TAMPA, Fla. – Pacira BioSciences, Inc. (NASDAQ:PCRX) announced that its gene therapy candidate, PCRX-201, has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for the treatment of osteoarthritis of the knee. This marks the first instance the FDA has awarded RMAT status to a gene therapy for osteoarthritis.
The RMAT designation for PCRX-201, also known as enekinragene inzadenovec, is based on preliminary data from a Phase 1 study involving 72 patients. The study explored the safety and efficacy of the therapy, which is administered directly into the joint and delivers a gene coding for the interleukin-1 receptor antagonist (IL-1Ra), a protein that inhibits inflammation associated with osteoarthritis.
Results from the trial, which included patients in two cohorts—one receiving a co-administered intra-articular steroid and one without—indicated that PCRX-201 was well tolerated. Efficacy was observed for at least 52 weeks across all doses. The most significant improvements were seen in the cohort that received the steroid, with a notable percentage of patients experiencing at least a 50% reduction in pain and stiffness, and enhanced functional assessment scores.
The RMAT designation, established under the 21st Century Cures Act, aims to expedite the development and review of promising therapies that treat serious conditions and fill an unmet medical need. It allows for early interactions with the FDA and potential for accelerated approval processes.
Pacira's CEO, Frank D. Lee, expressed optimism about the therapy's potential as a disease-modifying treatment for osteoarthritis. The company plans to present 52-week data at the Osteoarthritis Research Society International (OARSI) 2024 World Congress in April and expects to submit 104-week data later in the year.
PCRX-201 was acquired from GQ Bio Therapeutics GmbH and utilizes a high-capacity adenoviral gene therapy vector to produce therapeutic levels of IL-1Ra in the presence of inflammation, specifically targeting joint cells and remaining localized to the joint space.
Pacira, known for its non-opioid pain management solutions, holds a portfolio including EXPAREL, ZILRETTA, and iovera°. The company's mission is to reduce reliance on opioids, providing alternatives for pain management.
This announcement is based on a press release statement from Pacira BioSciences, Inc.
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