This week we have remdesivir news; A new opioid and spinal muscular atrophy treatments are approved; A sublingual epinephrine treatment; And the latest on Biogen’s investigational Alzheimer disease treatment. Also, we have a brief summary of the other drugs in the pipeline from the past week.
Evrysdi is a survival motor neuron-2 (SMN2) splicing modifier.
New data from the phase 2 NURTURE trial evaluating nusinersen (Spinraza®; Biogen) demonstrate that presymptomatic patients with spinal muscular atrophy (SMA) continue to benefit from treatment for up to 4.8 years.
Patients with spinal muscular atrophy (SMA) treated with a one-time infusion of onasemnogene abeparvovec-xioi (Zolgensma®;AveXis) continue to show clinically meaningful benefits even 5 years after dosing.
Risdiplam is an investigational, orally administered liquid survival motor neuron-2 (SMN2) splicing modifier designed to increase and sustain SMN protein levels throughout the central nervous system and peripheral tissues of the body.
Recently announced results from part 2 of the SUNFISH trial, which evaluated the safety and efficacy of risdiplam in patients with type 2 or 3 SMA aged 2 to 25 years, showed that the treatment met its primary end point.
Genentech announced positive data from the pivotal part 2 of the SUNFISH trial that evaluated the safety and efficacy of risdiplam in adult and pediatric patients with Type 2 or Type 3 spinal muscular atrophy (SMA).
The Food and Drug Administration (FDA) has placed a partial hold on clinical trials for the intrathecal administration of AVXS-101 (AveXis, a Novartis Company) in patients with spinal muscular atrophy (SMA) Type 2.
Positive data from phase 2 study of Spinraza for preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy.
According to the Food and Drug Administration (FDA), AveXis Inc, the manufacturer of the gene therapy Zolgensma, was aware of an issue related to “data manipulation” prior to approval, but did not inform the Agency until after the product was approved.
