording to data statistics, five new orphan drugs have been approved for marketing this year, bringing new treatment options for patients with related rare diseases. Looking forward to 2024, nine more orphan drugs are expected to be approved for marketing, including Huadong Medicine's Rilonacept for Injection, Takeda Pharmaceutical's Vonicog alfa for Injection, Novartis' Iptacopan Hydrochloride Capsules, Yuanyi Biotechnology's Ganaxolone Oral Suspension, Novo Nordisk's Pegnivacogin α for Injection, Recordati's Orazamide Film-Coated Tablets, Ipsen's Odevixibat Capsules, Beijing Hanyou Pharmaceutical Technology's Vamorolone Oral Suspension, and MSD's Belzutifan Tablets.
 

Among them, Rilonacept for injection is a recombinant dimeric fusion protein that can block the signaling of interleukin-1α (IL-1α) and interleukin-1β (IL-1β). On the evening of March 1, 2024, Huadong Medicine Co., Ltd. announced that the marketing registration application for Rilonacept for injection, introduced by its wholly-owned subsidiary Zhongmei Huadong from Kiniksa in the United States, has been officially accepted by the National Medical Products Administration. It is reported that Rilonacept for injection, approved by the U.S. Food and Drug Administration (FDA), is indicated for the treatment of recurrent pericarditis in patients aged 12 years and above. In China, it has been included in the list of urgently needed overseas new drugs for clinical use. The treatments for recurrent pericarditis and Cryopyrin-Associated Periodic Syndromes (CAPS) are expected to be approved for marketing within this year.
 

Vonicog alfa for injection is a recombinant von Willebrand factor developed by Takeda Pharmaceutical Company Limited. In January 2023, the marketing application of this product was granted priority review by the CDE for the indication: treatment of adult patients diagnosed with von Willebrand disease, including on-demand treatment and control of bleeding episodes, as well as perioperative bleeding management.
 

On June 5, 2023, the website of the Center for Drug Evaluation (CDE) under the National Medical Products Administration (NMPA) showed that Novartis' application for iptacopan capsules (iptacopan) is proposed to be included in the priority review for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH). According to publicly available information, this drug is a potential "first-in-class" oral complement pathway factor B inhibitor, iptacopan (LNP023), being developed by Novartis. It was previously designated as a breakthrough therapy by the CDE for the treatment of PNH. Data shows that PNH is a rare blood disease mediated by the complement system, with clinical manifestations mainly including anemia, paroxysmal hemoglobinuria, bone marrow failure, and thrombosis.
 

According to the CDE website in September 2023, the marketing application for Yuanyi Biotechnology (Tenacia)'s Class 5.1 chemical drug Ganaxolone oral suspension has been accepted. The drug was licensed by Yuanyi Biotechnology from Marinus and was proposed for priority review on September 8. It is reported that Ganaxolone, originally developed by Marinus, is a positive allosteric modulator targeting GABAA receptors. By acting on synaptic and extrasynaptic GABAA receptors in neurons, it achieves antiepileptic and anxiolytic activity.
 

On July 25, 2023, according to the CDE website, Novo Nordisk (NVO.US) submitted an application for the market approval of Pegylated Recombinant Factor VIII (Pegnivacog alfa) in China. The drug is a long-acting recombinant Factor VIII (FVIII) developed by Novo Nordisk based on Turoctocog alfa. It was first approved by the FDA in February 2019 for prophylactic and acute treatment in adult and pediatric patients with Hemophilia A. In June and September of the same year, it received approval in the EU and Japan, respectively.
 

On October 16, 2023, the CDE website announced that the osilodrostat phosphate film-coated tablets submitted by Rui Kang Di Pharmaceutical, the wholly-owned Chinese subsidiary of Recordati Group, are proposed for inclusion in the priority review. The drug is intended for the treatment of adult endogenous Cushing's syndrome (also known as: endogenous hypercortisolism). Public information shows that this is a novel oral cortisol synthesis inhibitor, Isturisa (osilodrostat), which has been approved in the European Union, the United States, and other regions for the treatment of adult Cushing's disease. In July 2019, Recordati acquired the global development and commercialization rights of osilodrostat phosphate film-coated tablets from Novartis.
 

On March 22, 2024, the CDE website announced that the marketing application for odevixibat capsules submitted by Albireo and Ipsen has been accepted. Public information indicates that odevixibat is a potent, non-systemically absorbed ileal bile acid transporter (IBAT) inhibitor. The product has been granted priority review by the CDE for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged six months or older. In March 2023, Ipsen announced the completion of its acquisition of Albireo, thereby gaining the latter's rare disease product (brand name: Bylvay).
 

In addition, on February 18, 2024, the CDE website showed that the Vamorolone Oral Suspension submitted by Shu Fang Medicine was proposed to be included in the priority review, with the proposed indication being: for the treatment of Duchenne Muscular Dystrophy (DMD) patients aged four years and above. On February 21, 2024, the CDE website indicated that MSD's Belzutifan Tablets were proposed to be included in the priority review, with indications for von Hippel-Lindau (VHL) disease-related Renal Cell Carcinoma (RCC), Central Nervous System (CNS) Hemangioblastoma, or Pancreatic Neuroendocrine Tumors (pNET).
 

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