Domestic and foreign drug companiesincrease their presence in rare diseases

    In fact, Raison Pharmaceuticals, which isinvolved in the latest acquisition, is also a rare disease company acquired byAstraZeneca. in late 2020, AstraZeneca announced that it had agreed to acquireRaison Pharmaceuticals, which was founded in 1992, for $39 billion in cash andstock, a move that marked its official entry into the rare disease sector.Later in September 2021, AstraZeneca also acquired Caelum BioSciences, a raredisease pharmaceutical company, for $500 million.

　　AstraZeneca is not the firstmultinational pharmaceutical company to add to the rare disease sector. inNovember 2021, Merck Sharp & Dohme announced the completion of itsacquisition of Acceleron Pharma, whose business includes the research anddevelopment of rare disease drugs, an acquisition worth nearly $11.5 billion.earlier, in May 2018, Japan's Takeda Pharmaceuticals acquired Irish raredisease pharmaceutical giant Char for $65 billion. The acquisition wascompleted in January 2019.

　　Compared to the large acquisitions bymultinational pharmaceutical companies, the actions of domestic pharmaceuticalcompanies in the rare disease field are mainly reflected in the layout of theproduct pipeline.

　　Beihai Kangcheng, known as the "No.1 local rare disease stock", was listed on the Hong Kong Stock Exchange atthe end of 2021. According to its semi-annual report published in Septemberthis year, Beihai Kangcheng's pipeline portfolio includes biologics, smallmolecules and gene therapy solutions with effective mechanisms of action forsome of the most common rare diseases and rare oncology indications that havesignificant market potential. Among its commercialized products, Edo sulfatasebeta injection is the first enzyme replacement therapy approved in China forthe treatment of the rare disease Hunter's syndrome.

　　In March 2022, Inelizumab Injection, ahumanized anti-CD19 monoclonal antibody of Henson Pharmaceuticals, was approvedfor marketing by the State Drug Administration, the first anti-CD19 monoclonalantibody for the treatment of optic neuromyelitis optica spectrum disorder(NMOSD) in China.NMOSD is a central nervous system autoimmune disease that wasincluded in the national list of 121 rare diseases in 2018, with a total globalThe prevalence is 0.5-4/100,000.

　　In April 2022, Treprolizumab for thetreatment of small cell lung cancer was granted orphan drug (also known as"orphan drug") status by the FDA, which is the fifth FDA orphan drugstatus for this drug. Later in July, Tremelimumab for nasopharyngeal carcinomawas granted orphan drug status by the European Commission.

　　It is worth noting that many of thedomestic pharmaceutical companies' layouts in rare diseases are notself-developed, but rather they acquire relevant rights and interests throughcooperation with foreign companies, such as Hansson Pharmaceutical's Inelizumabinjection, for which Hansson Pharmaceutical entered into a licensing agreementwith Viela Bio, Inc. in May 2019 to acquire the rights to develop andcommercialize the drug in China for $220 million; in December 2021, the TheState Drug Administration approved the import registration application for arare disease drug for injection of stauroximab through the priority review andapproval process, which is a rare disease drug in which Baiji Shenzhou and EUSAPharma have acquired rights.