Doron’s biologic osteoarthritis treatment gets FDA boost

Doron Therapeutics, a clinical-stage biotechnology company based in Chapel Hill, has received a Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for MOTYS, the company’s lead osteoarthritis biologic drug. The FDA also granted MOTYS Fast Track designation.

"Securing RMAT designation is a significant milestone for MOTYS that underscores the promise of our clinical evidence and the potential for this technology to improve the life of millions of patients whose lives are limited by the symptoms of knee osteoarthritis," said Alessandra Pavesio, CEO of Doron Therapeutics. "We are very pleased that the FDA has provided us with a pathway for expedited approval of MOTYS and look forward to working in close collaboration with the Agency to bring MOTYS to the patients we serve."

The RMAT designation helps accelerate the development and review of regenerative medicine therapies for treating serious conditions with unmet needs. RMAT status comes with more frequent and focused guidance from the FDA and can open the door to a faster review and approval of a Biologics License Application, helping promising treatments reach patients sooner. Similarly, Fast Track designation is designed to speed up developing and reviewing drugs that treat serious conditions and address unmet medical needs. It provides benefits like more frequent communication with the FDA and a potentially faster approval process.

Advancing osteoarthritis treatment

MOTYS provides a promising alternative to total knee replacement, which is often the only option for those with severe osteoarthritis. The placental tissue-derived therapy enables a unique multimodal approach to treating osteoarthritis that helps manage inflammation, slow joint damage and support tissue repair. Donated by mothers after planned healthy C-section births, placental tissue contains natural healing molecules and is safely accepted by the body without triggering an immune response.

Doron's RMAT and Fast Track designation requests were supported by a body of preclinical and clinical data available on MOTYS, especially clinical findings from its Phase 2 dose-finding study of 215 patients, which identified durable, dose-dependent effects of MOTYS on pain mitigation and improved functional outcomes.

“Obtaining RMAT and Fast Track designations for MOTYS is another major milestone for Doron,” said Sarah Caley, head of business development and strategy at Doron. “It allows us to collaborate further with FDA and to access multiple potential regulatory benefits, including accelerated and priority review approval pathways, access to senior FDA reviewers and the opportunity to discuss use of surrogate or intermediate endpoints to support accelerated approval.”

Read our previous coverage of Doron and the MOTYS osteoarthritis treatment:

Doron Therapeutics raises $11M to advance osteoarthritis biologic into Phase 3 trials