幹細胞論壇

美國食品藥物管理局(FDA)於2011年11月10日宣布，核准由美國紐約捐血中心(NYBC)申請的臍帶血造血前軀細胞(Hematopoietic progenitor cells-cord blood；HPC-C)- Hemacord 的生物製品執照申請(biologics license application；BLA)，成為第一個獲FDA核准的幹細胞產品。

  Hemacord (BLA125397)

Hemacord是用於臍帶血異體移植的造血幹細胞產品，可重建因先天性、後天性與化療引起的血液與免疫系統缺失疾病，例如血液惡性病、賀勒氏疾病(MPS-I)、腦白質萎縮症(Krabble disease)、X性聯性腎上腺腦白質失養症、免疫不全症、骨髓衰竭與乙型地中海型貧血。

Hemacord產品含有5x10⁸有核細胞總數(TNC)、1.25x10⁶造血幹細胞(CD34+ cells)與10%抗凍劑(DMSO)，以2.5x10⁷/kg 劑量與HLA4/6以上配對吻合度進行靜脈注射，其藥品成效與病患的疾病、發病時期、危險因子、副作用與移植方式有關。

Hemacord在移植後的100天會有25%死亡率風險，因此在使用包裝上會標示警語，說明移植後可能會有嚴重輸血後反應(Fatal infusion reaction)、 移植物抗宿主反應(GVHD)、植入症狀(Engraftment syndorme)與移植失敗(Graft failure)，讓醫師能針對病患情況做移植前的評估。

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