FDA

Humacyte receives FDA RMAT designation for Humacyl in vascular access for hemodialysis

Monday, March 27, 2017

Humacyte, an innovator in biotechnology and regenerative medicine, announced that the FDA has granted Humacyl, its investigational human acellular vessel (HAV), the Regenerative Medicine Advanced Therapy (RMAT) designation. This designation means that the FDA will help facilitate the efficient development and expedited review of the HAV for vascular access to patients in need of life-sustaining hemodialysis. Carved from the model of other FDA expedited programs such as Breakthrough Therapy, the RMAT designation is a new expedited program for regenerative medicine products.

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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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Ocular Therapeutix publishes positive results of Dextenza following cataract surgery

Wednesday, March 15, 2017

Ocular Therapeutix, a biopharmaceutical company focused on the development, manufacturing and commercialization of innovative therapies for diseases and conditions of the eye, has announced positive results of a patient experience study of Dextenza (dexamethasone insert) 0.4mg for intracanalicular use. The study, published in Patient Preference and Adherence, evaluated the overall patient experience and perceived value of Dextenza following cataract surgery.

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The importance of providing education and awareness surrounding biosimilar products

Monday, March 13, 2017

The Pulse on Site Success by Christophe Berthoux

It has been seven years since the passing of the Biologics Price Competition and Innovation Act, a provision of the Affordable Care Act that created the approval pathway for biosimilars in the U.S. To date, four biosimilar products have been approved by the FDA, compared to 23 products in the European Union where biosimilars have been in play since 2007.

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