Clinical Intelligence

Regenxbio’s RGX-111 gets FDA Rare Pediatric Disease designation

Monday, January 4, 2016

Regenxbio, a Rockville, Md.-based biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy, today announced that the FDA has granted Rare Pediatric Disease designation to RGX-111, the company’s investigational gene therapy product candidate for the treatment of mucopolysaccharidosis type I (MPS I).

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Minerva completes enrollment in MIN-101 phase IIb trial

Monday, January 4, 2016

Minerva Neurosciences, a Waltham, Mass.-based clinical-stage biopharmaceutical company focused on the development of therapies to treat central nervous system (CNS) disorders, has announced that the FDA has accepted the company’s Investigational New Drug (IND) application for MIN-101, a first-in-class 5-HT2a and sigma2 antagonist in clinical development for the treatment of schizophrenia.

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FDA approves emergency treatment for chemotherapy overdoses

Monday, December 14, 2015

The FDA has approved Vistogard (uridine triacetate) for the emergency treatment of adults and children who receive an overdose of the cancer treatment fluorouracil or capecitabine, or who develop certain severe or life-threatening toxicities within four days of receiving those cancer treatments.

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Treeway sees positive data results for ALS treatment

Thursday, December 10, 2015

Treeway, a biotech company developing therapies against amyotrophic lateral sclerosis (ALS), today announced the completion of two successful phase I trials with its lead compound TW001, a more patient-friendly oral formulation of the Japanese stroke and ALS therapy edaravone. [Read More]

Zafgen’s Beloranib IND gets complete clinical hold after second death

Monday, December 7, 2015

Zafgen, a Boston-based biopharmaceutical company dedicated to significantly improving the health and well-being of patients affected by obesity and complex metabolic disorders, has received verbal notice from the FDA that its beloranib investigational new drug (IND) application has been placed on complete clinical hold, affecting the ongoing open label extension (OLE) portion of the pivotal phase III ZAF-311 bestPWS clinical trial in patients with Prader-Willi syndrome (PWS). A complete clinical hold is an order that the FDA issues to a sponsor to suspend all clinical work requested under the company’s IND application.

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