Five Orphan Drugs Approved by U.S. FDA in October: Zilucoplan, Omvoh, Vamorolone, Entrectinib, and Toripalimab

　　【Pharmaceutical Network Product Information"Orphan drugs," also known as rare drugs, refer to medicines used for the prevention, treatment, and diagnosis of rare diseases, and their research, development, and market approval are closely watched by the industry. According to statistics, in October, the U.S. Food and Drug Administration (FDA) approved a total of 17 new drugs for marketing, including five "orphan drugs": zilucoplan from UCB, Omvoh from Eli Lilly, vamorolone from Swiss Santhera Pharmaceuticals, entrectinib from Genentech, and toripalimab from Junshi Biosciences.
 

Specifically, UCB's Zilucoplan is a complement C5 inhibitor, primarily used to treat generalized myasthenia gravis (gMG) in adults who are positive for anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibodies. Previously, the only drug approved by the FDA for the treatment of generalized myasthenia gravis was Argenx’s FcRn antibody Efgartigimod. Additionally, Argenx’s subcutaneous injection form of Efgartigimod was approved by the FDA on June 20 this year.
 

Eli Lilly's Omvoh received FDA approval in October for use in adult patients with ulcerative colitis. The drug has already been approved for marketing this year in Japan, the EU, and the UK. It is reported that Omvoh can bind to the p19 subunit of IL-23, blocking IL-23-mediated inflammatory responses. Inflammation caused by overactivation of the IL-23 pathway plays a key role in the pathogenesis of UC. In addition to ulcerative colitis, the drug is also being explored for the treatment of moderate to severe active Crohn's disease.
 

Vamorolone, developed by Swiss Santhera Pharmaceuticals, has been granted orphan drug designation for the treatment of Duchenne Muscular Dystrophy (DMD) in both the United States and Europe. It has also received FDA Fast Track designation and Rare Pediatric Disease designation. Santhera Pharmaceuticals has licensed the rights of Vamorolone in North America and China to Catalyst Pharmaceuticals and Sperogenix Med, respectively. According to Santhera’s previous estimates, the potential peak sales of Vamorolone for the DMD indication in the United States and the five major European markets are expected to exceed 500 million US dollars.
 

Genentech's new indication for entrectinib received accelerated FDA approval in October for the treatment of children aged one month and older with neurotrophic tyrosine receptor kinase (NTRK) gene fusion-positive solid tumors. The product is a potent and selective inhibitor targeting pan-TRK and ROS1 tyrosine kinases with central nervous system (CNS) activity, capable of crossing the blood-brain barrier. Since 2019, entrectinib has been successively approved for marketing in Japan, the United States, the European Union, and other regions. In August 2022, it was approved for marketing by China’s National Medical Products Administration and currently has two approved indications. A third indication application was accepted by the National Medical Products Administration in May this year, intended for pediatric patients with NTRK fusion-positive locally advanced or metastatic solid tumors.
 

In addition, toripalimab developed by Junshi Biosciences was approved for marketing in the United States in October. The indications cover the full-line treatment of recurrent/metastatic nasopharyngeal carcinoma, including combination with cisplatin/gemcitabine as first-line treatment for adult patients with metastatic or recurrent locally advanced nasopharyngeal carcinoma; monotherapy for adult patients with recurrent, unresectable, or metastatic nasopharyngeal carcinoma whose disease has progressed during or after prior platinum-containing therapy. This product has also become the first innovative biologic drug independently developed and produced in China to be approved for marketing in the United States. As the first approved PD-1 monoclonal antibody in China, this product has already received approval for six indications in China.
 

Notably, in addition to obtaining approval in the United States, Junshi Biosciences is also actively expanding into other overseas markets. The company has currently submitted marketing applications for this product to the European Medicines Agency (EMA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA), which have been accepted. The indications are for the drug's use in combination regimens as a first-line treatment for patients with locally recurrent or metastatic nasopharyngeal carcinoma, and as a first-line treatment for patients with locally advanced/recurrent or metastatic esophageal squamous cell carcinoma.
 

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