Industry Update: Regulations, approvals, acquisitions…
Latest developments compiled from 01 — 31 December 2014
Latest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from non-academic institutions 01 — 31 December 2014, scheduled to be published in Volume 10 Issue 3 of Regenerative Medicine.
Green light
Chiesi
The European Medicines Agency (EMA) has recommended Holoclar, the first advanced therapy medicinal product (ATMP) containing stem cells, for approval in the European Union (EU). Holoclar, made by Chiesi Farmaceutici (Italy; www.chiesigroup.com/en/home), is a treatment for moderate to severe limbal stem cell deficiency (LSCD) due to physical or chemical burns to the eye(s) in adults. It is the first medicine recommended for LSCD, a rare eye condition that can result in blindness. The recommendation was made by the Committee for Medicinal Products for Human Use (CHMP) based on a robust assessment carried out by the Committee for Advanced Therapies (CAT), the Agency’s expert committee for ATMPs.
Cytori
Cytori (CA, USA; www.cytori.com) has received from the US FDA a conditional approval for an Investigational Device Exemption (IDE) for a pivotal clinical trial, named the ‘STAR’ trial, to evaluate Cytori Cell Therapyâ„¢ as a potential treatment for impaired hand function in scleroderma, a rare autoimmune disease affecting approximately 50,000 patients in the US. The STAR trial is conditionally approved to be a randomized, double blind, placebo-controlled pivotal clinical trial of 80 patients in up to 12 US sites. The trial evaluates the safety and efficacy of a single administration of Cytori Cell Therapy in scleroderma patients affecting the hands and fingers. More than 90 percent of scleroderma patients have hand involvement that is typically progressive and can result in chronic pain, blood flow changes and severe dysfunction. The limited available treatments for scleroderma may provide some benefit but do little to modify disease progression or substantially improve symptoms. Cytori’s cell therapy processing technology, known as the Celution® System, is regulated as a Class III medical device by the US FDA and requires a successful pivotal trial and premarket authorization (PMA) before it can be marketed in the US. The Celution System produces an autologous cell product from adipose tissue known as adipose-derived regenerative cells (ADRCsâ„¢). The STAR trial is predicated on a completed pilot trial, SCLERADEC-I. In data presented earlier this year, patients in SCLERADEC-I showed, on average, 50% improvement at six months across four important and validated endpoints used to assess the clinical status in patients with systemic scleroderma with impaired hand function.
Acquisitions and mergers
GPI and RMF
Two leading stem cell and regenerative medicine advocacy groups, The Genetics Policy Institute (FL, USA; GPI; www.genpol.org) and the Regenerative Medicine Foundation (RMF; NC, USA; www.regenerativemedicinefoundation.org), have agreed to merge in order to leverage their resources in the advancement of stem cell research, regenerative medicine and other advanced technologies targeting cures. They made a joint announcement today at the opening session of the 10th Annual World Stem Cell Summit in San Antonio, Texas. The merged organization is to be called the Regenerative Medicine Foundation. The Genetics Policy Institute will become the think tank and policy arm of the merged organization. Bernard Siegel, executive director of GPI, will serve as executive director of the new Regenerative Medicine Foundation. Dr. Anthony Atala, founder and chairman of the board of RMF, will sit on the board of directors of the merged organization.