FDA Approves New Treatment for Hemophelia A and B

sickle cell disease

The U.S. Food and Drug Administration (FDA) approved Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors (neutralizing antibodies).

New Gene Therapies Are Transformative for People with Sickle Cell Disease and Frequent Vaso-Occlusive Crises

sickle cell disease

Affecting more than 100,000 American children and adults, sickle cell disease is an inherited hemoglobinopathy that results when a single-nucleotide mutation in the ß-globin gene yields an abnormal “sickle” hemoglobin (HbS). Now, these patients may be eligible for a one-time gene therapy that offers the potential of a durable functional cure by eliminating severe VOCs and associated hospitalizations.

Xembify Receives Expanded Label for PI Patients

The U.S. Food and Drug Administration (FDA) has approved an expanded label for Grifols’ XEMBIFY (20% subcutaneous immune globulin [SCIG]) to include treatment-naïve patients with primary humoral immunodeficiencies (PI).

The Protective Value of RSV Vaccines in Older Adults: A Deeper Dive

Now that safe and effective RSV vaccines are finally available, more evidence of their direct health benefits in older Americans will be forthcoming. It is likely as well that FDA will eventually expand the marketing approvals of these vaccines to include adults under age 60 with chronic conditions demonstrated to importantly increase the risk of RSV-associated LRTD and hospitalization.

HIV: A Physician’s Perspective

Mark Bloch, MD, has been working in the field of HIV medicine since 1983, and he is now the director of clinical research at Holdsworth House in Sydney, Australia. Dr. Bloch is actively involved in research surrounding HIV and sexually transmitted diseases.