FDA News

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.

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.

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.

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

The approval of donanemab marks the third antiamyloid therapy to gain the FDA's greenlight, following the conditional approval of aducanumab in 2021, and lecanemab in 2023—although aducanumab no longer remains on market after it was removed earlier this year.

In the CRL, the agency cited observations identified during inspection of a third-party manufacturer listed in the NDA. If approved, ABBV-951 would be the first subcutaneous delivery option of carbidopa/levodopa.

Pitolisant is the first and only drug in the class of antagonist/reverse agonists of the histamine H3 receptor for the treatment of patients with narcolepsy.

The therapy is the first and only neonatal Fc receptor blocker approved for the treatment of CIDP, and will be available as a once-weekly, 30- to 90-second subcutaneous injection.

The AAV vector-based gene therapy originally received FDA approval for treating ambulatory pediatric patients aged 4 to 5 years with Duchenne under the accelerated approval pathway.

The cleared phase 1/2 clinical trial will assess SPG302 as a once-a-day pill among patients with amyotrophic lateral sclerosis.

AMT-130, uniQure’s investigational gene therapy, is the first therapeutic candidate to receive regenerative medicine advanced therapy designation for Huntington disease.

Deflazacort, a corticosteroid approved for children with Duchenne muscular dystrophy, has been on the market since 2017 under the name Emflaza.