Three NMOSD drugs approved by the FDA in one year! Roche/Genetek deepens the drug layout of neurological disorders.

Guide: The FDA has approved three drugs, Soliris, Uplizna and Enspryng, for NMOSD treatment.
recently, the FDA approved Roche's Gene Tektronumab's Enspryng (satralizumab) for the treatment of adult patients with anti-water channel protein-4 (AQP4) antibody-positive optic neuromyelitis spectrum disorder (NMOSD).
Enspryng is the only white blood cell interleukin-6 (IL-6) monoantigen approved for NMOSD, which was approved for marketing in Japan in June, and China's CDE has accepted import applications for the drug, which has been included in the proposed priority review list, with the hope of accelerating the domestic approval of the innovative treatment.
early 2019, no NMOSD drugs have been approved for marketing at the FDA.
now, there are three, and the other two are Soliris of Alexion and Uplizna of Viviela Bio.
NMOSD is a rare, lifelong, degenerative autoimmune disease characterized by inflammatory diseases of the optic nerve and spinal cord, with a global prevalence rate of 1 to 5 per 100,000 people per year.
NMOSD occurs mostly at the golden age of young and middle-aged people, and patients often experience a recurrent disease process, and each recurrence can lead to nerve damage and disability gradually accumulate, resulting in severe disability consequences such as blindness and paralysis, and even more serious death.
FDA's approval is based on two randomized controlled Phase 3 clinical trials, the Sakura Star and SakuraSky studies.
SakuraStar study showed that 76.5 percent of patients in the AQP4 antibody-positive sub-group who received Enspryng single-drug treatment did not relapse at 96 weeks, up from 41.1 percent in the placebo group.
in the SakuraSky study, which evaluated Enspryng in cooperation with BaselineIST, 91.1 percent of AQP4 antibody-positive patients treated with Enspryng had no recurrence at 96 weeks, compared with 56.8 percent in the placebo group.
as a single-drug treatment or in combined with baseline therapy, Enspryng is an effective treatment option.
Enspryng is injected every four weeks, which is a convenient treatment option for patients and caregivers.
a spokesman for Genentic said Enspryng costs $13,190,000 a year, and 15 doses in its first year of treatment cost about $220,000.
at the end of June 2019, Alexion's first supplement inhibitor, Soliris (eculizumab), was approved by the FDA for use in adult patients who were anti-AQP4 antibody-positive NMOSD, and was the first FDA approved drug to treat NMOSD.
treatment of NMOSD, Soliris has previously been approved for the treatment of PNH, aHUS, anti-AchR antibody-positive gMG.
Notally, the drug is one of the world's best-selling orphan drugs, with sales of $3,946 million in 2019, and the industry predicts that the approval of NMOSD adaptation will add about $700 million to Soliris' sales.
black box warning that Soliris is at risk of meningococcal infection, which costs more than $500,000 a year.
June, Viela Bio's anti-CD19 monoantimmune drug Uplizna (formerly KNOWNI-551) was approved by the U.S. FDA to treat NMOSD-positive adult patients with anti-AQP4 antibodies.
Uplizna is the first and only B-cell expendant approved for the treatment of AQP4 antibody-positive NMOSD adult patients.
has yet to set a price, but analysts say it is expected to be between $200,000 and $280,000 a year.
These three approved NMOSD drugs act at different stages of the disease, in which Satralizumab mainly inhibits B cell differentiation into plasma cells to block the production of AQP4 autoantibodies, while Uplizna blocks the production of AQP4 autoantibodies by directly removing plasma cells expressing CD19, and Soliris mainly reduces brain damage by inhibiting activated complement systems.
, all three drugs have applied for import in China and are under review at the Drug Review Center, which is expected to improve the situation of shortage and shortage of drugs in NMOSD patients in China.
references: 1. Alexion Receives FDA Approval Of SOLIRIS® (Eculizumab) For The Treatment Of Adults Neuromyelitisa Spectrum Disorder (NMOSD) Who Are Anti-Aquaporin-4 (AQP4) Antibody Positive2.Viela Bio Announces U.S. FDAapproval of UPLIZNA ™ (inebilizumab-cdon) for the Treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD) 3. FDA Approves Roche's ENSPRYNG for Neuromyelitis. Optica Spectrum Disorder (NMOSD)