Grünenthal receives Breakthrough Therapy Designation from U.S. FDA for neridronic acid forthe treatment of complex regional pain syndrome (CRPS), a debilitating orphan disease with high medical need
- Neridronic acid is the first investigational medicine to receive Breakthrough Therapy Designation for the treatment of CRPS
- Neridronic acid was discovered and developed by Abiogen Pharma SpA, Pisa, Italy
- Currently in Phase III of clinical development, neridronic acid, could be the first FDA-approved treatment in the U.S. for CRPS, an orphan disease with severe, persistent pain without sufficiently effective treatment options today
Grünenthal, an R&D-driven, privately held international pharmaceutical company with headquarters in Germany, and Abiogen Pharma, privately held Italian pharmaceutical company with headquarter in Pisa, Italy announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to neridronic acid, an investigational medicine, for the treatment of complex regional pain syndrome (CRPS), a serious, disabling orphan disease. Today, with no FDA- or EMA-approved drug treatments, there is a clear need for effective treatment options to address this significant unmet medical need. CRPS is a debilitating condition characterized by severe, continuous, burning pain often occurring in anextremity after injury or surgery. It is one of the most painful conditions a patient can experience.
The Breakthrough Therapy Designation is supported by data from a randomized, double-blind, placebo-controlled phase II clinical trial showing significant reduction in pain and symptoms of CRPS-I with neridronic acid treatment. Neridronic acid is a new chemical entity (NCE) and investigational drug in the U.S. It received fast track designation in August 2015 and orphan drug designation in March 2013 by the FDA.
"The terrible burden for patients with CRPS motivates us every day to deliver true benefits to them," said Dr. Klaus-Dieter Langner, Chief Scientific Officer of Grünenthal. "It is very encouraging to see that the FDA recognizes the urgent need for new treatments for patients with CRPS and has granted neridronic acid the status of a Breakthrough Therapy. This supports our efforts to develop an efficacious treatment option to these patients. We are committed to working closely with the FDA to bring neridronic acid to patients with CRPS as fast as possible."
Gabriel Baertschi, CEO of the Grünenthal Group, points out, "Grünenthal is highly dedicated to improving the lives of patients with pain as well as rare diseases with limited treatment options. This is an area of high unmet medical need. As a worldwide leader in pain, our focus is to develop potentially life-changing treatments for patients with various diseases accompanied with pain. Through the acquisition of Thar Pharmaceuticals, we have recently added an orally available form of zoledronic acid to our pipeline which is to enter phase III development for treatment of CRPS. Together with neridronic acid as our lead compound in CRPS, our platform of promising development candidates for this debilitating disease is growing."
"We are very pleased to see how the ongoing collaboration with Grünenthal on a molecule born in our Research Center is evolving, and we are looking forward to continuing our relationship," said Dr. Massimo Di Martino, President and CEO of Abiogen Pharma. "Working to potentially improve the lives of patients with CRPS is very rewarding and we are committed to do everything supporting Grünenthal in its efforts."
The FDA's Breakthrough Therapy Designation is intended to expedite the development and review of medicines that treat a serious or life threatening disease and show early evidence of potential clinical benefit in such diseases, to help ensure that patients receive access to medicines as soon as possible.
Grünenthal obtained the development and commercialization rights for North America and South America in 2013.