Healio

FDA approves second Niemann-Pick disease type C treatment

Please provide your email address to receive an email when new articles are posted on . The FDA has approved Aqneursa as an oral treatment for neurological symptoms associated with Niemann-Pick ...
Business Wire

Positive Niemann-Pick disease type C (NPC) and GM2 gangliosidosis data from nizubaglustat Phase 2 RAINBOW study conducted by Azafaros presented at major metabolic disease ...

LEIDEN, Netherlands--(BUSINESS WIRE)--Azafaros has announced that data from the ongoing double-blind, placebo-controlled Phase 2 RAINBOW study investigating its lead asset, nizubaglustat in patients ...
Monthly Prescribing Reference

Miplyffa Approved for Niemann-Pick Disease Type C

Findings showed that among patients who also received miglustat, treatment with arimoclomol slowed NPC disease progression. The Food and Drug Administration (FDA) has approved Miplyffa ™ (arimoclomol) ...
Morningstar

Mandos Health to Present New Analyses in Niemann-Pick Disease Type C at the 2025 American Neurological Association Annual Meeting

Mandos Health® by Beren Therapeutics Public Benefit Corporation (P.B.C.) announced today that new analyses detailing the clinical and biological effects of its investigational drug, adrabetadex, in ...
Nasdaq

Zevra Launches Niemann-Pick Disease Type C Drug Miplyffa In The U.S.

(RTTNews) - Zevra Therapeutics Inc. (ZVRA), a rare disease therapeutics company, Thursday, announced the commercial availability of Miplyffa for the treatment of Niemann-Pick disease type C along with ...
WNDU

Notre Dame celebrates first FDA-approved treatment for Niemann-Pick Type C disease

SOUTH BEND, Ind. (WNDU) - The University of Notre Dame is celebrating a big medical breakthrough that’s been decades in the making. Within the past month, the FDA has announced the approval of new ...
BioWorld

Zevra’s Miplyffa wins first FDA approval in Niemann-Pick type C

Following the recent recommendation of its advisory committee, the U.S. FDA gave the nod to Zevra Therapeutics Inc.’s arimoclomol, a day ahead of its Sept. 21 PDUFA date, as the first treatment for ...
Morningstar

Azafaros Announces Initiation of two Global Phase 3 studies with Nizubaglustat in Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses, respectively

First patient dosed in global pivotal clinical trial program for late infantile/juvenile onset NPC and GM1/GM2 gangliosidoses. Both studies are part of Azafaros’ mission to develop treatments for the ...
Business Wire

Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C

LEIDEN, Netherlands--(BUSINESS WIRE)--Azafaros B.V. today announced positive topline data from its RAINBOW study, a Phase 2 clinical trial investigating nizubaglustat in patients with a genetic ...
Monthly Prescribing Reference

FDA Approves Aqneursa for Niemann-Pick Disease Type C

The recommended dosage is based on the patient’s body weight. The Food and Drug Administration (FDA) has approved Aqneursa ™ (levacetylleucine) for the treatment of neurological manifestations of ...
Nasdaq

FDA Approves Zevra Therapeutics' MIPLYFFA For Treatment Of Niemann-Pick Disease Type C

(RTTNews) - Zevra Therapeutics Inc. (ZVRA) said that the U.S. Food and Drug Administration approved MIPLYFFA (MY-PLY-FAH) (arimoclomol) capsules as an orally delivered treatment for Niemann-Pick ...
Healio

FDA approves Miplyffa for Niemann-Pick disease type C

Please provide your email address to receive an email when new articles are posted on . The FDA announced in a press release that it has approved Miplyffa to treat neurological symptoms associated ...