Sentences with phrase «granted breakthrough therapy»

Chicago, Ill., July 20, 2016 — AveXis, Inc., a clinical - stage gene therapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS - 101, the company's lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patients.

The US Food and Drug Administration (FDA) has granted a Breakthrough Therapy designation to an antibody - drug conjugate known as enfortumab vedotin.

AveXis, Inc., a clinical - stage gene therapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXS - 101.

Not exact matches

Because so many of the patients in the early testing showed significant long - lasting responses, the study was continued and the FDA granted the drug «breakthrough therapy» status, allowing it to be fast - tracked for approval.

In July 2014, the U.S. Food and Drug Administration granted CTL019 its Breakthrough Therapy designation for the treatment of relapsed and refractory adult and pediatric ALL, a step which is intended to expedite the development and review of new medicines that treat serious or life - threatening conditions, if a therapy has demonstrated substantial advantages over available treaTherapy designation for the treatment of relapsed and refractory adult and pediatric ALL, a step which is intended to expedite the development and review of new medicines that treat serious or life - threatening conditions, if a therapy has demonstrated substantial advantages over available treatherapy has demonstrated substantial advantages over available treatments.

Based in part on these promising findings, the FDA granted MPDL3280A a «Breakthrough Therapy» designation.