The U.S. Food and Drug Administration (FDA) has granted orphan drug designation and pediatric exclusivity to CRESEMBA (isavuconazonium sulfate) for treatment of invasive aspergillosis (IA) and invasive mucormycosis (IM) in pediatric patients.

 

PRIME Designation for GTX-102 for the Treatment of Angelman Syndrome

 

Aspergillosis is an infection, growth, or allergic response caused by the Aspergillus fungus. Invasive aspergillosis is one of several different kinds of Aspergillosis. IA usually affects people with weakened immune systems due to:

  • Cancer
  • AIDS
  • Leukemia
  • Organ transplantation
  • Chemotherapy
  • Or other conditions that reduce the number of normal white blood cells

In this condition, the fungus invades and damages tissues in the body. IA most commonly affects the lungs, kidneys, and brain, but can also spread and cause infection in many other organs.

Mucormycosis is a term for a group of infections caused by related molds from the order Mucorales. These infections are usually acquired when spores from the molds are breathed in or enter the body through a cut in the skin. Mucormycosis is an aggressive, life-threatening infection that occurs in immune-compromised people, including people with uncontrolled diabetes mellitus, neutropenia, or whose immune system is being suppressed by medications as part of their treatment for blood cancer, hematopoietic stem cell transplantation, or solid-organ transplant. Treatment usually consists of antifungal medications and surgery.

Isavuconazonium Sulfate

Isavuconazonium sulfate is the first and only azole antifungal therapy to obtain FDA approval for the treatment of IA and IM in pediatric patients as young as one. This orphan drug designation provides seven years of exclusive marketing rights for CRESEMBA in those indications beginning from the date of approval of the supplemental New Drug Application on December 8, 2023.

Pediatric exclusivity extends the period of orphan drug designation by another six months. The FDA granted orphan drug exclusivity for CRESEMBA in 2015 for the treatment of IA and IM in adults and has now extended this exclusivity to pediatric patients.

It is important to note that CRESEMBA has certain contraindications and may cause certain adverse reactions. It is contraindicated in persons with known hypersensitivity to isavuconazole and in patients with familial short QT syndrome. Adverse reactions reported include nausea, vomiting, diarrhea, headache, elevated liver chemistry tests, and hypokalemia.

For more information on rare diseases, visit https://checkrare.com/