Our Experience Transitioning From Ruzurgi to Firdapse
In May 2019, the U.S. Food and Drug Administration (FDA) approved Ruzurgi (amifampridine) for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in children ages 6-16. Our daughter Grace, then 14, was diagnosed with LEMS that July.
Although receiving the diagnosis was hard, we were relieved that medication was available. Ruzurgi seemed to work wonders for many others.
We quickly became aware of a lawsuit filed by Catalyst Pharmaceuticals against the FDA and others, arguing that Ruzurgi’s approval violated Catalyst’s right to orphan drug exclusivity for Firdapse (amifampridine), a chemically similar therapy approved for adults with LEMS.
For years, fear hovered in the backs of our minds as we waited to see if our daughter’s medication, which had improved her quality of life exponentially, would be taken off the market. If it was, what then? Would she be allowed to take the adult version, Firdapse? Would it be as effective as Ruzurgi?
The uncertainty was stifling. Our fears were realized a few weeks ago when a federal appeals court issued a mandate vacating the FDA’s 2019 approval of Ruzurgi.
I recognize that there are strong arguments for both sides of the issue. My goal is not to comment on the lawsuit, but to share our experience of transitioning from Ruzurgi to Firdapse.
I am acutely aware that many people have had different experiences than we have. My hope is that all adults and children with LEMS receive the most effective treatment for them.
Prior to switching medications, I had been made aware that some in the LEMS community could not tolerate Firdapse. I purposely shielded Grace from that information. I knew it was possible that she’d have to switch therapies eventually, and I didn’t want her to start a new treatment with any negative ideas or preconceived notions.
In our experience, Catalyst proved helpful, as representatives enrolled Grace in the correct program for her unique situation. They assured us that she would not go without medication. We received individualized attention as we maneuvered the insurance maze, prescriptions, and dosage.
Of course, the question that loomed was whether or not Firdapse would work as well for her as Ruzurgi. Thankfully, Grace hasn’t had any trouble with Firdapse. She said it takes longer than Ruzurgi to kick in, but the effects last longer. She didn’t notice any dips in efficacy while switching medications.
Although we had immediate access to Firdapse and it is working well for Grace, I know that may not be the case for everyone. I hope that all children will be offered access to lifesaving medication. I also hope the Ruzurgi formula will be made available for those who need it.
As a parent of a child with a debilitating disease, I honestly don’t care who makes the drug as long as it works and makes it into my child. For me, the issue is simple: If a drug helps someone to regain the ability to walk, it should be made available to them.
However, I acknowledge that the pharmaceutical business is complex, especially when it comes to rare diseases. I hope the efforts of companies investing in LEMS will bring about more health and healing for the entire LEMS community.
Note: Lambert-Eaton News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Lambert-Eaton News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to Lambert-Eaton myasthenia syndrome.