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US FDA cleared pricey rare disease drug over reviewer objections

On Wednesday, Manitoba Health Minister Uzoma Asagwara approved the Health Canada‑approved Miglustat for five‑year‑old Leo Dupont and said he will ask the federal government for help obtaining a second drug, Miplyffa.
Dupont's June diagnosis revealed Niemann‑Pick Disease, Type C, a rare disorder diagnosed in one out of every 150,000 people annually that causes cholesterol buildup and is usually fatal by about age 13.
A local anonymous couple offered to use their Mayo Clinic connections to help the family and may assist in getting Miplyffa through Rochester contacts, while Zevra Therapeutis did not respond and has not brought the drug to Canada.
The department is already working with the family to support next steps so they can begin the medication, but it is unclear if the family will pay part of the cost, which Manitoba Health's non‑insured benefits will determine with no time frame, Asagwara said.
Because the manufacturer hasn't brought Miplyffa to Canada, it cannot be sold without Health Canada approval, and Asagwara said he is contacting Health Canada to engage Zevra Therapeutis.

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