In recent years, the landscape of Myasthenia Gravis (MG) treatment has been revolutionized by the introduction of several new drugs approved by the FDA since 2017. These
innovative therapies offer hope and improved quality of life for patients grappling with this complex autoimmune disorder. During our Virtual Monthly Meeting, Dr. Farmakidis spoke on these advancements. Below is a recap of Dr. Farmakidis' presentation.
1. Eculizumab (Soliris):
Cost: Eculizumab is among one of the most expensive treatments for MG, often reaching $470,000 annually.
Frequency of Treatment: Administered every two weeks by intravenous infusion.
Pros: Eculizumab effectively targets the complement cascade, significantly improving MG symptoms and preventing disease exacerbations. Its long-acting formula provides prolonged protection against flares.
Cons: Eculizumab is less stable (requires refrigeration) and the intravenous administration requires visits to infusion centers, which can be inconvenient. Due to the large molecules it may cause immunogenicity and can conflict with IVIG & PLEX rescue therapy.
Onset of Action: You should feel the effects of the treatment in 4 weeks.
Side Effects: Common side effects include headache, upper respiratory tract infections, and hypertension. Infusion-related reactions and increased risk of infections are potential concerns.
2. Ravulizumab (Ultomiris):
Cost: Ravulizumab is also among the more expensive MG treatments. Cost can fluctuate because it varies by wait, but averages $520,000 per year.
Frequency of Treatment: Initially administered every two weeks for several doses, then every eight weeks thereafter. Ravulizumab is also administered by intravenous infusion.
Pros: Ravulizumab offers sustained suppression of the complement cascade with an extended dosing interval, enhancing convenience for patients.
Cons: High cost may limit access. Intravenous administration requires visits to infusion centers.
Onset of Action: You should feel the effects of the treatment in 4-12 weeks.
Side Effects: Common side effects include headache, upper respiratory tract infections, and hypertension. Serious reactions like infusion-related reactions or meningococcal infections are rare but possible.
3. Zilucoplan (ZILBRYSQ):
Cost: Zilucoplan is priced comparably to other biologic therapies for MG, about $380,000 a year.
Frequency of Treatment: Requires daily subcutaneous injections.
Pros: Self-administration at home offers flexibility and convenience. Effective inhibition of the complement cascade results in significant symptom improvement.
Cons: Daily injections may be challenging for some patients.
Onset of Action: You should feel the effects of the treatment in 4 weeks.
Side Effects: Common side effects include injection site reactions, headache, and upper respiratory tract infections.
4. Efgartigimod (Vyvgart):
Cost: Efgartigimod costs varying based on dosing frequency and insurance coverage, about $225,000 a year.
Frequency of Treatment: Initially administered weekly for 4 weeks, followed by a break of at least 50 days before beginning the cycle again.
Pros: Targets disease-causing antibodies, offering rapid symptom relief and sustained improvement.
Cons: Intravenous administration may necessitate visits to infusion centers.
Onset of Action: You should feel the effects of the treatment in 4 weeks but can diminish significantly by week 8.
Side Effects: Common side effects include headache, fatigue, and upper respiratory tract infections.
5. Efgartigimod-Hyaluronidase:
Cost: Similar to efgartigimod, with variations based on dosing and insurance coverage, about $225,000 a year.
Frequency of Treatment: Initially administered weekly for 4 weeks, followed by a break of at least 50 days before beginning the cycle again.
Pros: Utilizes a hyaluronidase enzyme to facilitate subcutaneous administration, offering an alternative to intravenous infusions.
Cons: Cost and potential side effects are similar to efgartigimod.
Onset of Action: Research has not determined a timeline for the effects of this treatment.
Side Effects: Common side effects include headache, fatigue, and upper respiratory tract infections.
6. Rozanolixizumab (Rystiggo):
Cost: Priced comparably to other biologic therapies for MG.
Frequency of Treatment: Initially administered weekly for 6 weeks, followed by a break of at least 63 days before beginning the cycle again.
Pros: Targets the neonatal Fc receptor, reducing levels of disease-causing antibodies and improving MG symptoms.
Cons: Requires intravenous administration, potentially limiting accessibility for some patients.
Onset of Action: You should feel the effects of the treatment in 6 weeks. Effects wear off gradually after 8 weeks.
Side Effects: Common side effects include upper respiratory tract infections, headache, and nausea.
7. Rituximab (Rituxan):
Cost: Rituximab is priced similarly to other biologic therapies for MG.
Frequency of Treatment: Administered as infusions in two different options, either 2 doses at days 1 and 15 or two doses a week for 4 weeks.
Pros: Targets B cells, reducing antibody production and improving MG symptoms.
Cons: Intravenous administration may require visits to infusion centers. Long-term safety data are limited.
Onset of Action: You should feel the effects of the treatment in 8-16 weeks.
Side Effects: Common side effects include infusion reactions, fatigue, and infections.
These new drug treatment options for Myasthenia Gravis represent significant advancements in the management of this complex autoimmune disorder. While each option comes with its own set of considerations, including cost, frequency of treatments, and potential side effects, they collectively offer patients a range of effective therapeutic choices.
Consulting with healthcare providers and weighing the available options is essential for making informed decisions tailored to individual needs and circumstances. Speak to your medical team if you feel you may want to explore any of these drugs/treatments
further.