SOLIRIS is a first-in-class terminal complement inhibitor discovered, developed, and commercialized by Alexion. SOLIRIS works by selectively inhibiting activation of specific proteins in the complement system (C5a and C5b), which play a role in the pathophysiology of multiple rare diseases.
SOLIRIS is indicated for: the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy, the treatment of adult patients with generalized Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody positive, and neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. SOLIRIS is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). It is not known if SOLIRIS is safe and effective in children with PNH, or gMG, or NMOSD.
SOLIRIS has earned some of the pharmaceutical industry’s highest honors for innovation, including the 2008 Prix Galien USA Award for Best Biotechnology Product and the 2009 Prix Galien France Award in the category of Drugs for Rare Diseases.
SOLIRIS and NMOSD
SOLIRIS is the first and only complement inhibitor approved by the FDA for the treatment of adults with anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD).1
SOLIRIS is the first and only FDA-approved drug for the treatment of NMOSD in adult patients who are anti-AQP4 antibody positive. The safety and efficacy of SOLIRIS were established in a phase 3, randomized, double-blind, placebo-controlled, multicenter, time-to-event trial in adults with anti-AQP4 antibody-positive NMOSD (PREVENT, N=143).
The primary endpoint of the PREVENT trial was time to first adjudicated on-trial relapse, which was significantly longer in SOLIRIS-treated patients (n=96) compared to placebo-treated patients (n=47) (relative risk reduction, 94%; hazard ratio, 0.058; P<0.0001).
The most frequently reported adverse reactions in the PREVENT trial (≥10%) were: upper respiratory infection, nasopharyngitis, diarrhea, back pain, dizziness, influenza, arthralgia, pharyngitis, and contusion.
SOLIRIS and gMG
SOLIRIS is the first and only complement inhibitor approved for adults with anti-acetylcholine receptor antibody-positive (AChR+) generalized Myasthenia Gravis (gMG), a chronic and debilitating neuromuscular disorder.1,2
SOLIRIS is the first and only complement inhibitor approved for the treatment of adult patients with gMG who are AChR+. The safety and efficacy of SOLIRIS for the treatment of gMG were established in a 26-week, Phase 3, randomized, double-blind, placebo-controlled, multicenter clinical study (REGAIN, N=125).1,2
The primary efficacy endpoint was a comparison of the change from baseline between SOLIRIS (n=62) and placebo (n=63) in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score at week 26.2 See detailed results of the REGAIN Study and Interim Analysis from Phase 3 Open-Label Extension Study.
The most frequently reported adverse reaction in the gMG placebo-controlled trial (≥10%) is musculoskeletal pain.1
SOLIRIS and aHUS
SOLIRIS was the first therapy approved for the treatment of atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.1 SOLIRIS is approved for the treatment of patients with aHUS in more than 40 countries, including the United States, European Union, and Japan.
The safety and efficacy of SOLIRIS for the treatment of aHUS has been demonstrated in 4 multinational, prospective studies (N=100). SOLIRIS treatment resulted in improvement in hematologic markers of complement-mediated TMA, including platelet counts and lactate dehydrogenase (LDH) levels.1,5-7 Patients treated with SOLIRIS also had improvements in renal function as measured by estimated glomerular filtration rate (eGFR).1,3-5
The most frequently reported adverse reactions in aHUS single-arm prospective trials (≥20%) are headache, diarrhea, hypertension, upper respiratory infection, abdominal pain, vomiting, nasopharyngitis, anemia, cough, peripheral edema, nausea, urinary tract infections, and pyrexia.1
SOLIRIS and PNH
SOLIRIS was the first therapy approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis.1 SOLIRIS is approved for the treatment of patients with PNH in nearly 50 countries worldwide, including the United States, European Union, and Japan.
The safety and efficacy of SOLIRIS in patients with PNH with hemolysis were assessed in the randomized, double-blind, multicenter, placebo-controlled, 26-week TRIUMPH Study, which comprised 87 transfusion-dependent patients with PNH receiving SOLIRIS (n=43) or placebo (n=44).6 Patients were also treated in a single-arm, multicenter study comprising 97 patients with PNH treated with SOLIRIS over 52 weeks (SHEPHERD Study).7 A long-term extension study included 187 patients with PNH initially enrolled in one of 3 parent trials (N=195) who continued to receive SOLIRIS for a range of 10 to 54 months.1,8
Data from the trials demonstrated that patients treated with SOLIRIS experienced significantly reduced hemolysis, as measured by lactate dehydrogenase (LDH) levels, leading to an improvement in symptoms and a reduction of thrombotic events, a major health problem associated with the disease. Approximately half the patients received concomitant anticoagulant therapy. The effect of anticoagulant withdrawal during SOLIRIS treatment has not been studied.1,9
The most frequently reported adverse reactions in the PNH randomized trial (≥10% overall and greater than placebo) are headache, nasopharyngitis, back pain, and nausea.1