Real-World Experience With Beti-cel for Patients With TDT Is Consistent With Trials
The real-world data support the recently published results from a phase 3 study, which showed most patients achieved transfusion independence.
The first 4 real-world patients treated with betibeglogene autotemcel (beti-cel; LentiGlobin; bluebird bio) for transfusion-dependent β-thalassemia (TDT) exhibited tolerability that was consistent with what investigators observed during clinical trials, and none of the 4 individuals required red cell transfusion as far as 332 days post-infusion treatment.
Andreas Kulozik, Dr. med, PhD, medical director of the clinic for Pediatric Oncology, Hematology, Immunology and Pulmonology, at University Medical Center for Children and Adolescents in Heidelberg, Germany, presented these findings in a poster during the Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR (2022 Tandem Meetings), held in Salt Lake City, Utah, and virtually April 23-26, 2022.1
The real-world data support the recently published results2 from a phase 3 study (HGB-207; NCT02906202) in which most adult and pediatric patients with transfusion-dependent β-thalassemia and a non–β0/β0 genotype treated with beti-cel achieved transfusion independence.
"I think the main take is that gene therapy really is a curative option for these patients who do not have an allogeneic stem cell donor," Kulozik told CGTL in a video interview. "My personal feeling is, if in the long run gene therapy...shows itself to be safe, it might even be superior to allogeneic bone marrow transplantation or peripheral blood cell transplantation because we do not run into the problems of [graft versus host disease]."
The FDA is set to review additional data on beti-cel, as well as bluebird bio’s cerebral adrenoleukodystrophy (CALD) candidate elivaldogene autotemcel (eli-cel), in a pair of meetings on June 9 and 10, after the agency moved in January to extend the PDUFA goal date to August 19.
“Gene therapies are complex, potentially transformative treatment options for those living with severe genetic diseases, and we all share a responsibility to be diligent for patients as we progress this novel field,” Andrew Obenshain, chief executive office of bluebird bio, said in a statement at the time.3 “We look forward to continuing to work with the FDA on its ongoing reviews of beti-cel and eli-cel, and to bringing these therapies to patients with β-thalassemia and CALD in the US later this year.”
References:
Kunz J, Roth E, Greil J, et al. The first real-world experience with betibeglogene autotemcel (beti-cel) gene therapy treatment for transfusion-dependent β-thalassemia (TDT). Poster presented at: Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR; April 23-26, 2022; Salt Lake City, UT. Accessed April 21, 2022. https://tandem.confex.com/tandem/2022/meetingapp.cgi/Paper/19562
Locatelli F, Thompson AA, Kwiatkowski JL, et al. Betibeglogeneautotemcelgenetherapy for non–β0/β0 genotype β-thalassemia. N Eng J Med. 2022; 386:415-427. doi: 10.1056/NEJMoa2113206
bluebird provides update on FDA review timelines for betibeglogeneautotemcel (beti-cel) for beta-thalassemia and elivaldogeneautotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD). News release. bluebird bio. January 18, 2022. https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-provides-update-fda-review-timelines-betibeglogene
Considerations for Administering Hemoglobinopathy Gene Therapies From a Pharmacist’s Perspective
April 20th 2024Alexis Kuhn, PharmD, BCOP, a pediatric oncology pharmacist at Mayo Clinic, discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.
