FDA News

The hope is that NP001 could potentially slow the progression of the disease through its unique immunologic mechanism.

The decision follows positive data from multiple trials, showing significant improvement in both cardiac function and muscle performance in patients with DMD.

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.

Zevra Therapeutics plans to immediately start its launch activities for arimoclomol, marketed as Miplyffa, which is anticipated to be commercially available in 8 to 12 weeks in the United States.

The agent is composed of 2 active ingredients with synergistic anti-oxidant and anti-inflammatory effects which can reduce brain cell injury or impairment caused by acute ischemic stroke.

Approved based on the phase 3 OCARINA II trial, the new subcutaneous formulation offers patients with multiple sclerosis more flexibility to choose treatment options that suit their individual needs.

Catch up on any of the neurology news headlines you may have missed over the course of August 2024, compiled all into one place by the NeurologyLive® team.

The agency’s letter of support for using the α-synuclein seed amplification assay biomarker was supported by data observed in the Parkinson's Progression Markers Initiative cohort study.

Ashwini Sharan, MD, chief medical officer of Medtronic Neuromodulation, provided commentary on the FDA’s decision to approve asleep deep brain stimulation for the treatment of Parkinson disease and essential tremor.

Jim Cassidy, MD, PhD, chief medical officer at SpringWorks Therapeutics, talked about how the prioritization of mirdametinib, a promising treatment for both adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas.

NeuroQuant previously received clearance from the FDA as a medical device intended for automatic labeling, visualization, and volumetric quantification of segmentable brain structures in MRI analysis.

AXS-07 has been supported by data from 2 major phase 3 trials in which treatment with the agent led to significant reliefs in pain freedom, the most bothersome symptoms, and acute medication use.

The system is composed of the Neuroblade, a single-use multifunctional neuroendoscope; the Neuropad, a reusable medical-grade tablet; and Clearpath, a disposable transparent access sheath.

Mirdametinib, a treatment in development for adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas, was granted a PDUFA target action date of February 28, 2025.

The FDA has granted fast track designation to [18F]PI-2620 for 3 neurodegenerative conditions, highlighting its potential to improve early diagnosis and treatment.

The submission was based on results from the phase 3 Vivacity-MG3 study, which demonstrated nipocalimab’s superiority over placebo when added to standard of care in patients with generalized myasthenia gravis.

Prior to this, the FDA granted regenerative medicine advanced therapy designation to the cell therapy for refractory stiff-person syndrome.

Earlier this year, IDP-023 received fast track designation by the FDA for the treatment of patients with multiple myeloma and non-Hodgkin lymphoma.

Catch up on any of the neurology news headlines you may have missed over the course of July 2024, compiled all into one place by the NeurologyLive® team.

Marketed as Zunveyl for mild-to-moderate Alzheimer disease, ALPHA-1062 is considered a new-generation acetylcholinesterase inhibitor with expected minimal gastrointestinal adverse events.

The application was supported by positive data of KYV-101 in patients with stiff-person syndrome treated in Germany under a named-patient treatment option.

The expanded indication, supported by a phase 2 open-label trial, includes children of all ages with CLN2 disease, regardless of whether they are symptomatic or asymptomatic.

Tiziana Life Sciences’ intranasal foralumab has shown potential in treating nonactive secondary progressive multiple sclerosis through innovative immune modulation.

With the approved IND, it expands research efforts of equecabtagene autoleucel from other autoimmune conditions like myasthenia gravis and NMOSD.

Pending positive results from an early-stage trial, a mid-stage study evaluating MyoPAXon’s effect on other targeted muscles and muscle function will be initiated.

The investigational therapy, which targets neurodegeneration, has shown promise in phase 2 and 3 studies of Alzheimer disease and Parkinson disease, with additional studies on the way.

Lomecel-B is made from living cells called medicinal signaling cells that are isolated from fresh bone marrow tissue that has been donated by adult donors aged 18 to 45.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Sameea Husain-Wilson, DO. [LISTEN TIME: 19 minutes]

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Joel Salinas, MD, MBA. [LISTEN TIME: 10 minutes]

Overall, treated patients showed improvements in multiple cardiac measures, including left ventricular ejection fraction, as well as indexed volumes.