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RT002 Granted FDA Orphan Drug Designation

NOVEMBER 21, 2017
Mathew Shanley
Revance Therapeutics’ lead drug candidate DaxibotulinumtoxinA for Injection (RT002), intended to treat cervical dystonia, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA).

RT002 is also currently in development for the treatment of glabellar lines and plantar fasciitis. Current standard of care for cervical dystonia includes oral medications, botulinum toxin injections, surgery and complimentary therapies.

Cervical dystonia, commonly referred to as spasmodic torticollis, is characterized by involuntary contractions in the neck muscles, which can cause twisting and turning of the head. The motions can be either sustained (tonic) or jerky (clonic). In some cases, patients can experience a combination of both. Additionally, some patients experience a combination of abnormal head postures, including the head being pulled toward the shoulder. Neck pain associated with the condition can radiate into the shoulders and cause excruciating headaches.

“In our Phase 2 trial, RT002 appeared to be generally safe and well-tolerated and showed a clinically significant reduction and long-lasting improvement of subjects’ symptoms,” said Dan Browne, President and CEO at Revance in a press release. “We are pleased that the FDA granted Orphan Drug Designation for this promising therapy. Once we integrate the Orphan Drug Designation benefits into our development plans and receive feedback from both the FDA and the European regulatory authority on our proposed clinical path, we plan to announce the next steps for our cervical dystonia program in early 2018.”

Cervical dystonia can occur in either gender and at any age, but most commonly presents in women after the age of 30. In most cases, there isn’t a known cause of the condition, leading many to believe that there is a genetic component.

Per the Mayo Clinic, cervical dystonia can spread to areas of the body near to the head and neck, including the face, jaw, arms, and trunk. I not treated effectively, it’s possible that the condition can cause the development of bone spurs which result in tingling, numbness and weakness in the limbs.

Current botulinum toxin treatments for cervical dystonia have a duration of effect of an estimated 3 months. RT001 has the potential to be the first long-acting neuromodulator.

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