66th ASH Annual Meeting: ASH-FDA Joint Symposium II: Challenges and Innovations in Rare Diseases in Non-Malignant Hematology

Special-Interest Sessions

Treatment Considerations

Room 7 (San Diego Convention Center)

Chair:

Donna A. Whyte-Stewart, MD, MSc, Food and Drug Administration

Disclosures:

No relevant conflicts of interest to declare.

Introduction

Donna A. Whyte-Stewart, MD, MSc

DNH/OCHEN/CDER, Food and Drug Administration, Silver Spring, MD

Mavorixafor for Treatment of WHIM Syndrome

Alison R. Moliterno, MD

DNH/OCHEN/CDER, Food and Drug Administration, Silver Spring, MD

Mavorixafor, a Novel CXC4 Agonist for WHIM Syndrome: Challenges in Successful Drug Development in Rare Hematologic Disorders

Hanny Al-Samkari, MD

Division of Hematology, Massachusetts General Hospital, Cambridge, MA

Marstacimab-hncq for Treatment of Hemophilia A or Hemophilia B

Roma Rajput, MD

CDER/OCHEN/DNH, Food and Drug Administration, Silver Spring, MD

Fidanacogene Elaparvovec-dzkt for Treatment of Adults with Moderate to Severe Hemophilia B

Megha Kaushal, MD, MSc

Division of Clinical Evaluation Hematology, Office of Therapeutic Products (OTP), Center for Biologics Evaluation and Research (CBER), US Food and Drug Administration, Silver Spring, MD

Clinical Discussant for Hemophilia

Robert F Sidonio Jr., MD

Emory University School of Medicine, Atlanta, GA

Exagamglobene Autotemcel (exa-cel) for Treatment of Transfusion-Dependent Beta-Thalassemia

Megha Kaushal, MD, MSc

Division of Clinical Evaluation Hematology, Office of Therapeutic Products (OTP), Center for Biologics Evaluation and Research (CBER), US Food and Drug Administration, Silver Spring, MD

Clinical Discussant for Thalassemia

Janet L. Kwiatkowski, MD

Children's Hospital of Philadelphia, Philadelphia, PA

Panel Discussion

Donna A. Whyte-Stewart, MD, MSc

DNH/OCHEN/CDER, Food and Drug Administration, Silver Spring, MD

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