REGAIN included patients with anti-AchR+ gMG with varying characteristics and disease burden1,2

The REGAIN trial included 125 patients with a range of demographic characteristics1,2

Demographics
  • Female: 66%2
  • ≥65 years: 20.8% (26/125)2
  • Mean MG duration (min, max): 9.55 years (1.0, 33.8)1
  • Mean age at first study dose (min, max): 47.2 years (19.0, 79.0)1
    • Female: 42.0 years (19.0, 74.0)1
    • Male: 57.0 years (22.0, 79.0)1
Prior treatments
  • Approximately 50% of patients had been treated with ≥3 immunosuppressants since diagnosis2,a
  • Use of IVIg or PLEX
    • IVIg: 28.0% chronic,b 63.5% acute setting1
    • PLEX: 11.2% chronic,b 42.4% acute setting1
  • 54.4% of patients had undergone thymectomy1
Immunosuppressants at baseline and during REGAINc
  • 80.0% (n = 100) received corticosteroids1
  • 85.6% (n = 107) received immunosuppressants other than prednisone1

During REGAIN, over 95% of patients in each group were receiving acetylcholinesterase (AchE) inhibitors, and 98% were receiving immunosuppressant therapies (ISTs)2

aImmunosuppressants used prior to enrollment included but were not limited to AZA, MMF, MTX, cyclosporine, tacrolimus, cyclophosphamide, corticosteroids, and rituximab. Use of rituximab within 6 months prior to screening was an exclusion criterion.1
bChronic PLEX or IVIg is defined as >4 treatments in a year.1
cOn stable dosages prior to screening, defined as AZA: on drug for ≥6 months, on a stable dose for ≥2 months; MMF, MTX, cyclosporine, tacrolimus, or cyclophosphamide: on drug for ≥3 months, on stable dosage for ≥1 months; oral steroids: on stable dosage for ≥28 days.1

dAt randomization stratification. MGFA class I encompasses ocular muscle weakness only (excluded from REGAIN); classes II, III, or IV refer to mild, moderate, or severe weakness, affecting muscles other than ocular, but may also include ocular weakness of any severity; class a: weakness predominantly affecting limb and/or axial muscles; class b: predominantly affecting oropharyngeal and/or respiratory muscles; class V refers to patients in MG crisis (excluded from REGAIN).1,3

Patients in the REGAIN trial had persistent respiratory, bulbar, limb, and ocular symptoms at baseline1

Persistent symptoms as assessed by the MG activities of daily living (MG-ADL) instrument1

Respiratory System1,3

  • Shortness of breath: 80.0% (n = 100)
  • With exertion or at rest. No patients needed ventilator support at baseline.

Bulbar Impairment1,3

  • Chewing: 82.4% (n = 103)
  • Fatigue chewing solid food or soft food.
  • Swallowing: 73.6% (n = 92)
  • Rare episodes of choking or frequent choking necessitating changes in diet. No patients needed a gastric tube at baseline.
  • Talking: 72.0% (n = 90)
  • Intermittent slurring or nasal speech; constant slurring or nasal speech, but can be understood; or difficult-to-understand speech.

Limb Impairment1,3

  • Lower limb (ability to rise from chair): 80.8% (n = 101)
  • Mild, sometimes uses arms; moderate, always uses arms; or severe, requires assistance.
  • Upper limb (impairment of ability to brush teeth or comb hair): 72.8% (n = 91)
  • Extra effort, but no rest periods needed; rest periods needed; or cannot do one of these functions.

Ocular Symptoms1,3

  • Ptosis: 88.8% (n = 111)
  • Symptom occurs, but not daily; occurs daily, but is not constant; or occurs constantly.
  • Diplopia: 83.2% (n = 104)
  • Symptom occurs, but not daily; occurs daily, but is not constant; or occurs constantly.

REGAIN included patients with anti-AchR+ gMG with persistent symptoms and considerable disease burden at baseline.1

REGAIN established the efficacy of Soliris for anti-AchR+ gMG.

See the data

For patients who responded to Soliris, available data suggest that clinical response is usually achieved by 12 weeks of Soliris treatment.2

Learn more

INDICATION & IMPORTANT SAFETY INFORMATION FOR SOLIRIS® (eculizumab)

INDICATION

Generalized Myasthenia Gravis (gMG)

Soliris is indicated for the treatment of adult patients with generalized Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody positive.

IMPORTANT SAFETY INFORMATION

WARNING: SERIOUS MENINGOCOCCAL INFECTIONS

Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early.

 

  • Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies.
  •  
  • Immunize patients with meningococcal vaccines at least 2 weeks prior to administering the first dose of Soliris, unless the risks of delaying Soliris therapy outweigh the risk of developing a meningococcal infection. (See Serious Meningococcal Infections for additional guidance on the management of the risk of meningococcal infection)
  •  
  • Vaccination reduces, but does not eliminate, the risk of meningococcal infections. Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected.

 

Soliris is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the Soliris REMS, prescribers must enroll in the program. Enrollment in the Soliris REMS program and additional information are available by telephone: 1-888-SOLIRIS (1-888-765-4747) or at www.solirisrems.com.

 

Contraindications

Soliris is contraindicated in:

  • Patients with unresolved serious Neisseria meningitidis infection
  • Patients who are not currently vaccinated against Neisseria meningitidis, unless the risks of delaying Soliris treatment outweigh the risks of developing a meningococcal infection

 

Warnings and Precautions

 

Serious Meningococcal Infections

 

Risk and Prevention

 

See Boxed WARNING for additional information on serious meningococcal infections.

 

Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. The use of Soliris increases a patient’s susceptibility to serious meningococcal infections (septicemia and/or meningitis).

 

Vaccinate for meningococcal disease according to the most current ACIP recommendations for patients with complement deficiencies. Revaccinate patients in accordance with ACIP recommendations, considering the duration of Soliris therapy.

 

Immunize patients without a history of meningococcal vaccination at least 2 weeks prior to receiving the first dose of Soliris. If urgent Soliris therapy is indicated in an unvaccinated patient, administer meningococcal vaccine(s) as soon as possible and provide patients with 2 weeks of antibacterial drug prophylaxis.

 

The benefits and risks of antibiotic prophylaxis for prevention of meningococcal infections in patients receiving Soliris have not been established.

 

Vaccination reduces, but does not eliminate, the risk of meningococcal infections.

 

Closely monitor patients for early signs and symptoms of meningococcal infection, and evaluate patients immediately if an infection is suspected. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Discontinue Soliris in patients who are undergoing treatment for serious meningococcal infections.

 

REMS

Because of the risk of meningococcal infections, Soliris is available only through a restricted program under a REMS. Under the Soliris REMS, prescribers must enroll in the program.

 

Prescribers must counsel patients about the risk of meningococcal infection, provide the patients with the REMS educational materials, and ensure patients are vaccinated with meningococcal vaccine(s).

 

Other Infections

Serious infections with Neisseria species (other than N. meningitidis), including disseminated gonococcal infections, have been reported.

 

Soliris blocks terminal complement activation; therefore, patients may have increased susceptibility to infections, especially with encapsulated bacteria. Additionally, Aspergillus infections have occurred in immunocompromised and neutropenic patients. Children treated with Soliris may be at increased risk of developing serious infections due to Streptococcus pneumoniae and Haemophilus influenzae type b (Hib). Administer vaccinations for the prevention of Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) infections according to ACIP guidelines. Use caution when administering Soliris to patients with any systemic infection.

 

Infusion Reactions

Administration of Soliris may result in infusion reactions, including anaphylaxis or other hypersensitivity reactions. In clinical trials, no patients experienced an infusion reaction that required discontinuation of Soliris. Interrupt Soliris infusion and institute appropriate supportive measures if signs of cardiovascular instability or respiratory compromise occur.

 

Adverse Reactions

The most frequently reported adverse reaction in the gMG placebo-controlled clinical trial (≥10%) is: musculoskeletal pain.

 

Please see full prescribing information for Soliris, including boxed WARNING regarding serious meningococcal infections.

Abbreviations: anti-AchR+, acetylcholine receptor antibody positive; AZA, azathioprine; gMG, generalized myasthenia gravis; ICU, intensive care unit; IVIg, intravenous immunoglobulin; MG, myasthenia gravis; MGFA, Myasthenia Gravis Foundation of America; MMF, mycophenolate mofetil; MTX, methotrexate; PLEX, plasma exchange, REGAIN, Eculizumab for Refractory Generalized Myasthenia Gravis.

References

  1. Data on file. Alexion Pharmaceuticals, Inc.
  2. Soliris [prescribing information]. Boston, MA: Alexion Pharmaceuticals Inc; 2018.
  3. Educational Materials. Myasthenia Gravis Foundation of America website. http://www.myasthenia.org/HealthProfessionals/EducationalMaterials.aspx. Accessed September 8, 2017.