Sentences with phrase «breakthrough therapy designation»

The Breakthrough Therapy Designation is based on preliminary clinical results from the ongoing trial of AVXS - 101, conducted in collaboration with The Research Institute at Nationwide Children's Hospital and The Ohio State University.

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.

«We are encouraged to have received Breakthrough Therapy Designation for AVXS - 101, and look forward to collaborating with the FDA to determine next steps in the development pathway for AVXS - 101,» said Sean P. Nolan, president and chief executive officer, AveXis.

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

In April 2015, it received a U.S. Food and Drug Administration (FDA) Breakthrough Therapy designation.

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 treatments.

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.

But for all of the attention paid to its cancer drug pipeline, Bristol just scored a breakthrough therapy designation from the Food and Drug Administration for a treatment that targets hepatitis C.

Hutchison China Meditech (HCM)- The stock offers investors a strong pipeline of differentiated candidates (up to 15 possible Breakthrough Therapy Designations), China exposure (via its sales team of over 3,200 employees and 1,900 medical professionals), validating partnerships and several upcoming catalysts.

Not exact matches

For example, Zykadia, a drug for metastatic lung cancer, received a «breakthrough therapy» designation that allows expedited approval based on preliminary evidence for certain drugs that treat serious conditions.

He noted that the panel played a big role in shepherding into law a 2012 measure — the FDA Safety and Innovation Act (FDASIA)-- which created a new «breakthrough therapy» designation to speed the approval process for certain treatments for seriously ill patients.

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