How Will an Appeals Court Ruling on Ruzurgi Affect My Daughter?

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by Lori Dunham |

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Most of us in the Lambert-Eaton myasthenic syndrome (LEMS) community have been following a court case involving Ruzurgi (amifampridine), the only LEMS treatment approved by the U.S. Food and Drug Administration for children under 17.

In 2019, Catalyst Pharmaceuticals sued the FDA and other parties over the agency’s approval of Ruzurgi, which was developed by Jacobus Pharmaceuticals.

As Lambert-Eaton News‘ Marisa Wexler noted last week, Catalyst Pharmaceuticals argued that Ruzurgi’s approval “violated the company’s right to orphan drug exclusivity for Firdapse — a therapy chemically similar to Ruzurgi and approved for adults with LEMS.”

On Sept. 30, the U.S. 11th Circuit Court of Appeals ruled in favor of Catalyst Pharmaceuticals, determining that the FDA should not have approved Ruzurgi for children with LEMS because of Firdapse’s orphan drug designation.

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The fallout

The appeals court ruling has created significant anxiety and worry in our community as we wait to see how it will affect our loved ones. As a parent and caregiver to a 16-year-old child with LEMS, I am anxious and uncertain about our daughter Grace’s future.

Grace was diagnosed in June 2019, one month after the FDA approved Ruzurgi. We were relieved that a medication was finally available for pediatric patients. Grace started treatment with Ruzurgi, and her life immediately changed in the following ways.

Before Ruzurgi:

  • She needed either a wheelchair or a walker.
  • She slurred her words when speaking.
  • She was homebound.
  • She was unable to get in and out of the car without assistance.
  • She had to give up all sports and physical activities.

With Ruzurgi:

  • She is walking on her own without assistance.
  • She speaks clearly and now takes a speech and debate class.
  • She is back in school and has a part-time job that she loves.
  • She has her learner’s permit and is practicing for her driving test so she can get her license.
  • She has begun riding horses at an equine therapy facility.

As dramatic as that all may seem, the most profound improvement was in Grace’s mental health.

A long road to healing

Before Grace started taking Ruzurgi, she felt hopeless. She had gone from a being healthy, 14-year-old aspiring artist to a listless, stripped-down version of herself. Grace could no longer play piano or basketball, or even grip a pencil. She lost over 20 pounds and was wasting away. We had to help her to the bathroom and lift her out of bed in the morning.

Grace has given me permission to share that one day, while in deep despair, she whispered to me that she wished she would die. It would be easier that way, she said.

Words cannot express what hearing this does to a mother. That day, I felt the deepest despair I had ever known, and probably ever will. We sought out a counselor as we continued to pursue physical healing. Although counseling was helpful, the greatest help came from Ruzurgi.

The day Grace started the treatment was the day her life was saved.

Today, as I write this, my Grace is singing at the top of her lungs in her bedroom. Her joy fills our home to the brim. She is her happy, carefree self again, now that she has medicine and a treatment plan. Ruzurgi has given us our daughter back!

She has no idea that the medicine she relies on to get out of bed in the morning may not be available to her tomorrow.

For now, we wait and pray that officials will consider the lives of those reliant upon this medication. The court’s decision notes that, “The number of pediatric cases is infinitesimal—believed to be a ‘couple of dozen’ nationwide.” But for me, one is enough.

One life is all we need to make this medication accessible.

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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.

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