Miplyffa treats Niemann-Pick disease type C, which progressively impacts the abilities to speak, swallow, and walk or move independently.
the first treatment for a rare genetic disorder often diagnosed in childhood that can cause damage to the nerves and brain and lead to organ failure over time. People with the condition live for about 13 years, on average.
Niemann-Pick disease type C, or NPC, “is a serious disease that leads to enormous adverse impacts on patients and families. Despite extensive research efforts, there have not been approved treatments to meet the significant needs of patients,” said Janet Maynard, MD, MHS, director of the FDA’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in a statement.
People taking Miplyffa with miglustat for 12 months halted disease progression, as measured by a scale for symptom severity, according to aIn its approval announcement, the FDA characterized the treatment as resulting in a “slower disease progression.” As part of its application, Zevra Therapeutics also submitted data from a 48-month study following the initial trial, the company said.in support of a statement that the drug had been shown effective.
United States Latest News, United States Headlines
Similar News:You can also read news stories similar to this one that we have collected from other news sources.
FDA OKs Adjuvant Ribociclib in Earlier Stage Breast CancerUnlike rival CDK 4/6 inhibitor abemaciclib, patients don't need to be lymph-node positive to receive ribociclib after surgery.
Read more »
FDA OKs Subcutaneous Atezolizumab Formulation for Multiple Cancer IndicationsSubcutaneous injections save time, taking about 7 minutes, vs 30-60 minutes for intravenous infusion.
Read more »
Biden holds first Cabinet meeting in nearly a year, first lady joins for first timePresident Biden convened a meeting of his Cabinet -- this time with First Lady Jill Biden attending -- for the first time Friday in nearly a year.
Read more »
US FDA Lifts Partial Clinical Hold on Rezolute's Low Blood Sugar TreatmentRezolute Inc said on Monday the U.S. Food and Drug Administration has removed the partial clinical hold on its late-stage study testing its treatment for low blood sugar,...
Read more »
Weight-loss drug safe, effective for kids as young as 6, study saysSaxenda was the FDA's first approved drug to treat obesity in adults back in 2014.
Read more »
First At-Home Syphilis Test Approved by FDA amid Nationwide Syphilis SurgeAn easy-to-access rapid test could lower barriers to detecting syphilis, which is surging nationwide
Read more »