Tradipitant

Johnson & Johnson proposed changes to the 340B drug discount program, but backed down after HRSA deemed them inconsistent with the statute. The 340B program, established in 1992, aims to help underserved communities by allowing eligible hospitals and clinics to purchase outpatient prescription medications at discounts. Recent regulatory developments include FDA approvals for Exact Sciences' Cologuard Plus, Partners Fresenius Kabi and Formycon's Otulfi, Eli Lilly's Retevmo, Sanofi and Regeneron's Dupixent, Bristol Myers Squibb's Cobenfy, and others. Additionally, Pfizer withdrew Oxbryta from the market due to safety concerns.

September 2024 FDA updates include approvals for Embecta's tubeless patch pump for diabetes, Travere Therapeutics' sparsentan for IgA nephropathy, Johnson & Johnson's guselkumab for ulcerative colitis, and the first over-the-counter hearing aid software device. Other approvals cover treatments for conditions like chronic rhinosinusitis with nasal polyps, atopic dermatitis, eosinophilic granulomatosis with polyangiitis, Niemann-Pick disease type C, and schizophrenia. The FDA also issued warnings and accepted NDAs for various treatments, including delgocitinib cream for chronic hand eczema and roflumilast foam for psoriasis.

September 2024 saw FDA approval of guselkumab (Tremfya) for ulcerative colitis and a CRL for Vanda Pharmaceuticals’ tradipitant for gastroparesis. Research highlighted treatment dissatisfaction in IBS patients, key bacteria affecting IBD response to thiamine, and FMT delivery methods for IBS. Upper GI mucosal damage was linked to Parkinson disease risk, while MASLD and MetALD increased liver and gastrointestinal cancer risks. The 2024 GHAPP conference featured discussions on medication positioning in Crohn disease and managing gut-brain interaction disorders.

Novo Nordisk shares fell 6% after mixed Phase 2 weight-loss drug results; Sanofi presented data supporting tolebrutinib for MS; Lasker Awards given to scientists for GLP-1 weight-loss drug research; Edgewise Therapeutics' heart failure drug shows promising early results; FDA declines to approve Vanda Pharmaceuticals' gastroparesis drug; House Energy and Commerce Committee supports extending pediatric rare disease voucher program.

Vanda Pharmaceuticals' drug tradipitant, intended to treat gastroparesis, was declined approval by the FDA, prompting a 14% share drop. The FDA requested additional animal studies and long-term safety data, raising concerns about chronic use. Vanda plans to continue pursuing approval despite the FDA's refusal to convene an expert panel.

The FDA declined Vanda Pharmaceuticals' NDA for tradipitant to treat gastroparesis, citing insufficient evidence. Vanda argues the FDA's decision was delayed and inconsistent with expert advice. Patients have filed a citizen petition urging approval, while Vanda plans to pursue marketing authorization and submit an NDA for motion sickness prevention by 2024.

FDA declined Vanda Pharmaceuticals' NDA for tradipitant to treat gastroparesis symptoms, despite evidence from studies and patient experience. Vanda plans to pursue marketing authorization and file another NDA for motion sickness prevention.

The FDA declined Vanda Pharmaceuticals' NDA for tradipitant to treat gastroparesis, citing insufficient evidence and suggesting further studies. Vanda argues the FDA's decision was delayed and inconsistent with expert advice, while patients have filed a citizen petition urging approval. Vanda remains committed to pursuing marketing authorization and supporting its expanded access program.

FDA declined to approve tradipitant for gastroparesis, citing need for additional studies. Vanda Pharmaceuticals criticized the decision, noting a positive phase II trial and mixed results from a phase III trial. Tradipitant remains investigational, with other NK1R antagonists approved for chemotherapy-induced nausea and vomiting.