FOR IMMEDIATE RELEASE
September 25, 2024

FDA approves Miplyffa for NPC

Today is a monumental day for the NPC Community and the first thing Chris and I wanted to do was share it with you.

In a historic and unprecedented move this morning, the FDA approved the first treatment for Niemann-Pick Type C (NPC), Arimoclomol, now referred to as Miplyffa. This novel breakthrough therapy promises to slow the progression of NPC and enhance the quality of life for NPC patients. Chris and I are eager to add Miplyffa to the therapeutic arsenal for Belle & Abby to have the best chance of rendering NPC a chronic, instead of fatal, disease.

Not only does approval demonstrate the FDA’s willingness to use regulatory flexibility for rare disease patients, but also importantly, serves as a pillar of hope for the entire rare disease community because it shows advocates globally that the FDA is willing, ready AND equipped to employ creativity and ingenuity when evaluating drugs for ultra-rare diseases.

Today’s win also underscores the agency’s understanding that NPC needs multiple drugs to treat multiple pathways in order to truly render this disease chronic. We do not plan, and the doctors do not plan, to take our girls off any medications they currently receive. Miplyffa will be taken in combination with what they are already taking.

We are deeply grateful to each of you. A special acknowledgment goes out to the NPC moms’ group that amplified the voice of the NPC patient over the past six years. To everyone who believed that a therapy could be found and approved for NPC, thank you!

Today, we celebrate a hard-fought victory. We will rest for a bit and will start again next week. On Tuesday September 24th, we anticipate the approval of another drug by IntraBio, IB1001, also for NPC. And then we will get NPC added to every newborn screening list worldwide. And then we will do it over again for each pediatric neurodegenerative rare disease. One at a time and with your help.

Our mission continues.

With heartfelt gratitude,

Pam and Chris Andrews