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business

FDA agrees accelerated approval pathway for GBT’s voxelotor

  GBT plans to submit a new drug application (NDA) under this accelerated approval pathway following a meeting in early 2019. Under the terms of a subpart H pathway, voxelotor will be subject to post-marketing studies to confirm its clinically meaningful benefit to SCD patients. GBT president and CEO Ted W Love said: “Gaining US regulatory alignment for voxelotor under the accelerated approval pathway is a significant achievement for SCD patients and their families.” “We commend the FDA, and the broader SCD community, for its continuous commitment toward understanding the critical need for new clinical endpoints that support the development of much-needed new therapies to treat this devastating disease.” Voxelotor is an oral, once-daily therapy. Professor Kim Smith-Whitley, clinical director and head of paediatrics at the Perelman School of Medicine’s Division of Haemtology, said: “A once-daily, oral therapy that has the potential to safely improve the anemia of SCD and thereby preserve brain function would be a major breakthrough.
Dec 5, 2018
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clinical trials and research

Early clinical trial data show gene therapy reversing sickle cell anemia

  After over a decade of preclinical research and development, a new gene therapy treatment for sickle cell anemia (SCA) is reversing disease symptoms in two adults and showing early potential for transportability to resource-challenged parts of the world where SCA is most common. Preliminary data from a pilot Phase 1-2 clinical trial testing the gene-addition therapy were presented on Dec. 3 at the American Society of Hematology's (ASH) annual meeting in San Diego by principal investigator Punam Malik, MD. She is a physician-scientist at the Cincinnati Children's Cancer and Blood Diseases Institute and director of its Comprehensive Sickle Cell Center. Malik called the data promising. One year after treatment of our first patient, and six months after treatment of our second patient, both have seen a remarkable improvement in the quality of life due to remarkable reduction in disease symptoms.  
Dec 4, 2018