→ The UK’s pharma price watchdog NICE has sanctioned coverage of GlaxoSmithKline’s gene therapy Strimvelis for “bubble baby” syndrome, which costs about $700,000 for the once-and-done therapy. About three babies a year are born with ADA-SCID in the UK every year, a condition characterized by a life-threatening case of immune deficiency that leaves children unprotected against lethal infections. NICE says this is the first OK where it used a different pricing guideline for extraordinarily rare diseases. The gene therapy is available in only one site in Italy, where UK patients will have to travel to for therapy.
→ Shares of Evoke Pharma shot up 22% $EVOK on its release citing positive top-line results from a pharmacokinetic study of a nasal delivery formulation of metoclopramide for diabetic gastroparesis. The biotech is now planning an NDA filing in early 2018 for Gimoti. “We are very pleased this study has met our objective of demonstrating bioequivalence on the measure of AUC to identify a Gimoti dose,” said Dave Gonyer, Evoke Pharma’s CEO.
→ Reata Pharmaceuticals $RETA enrolled the first patient in Part II of its pivotal study of omaveloxolone in patients with Friedreich’s ataxia. Reata is expecting top-line data in the second half of 2019.
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