BioCentury | May 12, 2020
Product Development

May 11 Quick Takes: Genfit’s elafibranor misses in NASH Phase III; plus AstraZeneca, Merck, Protalix and TolerogenixX

Genfit’s PPAR agonist fails to resolve NASH  Genfit S.A. (Euronext:GNFT; NASDAQ:GNFT) fell $10.88 (49%) to $11.22 in after-hours trading Monday after reporting that dual PPARα/δ agonist elafibranor missed the primary endpoint of the Phase III...
BC Extra | Mar 15, 2017
Politics & Policy

NICE updates appraisal policies

Under a new assessment system for its Highly Specialized Technologies program for rare diseases, the U.K.'s NICE replaced the fixed £100,000 ($122,220) threshold it had previously proposed with a sliding scale that could permit automatic...
BioCentury | Jan 28, 2017
Regulation

Ultra-rare, ultra-slow

NHS England and NICE have grappled for years with how to evaluate ultra-Orphan drugs, but neither has applied a cost-effectiveness analysis in funding decisions - until now. The proposed £100,000 threshold would not factor into NICE’s...
BC Innovations | Dec 10, 2015
Product R&D

Spark in the brain

Spark Therapeutics Inc. is expanding beyond eye and blood diseases with its first foray into the brain for the rare neuronal disorder Batten disease, using a gene therapy delivered to the ependymal cells in the...
BC Extra | Aug 21, 2014
Top Story

Amicus gains after Amigal meets in Phase III

Amicus Therapeutics Inc. (NASDAQ:FOLD) rose $0.93 (20%) to $5.50 on Wednesday after reporting that Amigal migalastat ( AT1001 ) met the co-primary endpoints of comparability to enzyme replacement therapy (ERT) on two measures of kidney function in...
BC Week In Review | Aug 18, 2014
Company News

Evotec, Shire deal

In May, Evotec partnered with Shire under a three-year deal to discover small molecule inhibitors against a target to treat Fabry’s disease. Evotec will use high throughput screening, fragment-based screening, computational chemistry and structure-based medicinal...
BC Week In Review | Jul 21, 2014
Company News

Shire, Sanofi, Icahn School of Medicine at Mount Sinai, Knowledge Ecology International endocrine/metabolic news

Non-profit Knowledge Ecology International sent a letter to the U.S. Federal Trade Commission requesting the agency investigate a March 2012 decision by Shire to withdraw a BLA for Replagal agalsidase alfa, effectively abandoning efforts to...
BioCentury | Jul 21, 2014
Strategy

AbbVie's plan B

Clinical setbacks and a rapidly evolving HCV landscape have left AbbVie Inc. less insulated from potential biosimilar competition for blockbuster Humira adalimumab. Now that Shire plc has decided to accept AbbVie's takeout bid, the specialty...
BC Extra | Jul 16, 2014
Company News

Non-profit wants FTC to investigate Replagal saga

Knowledge Ecology International sent a Shire plc (LSE:SHP; NASDAQ:SHPG) to withdraw a BLA for Replagal agalsidase alfa, effectively abandoning efforts to market the Fabry's disease drug in the U.S. According to the non-profit KEI, Shire's...
BioCentury | May 20, 2013
Strategy

R&D rewind

At first blush, new Shire plc CEO Flemming Ornskov's plan to invest in early R&D while integrating business units looks like a rehash of the company's strategy from 2000. But Ornskov expects the latest move...
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