C-CAR039 Earns RMAT, Fast Track Designations for DLBCL

According to the Cancer Network, a CAR T-cell therapy called C-CAR039 recently earned both Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the U.S. Food and Drug Administration. In particular, these designations centered around the treatment of those with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL). Additionally, both designations come with benefits for the drug developers.

C-CAR039

To begin, let’s take a look at what C-CAR039 is. This therapy is a Chimeric Antigen Receptor (CAR) T-cell therapy which targets CD19 and CD20. Developed by Cellular Biomedicine Group Inc., C-CAR039 is given via intravenous infusion. Beyond DLBCL, C-CAR039 also received Orphan Drug designation for the treatment of follicular lymphoma.

In this particular case, the RMAT and Fast Track designations stem from research data. In one study, patients with DLBCL who received C-CAR039 had an overall response rate of 92.6% and a complete response rate of 85.2%. Over 74% of patients were in remission during the follow-up period. The treatment was found to be relatively safe and well-tolerated. Some adverse reactions did occur, including relatively mild cytokine release syndrome or immune effector cell-associated neurotoxicity syndrome.

RMAT designation is granted to regenerative medicine therapies, such as cell therapies, human cell or tissue products, or therapeutic tissue engineering products. The designation is granted to products intending to treat, prevent, cure, reverse, or change serious or life-threatening conditions, which is shown through preclinical studies. Once a product receives RMAT designation, the drug developer also earns increased early FDA interactions and other benefits of the Fast Track and Breakthrough Therapy designations.

Fast Track designation is designed to facilitate the development and review of drugs and biologics intended to treat serious conditions and fulfill unmet needs. Benefits of this designation include Priority Review and Accelerated Approval eligibility; more frequent FDA meetings and written communication; and Rolling Review.

Learn more about RMAT and Fast Track designations.

About Diffuse Large B-Cell Lymphoma (DLBCL)

Diffuse large B-cell lymphoma (DLBCL) exists under the umbrella of non-Hodgkin’s lymphoma (NHL). In fact, DLBCL is the most common form of NHL. This aggressive cancer affects B-lymphocytes, a type of white blood cell. Although DLBCL is aggressive, its prognosis – following treatment – is usually promising.

Overall, doctors do not know what causes DLBCL. However, risk factors include older age, being male, being immunocompromised, or having a family history of DLBCL. Symptoms and characteristics include:

  • Drenching night sweats
  • Fever
  • Unintended weight loss
  • Painless but swollen lymph nodes
  • Chest or abdominal pain or pressure
  • Itchiness
  • Shortness of breath and/or difficulty breathing
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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