Who makes Ruzurgi and what is it used for?
Ruzurgi, also known as amifampridine, is a medication developed by Biohaven Pharmaceuticals [1]. It is used to treat Lambert-Eaton Myasthenic Syndrome (LEMS), a rare autoimmune disorder that affects the nerves and muscles.
Is Ruzurgi available for pediatric patients with LEMS?
Ruzurgi has received FDA approval for the treatment of LEMS in adults and children aged 6 years and older [2]. However, its availability for pediatric LEMS patients may be limited, and healthcare providers may need to work with pharmacies to access the medication.
Clinical trials and pediatric use
While there have been clinical trials of amifampridine in adults with LEMS, the number of pediatric trials is limited. The pediatric population (6-17 years) was not specifically studied in the clinical trials that led to Ruzurgi's FDA approval. This means that the safety and efficacy of Ruzurgi in pediatric patients have not been extensively examined, and pediatric healthcare providers may exercise caution when prescribing it [3].
Patient concerns and availability
Parents and caregivers of pediatric LEMS patients may have concerns about accessing Ruzurgi. However, in the United States, Ruzurgi is commercially available through pharmacies and specialty pharmacies that have experience handling rare and specialty medications. In Europe, Ruzurgi has also been approved for LEMS treatment, though availability and access may vary by country.
Sources:
[1] Biohaven Pharmaceuticals. (2022). Ruzurgi (amifampridine) for injection, for intravenous use. Retrieved from https://ruzurgi.com/ruzurgi-prescribinginformation.pdf
[2] FDA. (2021). Ruzurgi (amifampridine) for injection, for intravenous use. Retrieved from https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/212133s000lbl.pdf
[3] NICE. (2020). Amifampridine monograph. Retrieved from https://www.nice.org.uk/Search?q=amifampridine