Rare disease new drugs! Roche IL-6 monosalysalyzumab has long-term safety in adults and adolescents with psynocoeur cord itisis (NMOSD) adults and adolescents!

May 24, 2020 /
BiovalleyBIOON/ -- Roche recently announced at the 6th Annual Meeting of the European Academy of Neurology (EAN) new aggregated key results for the treatment of psynocopael prototypitis disorder (NMOSD) in the adolescent populationaggregatedata from two key Phase III studies on open label long-term expansion (OLE) show that satralizumab has good tolerance as a single therapy or as a combination of immunosuppressive therapy with baseline immunosuppressive therapyAdolescents who received the same dose and frequency were shown to be consistent with the overall benefit risk characteristics of the adult populationThe current dose achieves continuous suppression of the IL-6 signal within 4 weeksThese new data further support the potential of satralizumab as an NMOSD treatmentsatralizumab is a humanized monoantigen that targets IL-6 receptors to inhibit IL-6 signal conductionIL-6 is a cytokine that is thought to play a key role in nMOSD inflammation, triggering inflammatory cascades, leading to injury and disabilityNMOSD is a rare disease with few approved treatment options, and patients experience unpredictable and severe recurrences that directly lead to cumulative, permanent nerve damagesatralizumab uses a new antibody recovery technique design that allows antibody circulation to take longer and to be injected every 4 weeksThe drug is currently under review by regulators in the United States, the European Union and JapanIn the United States,FDAgranted satralizumab a breakthrough drug for nMOSD treatment in December 2018In the United States, the European Union, Japan, satralizumab has also been granted orphan drug statusProfessor Jerome de Seze, director of the Centre for Neurology and Clinical Research at the University of Strasbourg in, said: "Data from the Phase III study OLE reinforce the safety, observed tolerance and potential of satralizumab as a future treatment option for this chronic diseaseWhile significant progress has recently been made in understanding NMOSD, more approved treatment options are needed for those who do not have these services to provide well-tolerated safety while reducing the number of subcutaneous injections"
NMOSD (photo source: empr.com)results from two previously published phase III clinical studies (SakuraStar, SakuraSky) respectively confirmed that satralizumab's combination as a single therapy and combination with baseline immunosuppressants significantly reduced the risk of recurrence in NMOSD patients and showed good tolerance and safetyaggregate safety data from two studies for double-blind periods showed that the satralizumab group and placebo group were comparable in incidence of adverse events (AE) and severe adverse events (SAE) (SAE: 15.0 vs 18.0 events/100 patient years of pY) when combined with the drug with baseline therapyThe 2 most common groups of AE were urinary tract infections and upper respiratory tract infectionsNo deaths or allergic reactions have been reportedthe safety, AE nature and incidence of open label expansion (OLE) are consistent with the double blindness periodThere was no meaningful change in the incidence or type of infectionin another analysis of the SakuraSky study, adolescents treated with the same dose and frequency of satralizumab (n-8) showed the overall benefit risk characteristics of the adult populationData from the adolescent group found that when receiving a combination baseline therapy of placebo or satralizumab 120 mg at 0, 2, 4 weeks and every 4 weeks thereafter, the model predicted similar exposure to adultsfinally, in the third report, the administration of the drug (120 mg per 4 weeks) showed significant continuous IL-6 signal suppression, based on pharmacokinetics and pharmacodynamics analysis from Phase I and 2 key Phase III studiesIn the NMOSD population, the pharmacokinetics (PK) of satralizumab showed that a 120 mg dose allowed to bind more than 95% of IL-6 receptors over the entire 4-week administration periodCheryl Hemingway, M.D., of Great Ormond Street Children's Hospital in London,, said: "It is very encouraging to see positive results from the satralizumab trial in young people with NMOSD Currently, treatment options are not yet approved for Young NMOSD, who live every day in an unpredictable, severe relapse that can lead to permanent disability The satralizumab study represents a large population, including adolescents, and we hope that the drug will make a real difference for young people with this rare disease "
Soliris: The first NMOSD treatment NMOSD is a rare, lifelong, decaying autoimmune disease that becomes a feature of inflammatory diseases of the optic nerve and spinal cord NMOSD patients often go through the process of recurrent disease, which leads to the gradual accumulation of nerve damage and disability, with symptoms including visual impairment, motor impairment, and reduced quality of life In some cases, NMOSD attacks can result in death NMOSD is usually associated with pathogenic antibodies (water channel protein-4 (AQP4)-IgG), which targets and injures a specific cell type called astrocytes, resulting in inflammatory damage to the optic nerve, spinal cord, and brain Most NMOSD patients were identified as AQP4-IgG serologically positive through diagnosis and biomarker testing; This condition is often misdiagnosed as multiple sclerosis satralizumab is a humanized monoantigen that targets IL-6 receptors to inhibit IL-6 signal conduction IL-6 is a cytokine that is thought to play a key role in nMOSD inflammation, triggering inflammatory cascades, leading to injury and disability NMOSD patients experience unpredictable and severe recurrences that directly lead to cumulative, permanent nerve damage it's worth noting that at the end of June 2019, Soliris, the pioneering tonic inhibitor of rare disease giant Alexion, won the U.S FDA approved for anti-water channel protein-4 (AQP4) antibody-positive psynocoeur syndrome (NMOSD) adult patients At the end of August 2019, Soliris was again approved by the European Union for use in adult patients with AQP4 antibody-positive NMOSD with a recurrent course In the United States and the European Union, Soliris is the first and only drug approved to treat NMOSD (biovalleybioon.com) original origin: New long-term data reinforce safety of Roche's satralizumab in adults and the weeks with neuromyelitis optica spectrum