Potential New Therapies for MG
There are several new emerging pharmaceuticals for myasthenia gravis (MG). Many of these therapies have a novel mechanism of action, thereby offering a new potential treatment for people who do not respond to the current standard of care. New therapies typically have to go through rigorous testing for safety and effectiveness before they can be marketed and sold in their respective authorizing country.
Drug approval process
While some of these therapies are currently available, not all have come to market, and some may never end up being sold. For example, in the United States, before a drug can be prescribed, the Food and Drug Administration (FDA) must approve the therapy. The drug must demonstrate safety and efficacy (how well it works) in people with MG.
However, it can be possible to get access to these therapies before FDA approval, as in the case for manufacturer-sponsored clinical trials. To check whether a therapy is approved in your country, ask a member of your healthcare team or check the regulatory body of your country’s website (e.g., Health Canada, FDA).
Just because a drug is marketed, it does not necessarily mean that your insurance plan or the public health plan will cover the therapy just yet. There is typically another level of review that insurers and payers use to determine whether a therapy will be funded.
Here are some upcoming drugs, also known as pipeline drugs, for MG. Note that this list is not exhaustive.
Efgartigimod (Vyvgart™) was approved by the FDA in December 2021. It is used in people who have the anti-acetylcholine receptor (AChR) antibody. About 85 percent of people with MG have this antibody. One of the goals of the treatment is to reduce the levels of this antibody, which is how this drug works.1
Efgartigimod is given through intravenous (IV) infusion once weekly. However, there are some studies that are assessing a subcutaneous (under the skin) formulation, which is generally better tolerated and allows for self-administration. The drug can be given continuously or pulsed short-term.1
One of the trials assessed how well the drug works based on the change in the MG-ADL score, which is the Activity of Daily Living scale. This scale measures how well a person is able to perform activities of day-to-day living. In the trial, more people treated with efgartigimod versus the current conventional therapies showed at least a 2-point improvement on this scale.1
The most common side effect observed in the trials was headache. Some people also experienced nausea, diarrhea, and infections (e.g., urinary tract).1
Rozanolixizumab works in the same way as efgartigimod. This drug is given as a subcutaneous injection administered once weekly. Similar to efgartigimod, preliminary trials show that people who take this drug may experience improvements in their MG-ADL score.1
Zilucoplan has a different mechanism of action than the above 2 treatments and can be administered at home as a subcutaneous injection. Unlike some of the other new drugs for MG, this drug must be injected daily.1Have you heard of these new potential therapies for MG? Comment down below!
Have you made any MG-friendly adaptations to your home?