FDA Approves Designation of IV Baclofen as Orphan Product

Minneapolis, Minn.

Jul. 1, 2020

IV Baclofen, a muscle relaxant drug, benefits individuals who experience dystonia (abnormal muscle tension) or spasticity (muscle stiffness or rigidity) caused by cerebral palsy, multiple sclerosis, brain or spinal cord injury. The drug is currently available as oral tablets and as an intrathecal solution delivered through a pump into the spinal column. However, if illness or mechanical problems with the intrathecal pump occur, or if a patient cannot take the drug by mouth, baclofen administration can be interrupted, causing withdrawal symptoms. The intravenous formulation of the drug will allow more effective management and prevention of complications associated with abrupt drug withdrawal.

The orphan product designation application was submitted by Allaysis, the company developing IV baclofen with whom the University has a license agreement. It was submitted in close collaboration with faculty in the college's Center for Orphan Drug Research including Linda Krach, MD; Bob Kriel, MD; Lisa Coles, PhD; and James Cloyd, PharmD. The drug formulation previously passed a Phase I study designed by Lisa Coles and recent graduate student, Natalie Schmitz, PharmD, MPS, PhD.

The next step in the development of the product is for Allaysis to submit a new drug application to the FDA in the next month with an anticipated Agenct approval sometime in the scond half of 2021.