Cell therapy was recently described as “One of the most promising new fields of science and medicine,” by FDA Commissioner Scott Gottlieb. He went on to state that cell therapy and regenerative medicine “…hold significant promise for transformative and potentially curative treatments for some of humanity’s most troubling and intractable maladies.” In the FDA statement issued August 28th, Gottlieb also revealed that the U.S. regulator is looking improve upon its approval process for cell therapies. [Read more…]
• Trial was recently awarded $8.7 million grant from the European Horizon 2020 program
• Pluristem plans to use results of trial to achieve marketing approval in both the U.S and
HAIFA, Israel, Sept. 26, 2017 — Pluristem Therapeutics Inc. (NASDAQ:PSTI) (TASE:PSTI), a leading developer of placenta-based cell therapy products, has today reported the status of its planned Phase III hip fracture study. In previous discussions held with the U.S. Food and Drug Administration (FDA) and the European Medicine Agency (EMA), the company received positive feedback on the proposed study design and endpoints of its Phase III trial in PLX-PAD cells as a treatment for muscle recovery following arthroplasty for hip fracture. This trial was recently awarded an $8.7 million grant by the Horizon 2020 program, the European Union’s largest research and innovation program.
The proposed Phase III trial is intended to support marketing authorization and will be a double-blind, randomized, placebo-controlled trial with approximately 180 patients enrolled in clinical sites across the U.S. and Europe. Patients will be injected intramuscularly with 150 million PLX-PAD cells during arthroplasty for hip fracture. The primary endpoint for the trial will be the change of Short Physical Performance Battery (SPPB) from baseline to six months after surgery. Additional endpoints may include objective measurements of muscle strength and muscle volume.
This Phase III trial follows positive results from a Phase I/II trial which demonstrated significant muscle regeneration when PLX-PAD cells were injected following arthroplasty, including a 300% improvement in muscle volume (p=0.004) and a 500% (p=0.0067) boost in muscle force when observed six months after surgery compared to the control group. [Read more…]
Pluristem’s Ongoing Phase III CLI trial has been selected for accelerated approval pathways in both the U.S. and Europe
Fast Track Designation allows for expedited review of drugs to treat serious conditions with unmet medical need
HAIFA, Israel, Sept. 18, 2017 (GLOBE NEWSWIRE) — Pluristem Therapeutics, Inc. (NASDAQ:PSTI) (TASE:PSTI), a leading developer of placenta-based cell therapy products, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to the company’s ongoing Phase III study of PLX-PAD cells for the treatment of Critical Limb Ischemia (CLI) in patients ineligible for revascularization. The FDA’s Fast Track Designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and unmet medical needs. With Fast Track Designation, there is an increased possibility for a priority review by the FDA of PLX-PAD cells for the treatment of CLI.
“We are extremely pleased with the FDA’s decision to grant Fast Track Designation to PLX-PAD in the treatment of CLI. Up to 40% of patients with CLI are ineligible for revascularization and are at high risk of amputation and death within the first year of diagnosis. This disease takes a heavy toll on patients and their families, while the cost of treating CLI in the U.S. alone is estimated at over $25 billion per year. We are working tirelessly to provide a cell therapy that will address this severe unmet medical need,” said Zami Aberman, Chairman and Co-CEO of Pluristem.
“Regulators in some of the largest healthcare markets in the world are now in alignment regarding the need for accelerated approval pathways for our cell therapy product in the treatment of CLI. Programs like the Fast Track Designation offer real hope for patients battling this disease and we look forward to accelerating the path to market for PLX-PAD,” stated Pluristem President and Co-CEO, Yaky Yanay. [Read more…]
CAR T-cell therapy approved to treat certain children and young adults with B-cell acute lymphoblastic leukemia
U.S. FDA, August 30, 2017 – The U.S. Food and Drug Administration issued a historic action today making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases. [Read more…]
Over the past few years, the regulatory landscape for cell therapy development has grown increasingly complex. There are now accelerated pathways for advanced therapy medicinal products (ATMPs) in several countries worldwide, including the U.S., Japan, and South Korea. While the possibility for accelerated commercialization has resulted from these changes, substantial complexity has also been introduced, making it a more elaborate process to move cell therapy products from “bench to bedside.”
In the interview with Yaron Ramati, Director of Regulatory Affairs at Pluristem Therapeutics, we get an expert’s perspective on how the regulatory environment has changed and new opportunities that exist for bringing cell therapy products through the clinical trial process and into the global marketplace. [Read more…]