Mallinckrodt Receives FDA Fast-Track Designation for Synacthen® Depot IND Application

August 25, 2016

Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, today announced the U.S. Food and Drug Administration (FDA) has granted the company's request for a Fast Track designation for its Investigational New Drug (IND) application for Synacthen® Depot in the treatment of Duchenne muscular dystrophy (DMD).

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Tivorsan Pharmaceuticals Granted FDA Orphan Drug Designation for Human Recombinant Biglycan in the Treatment of Duchenne Muscular Dystrophy

August 15, 2016

ivorsan Pharmaceuticals, Inc., a biotechnology company that is pioneering a novel approach to treating all forms of Duchenne Muscular Dystrophy (DMD) and other serious, debilitating neuromuscular disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead investigational drug, human recombinant Biglycan, for the treatment of DMD.

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FDA Accepts Marathon Pharmaceuticals’ New Drug Applications for Deflazacort for the Treatment of Duchenne Muscular Dystrophy and Grants Priority Review

August 10, 2016

Marathon Pharmaceuticals, LLC, a biopharmaceutical company developing treatments for rare diseases, today announced the New Drug Applications (NDA) for the investigational drug deflazacort have been accepted for filing and granted Priority Review by the U.S. Food and Drug Administration (FDA).

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PhaseBio Announces Dosing of First Patients in Two-Part Phase 2a Study of PB1046 in Cardiopulmonary Disorders

August 8, 2016

PhaseBio Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on biopolymer-based drugs for orphan, cardiopulmonary and metabolic diseases, today announced the dosing of the first patients in Part One of a Phase 2a clinical study of PB1046, a once-weekly vasoactive intestinal peptide (VIP) receptor agonist in development for the treatment of cardiopulmonary disorders. Part One will assess PB1046 in adult subjects with stable heart failure with reduced ejection fraction. Part Two, which PhaseBio expects to initiate later in 2016, will examine PB1046 in subjects with cardiac dysfunction secondary to Duchenne muscular dystrophy.

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August 1, 2016

Pfizer Inc. (NYSE:PFE) today announced that it has acquired Bamboo Therapeutics, Inc., a privately held biotechnology company based in Chapel Hill, N.C., focused on developing gene therapies for the potential treatment of patients with certain rare diseases related to neuromuscular conditions and those affecting the central nervous system. This acquisition significantly expands Pfizer’s expertise in gene therapy by providing Pfizer with a clinical and several pre-clinical assets that complement the company’s rare disease portfolio, an advanced recombinant Adeno-Associated Virus (rAAV) vector design and production technology, and a fully functional Phase I/II gene therapy manufacturing facility that Bamboo acquired from the University of North Carolina earlier this year.

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