Newswise — Cell therapy company jCyte announced that the U.S. Food & Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for the company’s developmental retinitis pigmentosa (RP) treatment, jCell. RMAT seeks to accelerate approval for regenerative therapies that demonstrate the potential to address an unmet medical need.
“This is uplifting news for patients with RP,” said jCyte co-founder Henry Klassen, MD, PhD. “At this point, there are no therapies that can help them avoid blindness. We look forward to working with the FDA to speed up the clinical development of jCell.”
RP is a genetic condition in which retinal cells (rods and cones) undergo progressive degeneration. The disease usually strikes people in their teens and many patients are legally blind by the time they reach 40. Worldwide, nearly 1.5 million people suffer from RP, making it the leading cause of inherited blindness.
jCell contains human retinal progenitor cells (hRPCs), which release neurotrophic factors that may rescue diseased retinal cells, as well as possibly integrating into the retina as new cells. This investigational therapy is delivered with a simple intravitreal injection, performed under topical anesthesia, in a matter of minutes.
Part of the 21st Century Cures Act, RMAT designation facilitates expedited reviews and approvals for stem cell and other cellular and tissue therapies that manage serious or life-threatening conditions. An eligible therapy must show preliminary clinical evidence that it has the potential to address an unmet need.
“This is among the first ophthalmic products to receive RMAT designation.” said jCyte CEO, Paul Bresge. “We are gratified by the FDA’s interest in the therapeutic potential of jCell and greatly appreciate their decision to provide extra support. We are now conducting the all-important trials to validate that confidence.”
The RMAT designation is the latest in a series of positive reports from jCyte. Enrollment in the company’s phase 1/2a trial was completed in July 2016. In April 2017, they announced a phase 2b trial to investigate jCell’s efficacy. This study is recruiting around 70 patients, who will receive single injections in their worst-sighted eye, and is supported by an $8.3 million grant from the California Institute for Regenerative Medicine (CIRM). jCell has been granted orphan drug designation by both the FDA and the European Medicines Agency (EMA).
“We are seeing a lot of momentum with this therapy,” said Bresge. “Because it is well-tolerated and easy to administer, progress has been rapid. I feel a growing sense of excitement among patients and clinicians. We look forward to getting this critical therapy over the finish line as quickly as possible.”