Quick Facts:
MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative disease
MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adults and pediatric patients 2 years of age and older
Zevra receives rare pediatric disease priority review voucher in conjunction with approval.
As parents of children diagnosed with Niemann-Pick disease type C (NP-C), we understand the daily challenges and emotional rollercoaster that come with this journey. Today, we bring you a beacon of hope: the recent approval of Miplyffa (arimoclomol) by the U.S. Food and Drug Administration (FDA) as the first-ever treatment for NP-C.
What is Miplyffa?
Miplyffa is an oral medication designed to treat the neurological symptoms associated with NP-C. It works in combination with miglustat, another drug that helps manage the disease. This combination has shown promising results in slowing down the progression of NP-C, offering a new avenue of hope for families affected by this rare genetic disorder¹
How Does Miplyffa Work?
Miplyffa targets the cellular stress response, helping cells to function more effectively despite the genetic mutations that cause NP-C. In clinical trials, patients treated with Miplyffa experienced a slower progression of symptoms, particularly in areas such as mobility, speech, swallowing, and fine motor skills¹. This means that children and adults with NP-C can potentially enjoy a better quality of life for a longer period.
What Does This Mean for NP-C Families in Australia?
While Miplyffa has been approved by the FDA, it is essential for Australian families to stay informed about its availability and approval status within Australia. The Australian NPC Disease Foundation (ANPDF) is actively working to advocate for the swift approval and accessibility of Miplyffa in our country. We encourage you to stay connected with ANPDF for updates and to participate in advocacy efforts to bring this treatment to Australia.
The journey with NP-C is undoubtedly challenging, but as a community, we can support each other through these times.
Looking Ahead
The approval of Miplyffa marks a significant milestone in the fight against NP-C. It represents a step forward in providing our children with the treatments they deserve. Let us continue to support each other, stay informed, and advocate for the best possible care for our loved ones.
Together, we can make a difference.
To join our fight, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere."
Ways to support NP-C in Australia:
Check out upcoming events
Shop at our online NPC store
Fundraise your way! Check our free resources and inspo online.
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