Oxycodegol has reduced permeability across the blood-brain barrier, so it may cause less euphoria and sedation. In July 2019, the FDA put review of NKTR-181 on hold and canceled an advisory committee review of the drug. On 1/14/2020 the FDA's Anesthetic and Analgesic Drug Products Advisory Committee and Drug Safety and Risk Management Advisory Committee voted 27-0 against approval, due to abuse potential of the drug and lack of adequate efficacy studies. Nektar Therapeutics withdrew the NDA and ceased development of oxycodegol due to the decision.
Roche indicated they plan to price risdiplam lower than nusinersen and onasemnogene abeparvovec when the drug is approved. Risdiplam has a PDUFA date of 5/24/2020.
The EMA approved osilodrostat (Isturisa) in January 2020 for the treatment of endogenous Cushing’s syndrome.
Sarepta initiated a rolling NDA submission for casimersen for the treatment of Duchenne muscular dystrophy amenable to exon 43 skipping.
TG Therapeutics initiated a rolling NDA submission for umbralisib for patients with previously treated marginal zone lymphoma and follicular lymphoma.
Published Research Updates
In the 12-month, 362 patient, Phase III, TULIP 2 trial, 47.8% of patients treated with anifrolumab had a decrease in disease activity (BICLA response) compared to 31.5% with placebo in patients with systemic lupus erythematosus.
In two randomized, crossover, dose-escalation studies, treatment with gefapixant in doses of 30 mg or > decreased cough frequency and severity compared to placebo in patients with refractory chronic cough.
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