October is Niemann-Pick Awareness Month. FDA approved a treatment for type 3 of the ultra-rare genetic disease September 20. The quest has been ongoing for three decades, said Laurie Turner, Family Services Manager of the National Niemann-Pick Disease Foundation.
Miplyffa (arimoclomol), an oral drug taken three times a day, is prescribed for patients with Niemann-Pick disease type 3, aka NPD3, two years of age and older.
NPD3 typically begins with enlarged liver and spleen and progresses to neurological symptoms. Average length of life is 13 years.
"Impacts on patients and families are enormous," said Janet Maynard, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine at FDA's Center for Drug Evaluation and Research. "Despite extensive research efforts, there have not been approved treatments to meet the significant needs of patients. The first-ever approval of a safe and effective drug option will undoubtedly support the essential medical needs of those suffering."
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