Nearly all patients in this cohort achieved a clinically meaningful >3-point increase during the study period, demonstrating a consistent response to the gene therapy.
Catabasis’s small molecule NF-kB inhibitor showed promise in phase 2, and the phase 3 PolarisDMD study (NCT03703882) of edasalonexant is fully enrolled and expected to read out in late 2020.
Although the drug requires no CYP2C9 genotyping or first-dose observation, an up-titration scheme should be used due to the risk of transient cardiac conduction issues.
SK Life Science’s newly approved treatment for partial-onset seizures is set to launch in the second quarter of 2020, after being designated Schedule V by the DEA.
The report suggests with moderate certainty that all 3 therapies offer a small or substantial net health benefit and with a high certainty of at least a small net health benefit.
Disease-modifying therapies used to treat several immune-mediated disorders in neurology may make patients more susceptible to the novel coronavirus.
The drug was previously approved for the treatment of acute repetitive seizures and granted 7 years of orphan drug exclusivity in January 2020.
The investigational MS therapy is currently under review with the FDA, with a PDUFA date set for June 2020.
Rimegepant, the only orally disintegrating anti-CGRP tablet, marketed as Nurtec ODT, will be available in a 75-mg dose.
The VMAT2 inhibitor failed to meet the primary end point in the ARTISTS 1 and ARTISTS 2 trials in the treatment of tics in pediatric patients with Tourette syndrome.