Drug Therapy

In a review of the 2024 milestones, Healio | Endocrine Today compiled a list of drugs and devices related to the field that were newly approved, cleared or had label or indication changes. Information was gathered from the FDA website and news releases. We put our best effort into making a complete list and regret any omission. Appearance of a drug or device does not constitute any endorsement by Healio | Endocrine Today, its editors, publishers or editorial board. — The Editors

Treatment with an oral allosteric modulator was nonsuperior to placebo at 6 weeks in adults with Parkinson’s disease whose symptoms included fall risk and cognitive issues, data from JAMA Neurology show.

Topline results from a phase 2b/3 clinical trial of blarcamesine for individuals with early-stage Alzheimer’s disease showed that earlier initiation of the oral therapeutic led to significant disease slowing over 3 years.

Topline results from a phase 2/3 clinical trial showed that an eIF2B agonist to treat ALS failed to meet its primary endpoint of slowing disease progression compared with placebo at week 24.

According to results from the exploratory phase 2 SHIMMER clinical trial, a small molecule oligomer antagonist improved outcomes in patients with dementia with Lewy bodies.

There has been little research on optimal treatments for pediatric patients with pulmonary arterial hypertension, but the Kids MoD PAH Trial is expected to change this by clearing up if sildenafil plus bosentan or sildenafil only is better.

Zevra Therapeutics has announced that Miplyffa, the first Nieman-Pick disease type C treatment approved by the FDA, is commercially available.

For individuals with ALS, a daily dosing regimen of oral edaravone was nonsuperior to an FDA-approved staggered regimen of the drug plus placebo, according to a poster presented at AANEM.

Treatment with oral blarcamesine for older adults with Alzheimer’s disease led to significant slowing of clinical decline compared with placebo at 48 weeks, according to new research presented at CTAD.

An oral therapeutic significantly reduced the number of tumors in adults with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas, according to topline results from a phase 3 study.