Serotonin

The FDA approved Kebilidi (eladocagene exuparvovec-tneq) for AADC deficiency, making it the first brain-administered therapy in the US. Developed by PTC Therapeutics, Kebilidi is a gene replacement therapy targeting the putamen, restoring dopamine synthesis and improving motor development. The approval is based on ongoing clinical trial data, with EU and UK approvals as Upstaza in 2022 and 2023, respectively.

The FDA granted accelerated approval for Kebilidi, a gene therapy for AADC deficiency, a rare genetic disorder causing severe disability. Developed by PTC Therapeutics, Kebilidi is the first U.S. gene therapy directly administered to the brain, delivered via a stereotactic surgical procedure. It showed efficacy in a trial with 13 pediatric patients, with 8 meeting new gross motor milestones, compared to none in an untreated cohort. Common adverse reactions include dyskinesia and procedural complications.

Researchers, including Dr. Lynnette Averill, study psychedelics for PTSD treatment in veterans, finding rapid symptom improvement in some cases. Despite challenges, psychedelic-assisted therapy shows promise for mental health issues like depression and anxiety, offering a potential lifesaving alternative to traditional treatments.

Ms Anderson, a 36-year-old with a history of major depressive disorder and recurrent pregnancy losses, experienced a stillbirth at 34 weeks, leading to increased anxiety and sadness. Perinatal loss is common, affecting 15% of pregnancies, with disparities among racial and ethnic groups. Psychiatric sequelae include grief, postpartum depression, anxiety, and PTSD. Treatment strategies involve nonpharmacologic approaches like support groups and psychotherapy, with pharmacologic options considered for severe cases.

New Jersey considers legalizing psilocybin for mental health treatment, with strict regulations including license requirements, medical supervision, and data collection. Research supports psilocybin's potential benefits for depression, anxiety, PTSD, and addiction. If passed, New Jersey would join Oregon and Colorado in regulating psilocybin for therapeutic use.

Lumateperone (Caplyta) added to existing antidepressants significantly improved depression symptoms in a phase III trial, with significant MADRS score improvement by day 43. Lumateperone, already approved for schizophrenia and bipolar depression, showed efficacy as early as day 8. The study involved 484 patients with major depressive disorder who had inadequate response to prior antidepressant therapy. Lumateperone also improved CGI-S and QIDS-SR-16 scores, with stable weight and BMI, and no notable changes in extrapyramidal symptoms. A supplemental new drug application for adjunctive treatment of major depressive disorder is expected by end of 2024.

Seltorexant, a potent, selective orexin-2 receptor antagonist, improved symptoms of major depressive disorder (MDD) and insomnia in a phase III trial. The study drug, when combined with current antidepressant treatment, showed a significant reduction in Montgomery-Åsberg Depression Rating Scale (MADRS) total score compared to placebo. Seltorexant is unique as it targets only OX2 receptors, potentially filling an unmet need for new therapies for MDD and insomnia.

FDA issued a Complete Response Letter to Lykos Therapeutics for midomafetamine capsules (MDMA) for PTSD, citing insufficient data. Despite concerns about efficacy and safety, experts remain hopeful for future approval. The FDA also released draft guidelines for psychedelic drug research, emphasizing proper study design and safety assessments.