Clinical Intelligence

Abeona receives FDA breakthrough designation for epidermolysis bullosa

Wednesday, August 30, 2017

Abeona Therapeutics, a clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced that the FDA has granted Breakthrough Therapy designation status to the company’s EB-101 gene therapy program for patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB). The designation from the FDA enables collaborative discussions with senior FDA personnel, priority review and an expedited approval process to drug candidates where preliminary clinical trials indicate that a therapy may offer substantial treatment advantages over existing options for patients with serious or life-threatening diseases.

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FDA grants Priority Review for Genentech’s Gazyva

Tuesday, August 29, 2017

Genentech, a member of the Roche Group, announced that the FDA has accepted the company’s supplemental Biologics License Application (sBLA) and granted Priority Review for Gazyva (obinutuzumab) in combination with chemotherapy followed by Gazyva alone for people with previously untreated follicular lymphoma, one of the most common blood cancers among adults. Follicular lymphoma, a slow-growing (indolent) form of non-Hodgkin’s lymphoma, is incurable and characterized by cycles of remission and relapse.

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Boehringer Ingelheim initiates phase IIa study of liver disease NASH compound

Tuesday, August 29, 2017

Boehringer Ingelheim and pharmaceutical company Pharmaxis announce that Boehringer Ingelheim has initiated a European and North American phase IIa trial in NASH with BI 1467335 (formerly known as PXS-4728A), acquired from Pharmaxis in May 2015. The compound is an oral inhibitor of amine oxidase, copper containing 3 (AOC3), and works by blocking leucocyte adhesion and tissue infiltration in inflammatory processes underlying NASH.

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Novartis launches smartphone research study in multiple sclerosis

Friday, August 25, 2017

Novartis announced the launch of a mobile research study for people with multiple sclerosis (MS) that collects data via their smartphone, without the need for clinic visits. The study, Evaluation of Evidence from Smart Phone Sensors and Patient-Reported Outcomes in Participants with Multiple Sclerosis (elevateMS), is designed to collect sensor-based data from physical tasks and symptoms. It aims to improve understanding of the daily challenges patients with MS can have and to uncover new potential measurements of treatment effectiveness through real-time data collection from participants in their everyday life.

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FDA approves Ironwood’s duzallo for gout

Thursday, August 24, 2017

Ironwood Pharmaceuticals announced DUZALLO was approved by the FDA as a once-daily oral treatment for hyperuricemia associated with gout in patients who have not achieved target serum uric acid (sUA) levels with a medically appropriate daily dose of allopurinol alone. DUZALLO is not recommended for the treatment of asymptomatic hyperuricemia. Ironwood expects DUZALLO to be commercially available early in the fourth quarter of 2017.

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