In this new weekly column, Editor Freya Leask rounds up her news highlights from the last 7 days.
This week: FDA issues strong warning, diabetes gene therapy shows promise in mice, the Cell and Gene Therapy Catapult unveils new project and Takeda eyes an acquisition
In a warning letter, the FDA has warned American CryoStem Corporation (NJ, USA) over the marketing of an unapproved stem cell therapy. On recent inspection, the FDA found that American CryoStem were manufacturing an autologous adipose-tissue derived therapy and were processing it beyond ‘minimal manipulation’ and, as such, requires an FDA review to ensure safety. The FDA also found evidence of ‘significant deviations’ from current GMP requirements, such as inadequately controlled environment and components, and lack of suitable product testing.
“The use of Atcell raises potential significant safety concerns, due in part to the fact that there is little basis on which to predict how the product will perform in a patient,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research. “In addition, this product may also cause harm to patients who may put their trust in an unproven therapy and make the decision to delay or discontinue medical treatments proven to be safe and effective.”
Read the full press release here.
Pluristem Therapeutics Inc. (Israel) has received FDA clearance for an expanded access program to treat patients with critical limb ischemia (CLI) with their PLX-PAD cell therapy. CLI is advanced stage of peripheral artery disease and patients unsuitable for revascularization are left with no other treatment options. PLX-PAD utilize placental cells to support the body’s own repair mechanisms through secretion of therapeutic proteins. PLX-PAD will now be available to a limited number of CLI patients in the US that are unsuitable to take part in the current Phase III clinical trials enrolling patients in the US and Europe.
“This is a true vote of confidence by the FDA in our cell therapy and a landmark achievement for Pluristem and its shareholders. It gives us the ability to begin treatments using our cell product, offering treatment to certain CLI patients who have poor therapeutic options, while also collecting real-world data alongside our ongoing Phase III clinical study,” commented Yaky Yanay, Co-CEO and President of Pluristem. “We are hopeful that the FDA may also allow us to be compensated for the costs of treatment, which can support our work developing effective cell therapies for millions of patients worldwide.”
Read the article here
Takeda Pharmaceutical Company (Japan) has announced its intention to acquire TiGenix NV (Belgium), its partner for the license, development and commercialization of Cx601, an allogeneic expanded adipose-derived stem cell therapy. In December 2017, the European Medicines Agency recommended a marketing authorization for Cx601 for patients with non-active/mildly active luminal Crohn’s disease that have had an inadequate response to at least one conventional or biologic therapy. Cx601 is the first allogeneic stem cell therapy to achieve this. Through the potential voluntary public takeover bid, Takeda will pay approximately EU$520 million.
Read the full press release here
Cell and gene therapies currently have complex manufacturing and administration procedures, often leading to very low numbers of centers approved to administer them. For example, Strimvelis, a gene therapy for the treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), has one approved treatment center in Milan (Italy). With the Advanced Therapy Treatment Centers, the Cell and Gene Catapult hope to enable these advanced therapies to be administered throughout the UK.
Read the full article here