The U.S. Food and Drug Administration (FDA) granted orphan drug status to LMP400 (indotecan) for use in patients with malignant glioma, a cancer of the brain that begins in glial cells (cells that surround and support nerve cells).

The Food and Drug Administration (FDA) approved belumosudil on July 16, 2021, for people 12 years and older with chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy. Chronic GVHD is a complex condition that can be life threatening and occurs when donated stem cells attack healthy tissues in a patient’s body. Steven Z. Pavletic, M.D., M.S., Senior Clinician in the Immune Deficiency Cellular Therapy Program, guided CCR’s involvement in the pivotal consortium study that led to FDA approval of belumosudil. Pavletic was part of the trial clinical leadership at CCR, one of the 28 centers that enrolled study patients. The study found belumosudil to be safe and well-tolerated, and it may have the potential to improve overall patient well-being.

On August 13, 2021, the Food and Drug Administration approved belzutifan, a new drug for adult patients with von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC), central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors, not requiring immediate surgery.

Ramaprasad Srinivasan, M.D., Ph.D., Investigator in the Urologic Oncology Branch (UOB), designed the ongoing study and played a key leadership role as the principal investigator on the cooperative research and development agreement under which NCI served as a site in the study. Belzutifan is now the first and only approved systemic therapy for certain patients with VHL-associated RCC.

VHL disease is a rare, inherited disorder that causes tumors and cysts to grow in certain parts of the body. Patients with this disease have an increased risk of certain types of cancer, especially kidney cancer and pancreatic cancer. The VHL gene was originally identified by Marston Linehan, M.D., and colleagues in the UOB in the 1990s, and the group continues to define the methods for clinical management of VHL disease.

The Food and Drug Administration (FDA) has approved selumetinib (Koselugo) to treat children with neurofibromatosis type 1 (NF1) and tumors called plexiform neurofibromas. The approval was based on results from a clinical trial, designed and led by Brigitte Widemann, M.D., Chief of the Pediatric Oncology Branch (POB), and Andrea M. Gross, M.D., Assistant Research Physician in POB, that showed the drug shrank neurofibromas in 70 percent of patients. For many children in the trial, treatment with selumetinib also had clinical benefit, improving their pain, function and quality of life.

On May 14, 2020, the Food and Drug Administration expanded the indication of pomalidomide (POMALYST, Celgene Corporation) to include treating adult patients with AIDS-related Kaposi sarcoma after failure of highly active antiretroviral therapy and Kaposi sarcoma in adult patients who are HIV-negative. This oral therapy is the first new treatment option available for those with Kaposi sarcoma in more than 20 years.

In May 2019, the FDA granted breakthrough therapy designation to a tumor-infiltrating lymphocyte (TIL) therapy technology known as LN-145 to treat advanced cervical cancer based on data presented by CCR collaborator Iovance Biotherapeutics. 

The U.S. Food and Drug Administration granted orphan drug status in December to zotiraciclib for use in patients with glioma, a cancer of the brain that begins in glial cells (cells that surround and support nerve cells). This designation is based on results from an ongoing NCI-sponsored phase 1 trial led by the CCR Neuro-Oncology Branch at the NIH Clinical Center.

The U.S. Food and Drug Administration granted orphan drug status in December to zotiraciclib for use in patients with glioma, a cancer of the brain that begins in glial cells (cells that surround and support nerve cells). This designation is based on results from an ongoing NCI-sponsored phase 1 trial led by the CCR Neuro-Oncology Branch at the NIH Clinical Center.

The Food and Drug Administration has granted orphan drug designation to PRGN-2012 immunotherapy for use in patients with recurrent respiratory papillomatosis.

A cell-based immunotherapy that is currently being evaluated at the NIH Clinical Center for the treatment of B-cell acute lymphoblastic leukemia (ALL) has been designated by the U.S. Food and Drug Administration as a drug for a rare pediatric disease. Designation could encourage development of this novel therapy for children with relapsed or refractory ALL.

The U.S. Food and Drug Administration granted orphan drug status in February to selumetinib for use in patients with the genetic disorder neurofibromatosis type 1 (NF1), who often develop tumors of the peripheral nervous system. Receiving orphan drug designation is a helpful step for selumetinib.

The Food and Drug Administration approved idecabtagene vicleucel (Abecma) for people with multiple myeloma that has not responded to or has returned after at least four different prior cancer treatments. The approval is based, in part, on earlier clinical work led by James N. Kochenderfer, M.D., Senior Investigator in the Surgery Branch.

Autumn Schierling is one of 50 children who have been participating since 2015 in a clinical trial of neurofibromatosis type 1 (NF1) at the NIH Clinical Center. In March 2020, the results of the trial were published and in April the FDA approved selumetinib as the first treatment for NF1. Brigitte Widemann, M.D., Chief of the Pediatric Oncology Branch who started conducting clinical trials for NF1 in 2001 was quoted as saying, “The medication’s approval is ‘huge progress’ and we will work to make more progress.”

In August 2019, the U.S. Food and Drug Administration granted breakthrough therapy designation to an experimental immunotherapy being developed in the Center for Cancer Research (CCR) for the treatment of B-cell acute lymphoblastic leukemia (ALL), a type of blood cancer. The designation will advance CCR’s development and testing of an immunotherapy for children and young adults whose B-cell ALL is resistant to CD19-targeted immunotherapies.  

The U.S. Food and Drug Administration last week granted breakthrough therapy designation for the MEK 1/2 inhibitor selumetinib. The designation is for the treatment of pediatric patients aged three years and older with neurofibromatosis type 1 (NF1) symptomatic and/or progressive, inoperable plexiform neurofibromas (PN), a rare, incurable genetic condition.