Three years after Qalsody was approved by the FDA as the first drug to target a genetic cause of ALS, some patients are reporting not only disease stabilization but also improvement of symptoms.

Original title: “Biogen’s targeted ALS treatment is reversing decline in some patients. Can more be helped?”

FDA today issued an Emergency Use Authorization (EUA) for CLiK Extra (dicyclanil topical suspension) wound spray for application on or around wounds for the prevention of New World screwworm (NWS) infestations (myiasis).

Original title: “FDA Issues Emergency Use Authorization for Drug to Prevent New World Screwworm in Multiple Species, Including Sheep, Cattle, Goats, and Swine”

The Phase 2 study was small and was not designed to establish the statistical difference between Vistagen’s drug candidate and placebo, according to Vistagen. The biotech plans to meet with the FDA to determine a registrational path for the asset.

Original title: “Vistagen’s repeat-dose anxiety nasal spray can’t beat placebo in mid-stage study”

Postmenopausal study highlights oral anabolic potential

Original title: “Entera Bio data selected for ASBMR plenary session”

Replimune’s immunotherapy for advanced melanoma faced a perilous regulatory road with two previous rejections, but the drug—to be marketed as Tudriqev—prevailed with strong support from oncologists and a 10-3 advisory committee vote in its favor.

Original title: “Third time’s the charm for Replimune as melanoma drug earns FDA greenlight”

BioVie’s stock fell by nearly 50% as the biotech claimed victory in a mid-stage Parkinson’s disease trial using an alternative endpoint to the one submitted to the FDA.

Original title: “BioVie shares halve on murky Parkinson’s disease readout”

An FDA nod mostly clears the way for Orzeyful to enter, by Wall Street estimates, a multibillion-dollar market for drugs that boost “orexin” proteins.

Original title: “Takeda approval ushers in new wave of sleep drugs”

Orzeyful is the first orexin agonist cleared by the FDA, and is the first narcolepsy therapy that addresses the full host of disease symptoms. It is "likely to reshape the treatment paradigm” for the condition, Jefferies told investors.

Original title: “Takeda’s narcolepsy nod opens orexin doors”

The FDA in February slapped Moderna’s vaccine with a refusal-to-file letter—though the agency backtracked a few days later and accepted a revised submission for review. An advisory committee in June unanimously backed mFLUVISA’s approval.

Original title: “Moderna’s flu shot crosses FDA finish line, bouncing back from regulatory roadblock”

Adaptive Clinical Trial Design ... Disease Pediatric Oncology Platform Trials PSYCH PULSE Real-World Evidence site activation Site Operations study startup Treatment-Resistant Depression ... Three CNS trial design decisions that rarely appear together in the same protocol just appeared in one study, and the clinical operations ... Adaptive Clinical Trial Design Adaptive Trial Design Addiction Psychiatry AI in Clinical Trials alcohol use disorder Article Artificial Intelligence in Clinical Trials Clinical Operations Clinical Research Clinical Trial Design Clinical Trial Endpoints Clinical Trial Operations Clinical Trial Ops Brief Clinical Trial Quality Clinical Trials CLINOPS WATCHDOG CNS Drug Development CNS Trials Conference Coverage Data Analysis Decentralized Clinical Trials Digital Health Technologies dual diagnosis eClinical Tech News eClinical Technology EMA Executive Interviews FDA Enforcement FDA Guidance FDA Re A dronabinol RCT in Nature Medicine just outperformed prazosin's VA trial with 170+ PTSD patients. Here's what the trial design gets right that most CNS… The gap between site capability and trial requirement here is real, and the eCOA vendors who build it into their configurability roadmap before the next wave of PTSD nightmare trials launch will capture the configuration contracts that matter. The 2023 VA/DoD Clinical Practice Guideline for PTSD contains 34 evidence-based recommendations and still lacks a strong pharmacological recommendation for nightmare-specific treatment after prazosin failed at scale. Prazosin, the alpha-1 blocker that VA clinicians used for years, failed to separate from placebo in the large multisite Prazosin and Combat Trauma PTSD trial published in the New England Journal of Medicine, leaving a gap that the 2023 VA/DoD Clinical Practice Guideline for the Management of Posttraumatic Stress Disorder has never cleanly filled. That vacuum is exactly what the dronabinol trial entered.

Original title: “The PTSD Nightmare Drug No One Expected: What Dronabinol’s RCT Tells Us About CNS Trial Design”