Clinical Intelligence

FDA grants Breakthrough designation for Rituxan (Rituximab) in pemphigus vulgaris

Friday, March 24, 2017

Genentech, a member of the Roche Group, has announced that the FDA granted Breakthrough Therapy Designation status to Rituxan (rituximab) for pemphigus vulgaris, a rare, serious and life-threatening condition characterized by progressive painful blistering of the skin and mucous membranes. FDA Breakthrough Therapy Designation is intended to expedite the development and review of medicines with early evidence of potential clinical benefit in serious diseases and to help ensure that patients receive access to medicines as soon as possible. Genentech is currently enrolling a phase III study in pemphigus vulgaris (PEMPHIX, NCT02383589), a disease for which there are limited treatment options. 

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NantKwest granted FDA Orphan Designation for aNK natural killer cell therapy in merkel cell carcinoma

Monday, March 20, 2017

NantKwest, a pioneering, next generation, clinical-stage immunotherapy company focused on harnessing the unique power of our immune system using natural killer (NK) cells to treat cancer, infectious diseases and inflammatory diseases, announced that the FDA has granted Orphan Drug Designation to the company’s activated natural killer (aNK) cell therapy for treatment of patients with advanced Merkel cell carcinoma.

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PTC Therapeutics to acquire Emflaza for Duchenne muscular dystrophy

Thursday, March 16, 2017

PTC Therapeutics has entered into an asset purchase agreement with Marathon Pharmaceuticals to acquire all rights to Emflaza (deflazacort). Emflaza is the first treatment approved in the U.S. for all Duchenne muscular dystrophy (DMD) patients five years and older, regardless of their genetic mutation. DMD is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death due to heart and respiratory failure. Emflaza aligns with PTC’s mission to bring therapies to patients who have rare diseases with limited or no treatment options.

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Alkermes initiates phase III study of ALKS 8700 for multiple sclerosis

Thursday, March 16, 2017

Alkermes has announced the initiation of a new phase III study of ALKS 8700, a novel, oral monomethyl fumarate (MMF) prodrug candidate in development for the treatment of relapsing forms of multiple sclerosis (MS). ALKS 8700 is designed to rapidly and efficiently convert to MMF in the body and offer differentiated features as compared to the currently marketed dimethyl fumarate, TECFIDERA. The five-week, head-to-head study will evaluate the gastrointestinal (GI) tolerability of ALKS 8700 compared to TECFIDERA in approximately 420 patients with relapsing-remitting MS (RRMS). This elective study is part of the ongoing clinical development program for ALKS 8700, named EVOLVE-MS (Endeavoring to Advance Treatment for Patients Living with Multiple Sclerosis). The company plans to submit a New Drug Application (NDA) for ALKS 8700 for the treatment of RRMS to the FDA in 2018.

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