美国食品药物管理局(FDA)于2011年11月10日宣布，核准由美国纽约捐血中心(NYBC)申请的脐带血造血前躯细胞(Hematopoietic progenitor cells-cord blood；HPC-C)- Hemacord 的生物制品执照申请(biologics license application；BLA)，成为第一个获FDA核准的干细胞产品。

  Hemacord (BLA125397)

Hemacord是用于脐带血异体移植的造血干细胞产品，可重建因先天性、后天性与化疗引起的血液与免疫系统缺失疾病，例如血液恶性病、贺勒氏疾病(MPS-I)、脑白质萎缩症(Krabble disease)、X性联性肾上腺脑白质失养症、免疫不全症、骨髓衰竭与乙型地中海型贫血。

Hemacord产品含有5x10⁸有核细胞总数(TNC)、1.25x10⁶造血干细胞(CD34+ cells)与10%抗冻剂(DMSO)，以2.5x10⁷/kg 剂量与HLA4/6以上配对吻合度进行静脉注射，其药品成效与病患的疾病、发病时期、危险因子、副作用与移植方式有关。

Hemacord在移植后的100天会有25%死亡率风险，因此在使用包装上会标示警语，说明移植后可能会有严重输血后反应(Fatal infusion reaction)、 移植物抗宿主反应(GVHD)、植入症状(Engraftment syndorme)与移植失败(Graft failure)，让医师能针对病患情况做移植前的评估。

FDA approves first cord blood product

The U.S. Food and Drug Administration today approved HEMACORD, the first licensed hematopoietic progenitor cells-cord (HPC-C) cell therapy. 

HEMACORD is indicated for use in hematopoietic stem cell transplantation procedures in patients with disorders affecting the hematopoietic (blood forming) system. For example, cord blood transplants have been used to treat patients with certain blood cancers and some inherited metabolic and immune system disorders.

“The use of cord blood hematopoietic progenitor cell therapy offers potentially life-saving treatment options for patients with these types of disorders,” said Karen Midthun, M.D., director, FDA’s Center for Biologics Evaluation and Research.

HEMACORD contains hematopoietic progenitor cells (HPCs) from human cord blood. Cord blood is one of three sources of HPCs used in transplants; the other two are bone marrow and peripheral blood. Once these HPCs are infused into patients, the cells migrate to the bone marrow where they divide and mature. When the mature cells move into the bloodstream they can partially or fully restore the number and function of many blood cells, including immune function.

In an effort to assist manufacturers in applying for licensure for certain cord blood units, FDA issued the 2009 guidance document entitled “Guidance for Industry: Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications.” FDA instituted a two-year phase-in period for HPC-C manufacturers to submit either a license application or an investigational new drug application. That phase-in period ended Oct. 20, 2011, and these manufacturers now must submit such applications.

Approval of HEMACORD was based on reliance on safety and effectiveness data submitted to a public docket and data submitted in the license application demonstrating compliance with other regulatory requirements. This is the first approval of a license application for cord blood.

HEMACORD has a boxed warning regarding the risks of Graft Versus Host Disease (GVHD), engraftment syndrome, graft failure, and infusion reactions, each of which may be fatal. Patients who receive HEMACORD should be monitored carefully. A risk benefit assessment, unit selection and administration of HEMACORD should be done under the direction of a physician experienced in hematopoietic stem cell transplantation.

HEMACORD is manufactured by the New York Blood Center, Inc., based in New York, NY.

FDA NEWS RELEASE
For Immediate Release: November 10, 2011

http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm279575.htm