October Advances in Rare Disease Drug Development: New Approvals and Key Product Updates

☞ Biogen Tofersen Injection Approved for Marketing in China

☞ Qilu Pharmaceutical's First Generic Riociguat Approved for Marketing in China

☞ Pfizer's Hemophilia Biologic Therapy Approved for Marketing in the United States

The NMPA official website shows that Biogen's Tofersen Injection (Tofersen, trade name: Qalsody) has been approved for import to treat adult patients with amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. This is the world’s first treatment for SOD1-ALS and was approved for marketing in China through a clinical trial exemption pathway.

Tofersen is an antisense oligonucleotide (ASO) drug that reduces the synthesis of SOD1 protein by binding to and degrading SOD1 mRNA, preventing the accumulation of toxic SOD1 proteins, thereby alleviating motor neuron damage and slowing disease progression. Tofersen, jointly developed by Biogen and Ionis, has been approved for marketing in the United States and the European Union.

The NMPA official website shows that the marketing application for Riociguat Tablets submitted by Qilu Pharmaceutical Co., Ltd. has been approved for the treatment of pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH). This is the first domestically produced generic Riociguat to be approved for marketing in China.

RiociguatRiociguat, jointly developed by Bayer and Merck, is a soluble guanylate cyclase (sGC) stimulator. It can both stabilize the nitric oxide (NO)-sGC complex, enhancing sGC's sensitivity to NO, and directly stimulate sGC independently of NO, thereby increasing the production of cyclic guanosine monophosphate. This dual mechanism of action makes riociguat show good efficacy in PAH and CTEPH.

Rare Neurological Disorders Communication Group

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