Published 09/25/2024 3:28:30 p.m.

FDA Approves Levacetylleucine for Niemann-Pick Disease Type C

Summary by hcplive.com

Announced on September 24, 2024, the approval marks the second treatment approved by the FDA for Niemann-Pick disease type C within one week.

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hcplive.com

FDA Approves Levacetylleucine for Niemann-Pick Disease Type C

Announced on September 24, 2024, the approval marks the second treatment approved by the FDA for Niemann-Pick disease type C within one week.

09/25/2024 2:25:21 p.m.

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neurologylive.com

FDA Approves Stand-Alone Thearpy Levacetylleucine for Niemann-Pick Disease Type C

The approval follows phase 3 data that showed significant improvements in neurological symptoms and functional benefit over 12 weeks in patients with Niemann-Pick disease type C.

09/25/2024 4:19:49 p.m.

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drugtopics.com

FDA Approves Levacetylleucine for Treatment of Niemann-Pick Disease Type C

The approval comes shortly after the FDA approved arimoclomol from Zevra Therapeutics to treat neurological manifestations of the disease in combination with miglustat.

09/25/2024 3:28:30 p.m.

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contemporarypediatrics.com

FDA approves levacetylleucine for the treatment of Niemann-Pick disease, type C

On average, participants treated with levacetylleucine for 12 weeks had better fSARA scores compared to when they received a placebo.

09/25/2024 3:31:12 p.m.

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pharmtech.com

FDA Approves Treatments for Niemann-Pick Disease, Type C

Two drugs, Miplyffa (arimoclomol) and Aqneursa (levacetylleucine), have been approved by FDA to treat neurological symptoms associated with Niemann-Pick disease, type C in adults and children.

09/25/2024 3:25:00 p.m.

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FDA Approves Levacetylleucine for Niemann-Pick Disease Type C

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FDA Approves Levacetylleucine for Niemann-Pick Disease Type C

FDA Approves Levacetylleucine for Niemann-Pick Disease Type C

FDA Approves Stand-Alone Thearpy Levacetylleucine for Niemann-Pick Disease Type C

FDA Approves Stand-Alone Thearpy Levacetylleucine for Niemann-Pick Disease Type C

FDA Approves Levacetylleucine for Treatment of Niemann-Pick Disease Type C

FDA Approves Levacetylleucine for Treatment of Niemann-Pick Disease Type C

FDA approves levacetylleucine for the treatment of Niemann-Pick disease, type C

FDA approves levacetylleucine for the treatment of Niemann-Pick disease, type C

FDA Approves Treatments for Niemann-Pick Disease, Type C

FDA Approves Treatments for Niemann-Pick Disease, Type C

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