Pluristem Therapeutics ($PSTI) has bagged itself a spot on the European Medicines Agency's (EMA) adaptive pathways pilot project. The EMA's decision gives Pluristem a shot at slashing the time it takes to bring its PLX cell program in critical limb ischemia (CLI) to market in a subpopulation.
|Pluristem CEO Zami Aberman|
The EMA created the adaptive pathway pilot program to test the feasibility of shifting more of the burden of data generation to post-approval. Instead of an all-or-nothing charge at a large Phase III trial, companies following the adaptive pathway can file for approval in a subpopulation based on limited data. The company can then expand the indication at a later date based on data generated through subsequent clinical trials and real-world evidence gathering initiatives. Alternatively, companies can apply for conditional approvals that are tied to the collection of post-marketing data.
Haifa, Israel-based Pluristem was the first company to reveal it has been accepted into the program. The EMA received 34 applications when it opened the program, 6 of which were for advanced-therapy medicinal products, the regulator's catch-all term for treatments based on genes, cells or tissue engineering. A further 12 applications were for orphan medicines. Pluristem survived the EMA's cull of the applications--which whittled the pool down to 6 promising candidates late last year--and has now been accepted in to the program.
Participation in the program is by no means a guarantee that the CLI program will succeed, but it does give Pluristem access to some extra regulatory support and a chance of an early approval. "Reducing time to market is a critical element of our strategy," Pluristem CEO Zami Aberman said in a statement. A Phase II trial of Pluristem's cell therapy for CLI is still at the design stage. But with abbreviated pathways available to the company in Japan and Europe, Aberman is aiming to bring the treatment to market within three years.
Some big outstanding questions need answering if Pluristem is to pull off its plan. The business is based upon a process of expanding cells from full-term human placentas in bioreactors. When the cells are given to a patient, they are thought to reverse tissue damage by releasing cytokines, chemokines and growth factors. The idea has yet to be tested in late-phase trials, with an ongoing Phase II in patients with intermittent claudication the most advanced program. A severe allergic reaction, FDA clinical hold and controversies over patient deaths have marred earlier studies.
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