FDA Grants Orphan Drug Designation to Novel CAR-T Cell Therapy to Treat Waldenstrom Macroglobulinemia, a Rare Type of Cancer

Treatment with the novel CAR-T cell therapy was associated with favorable efficacy and safety results in a group of patients with follicular lymphoma, including a few with Waldenstrom macroglobulinemia, according to findings from a study presented at a recent medical conference.

The Food and Drug Administration (FDA) recently granted the novel CD20-targeted autologous CAR-T cell therapy, MB-106, an orphan drug designation for its use to potentially treat patients with Waldenstrom macroglobulinemia, an extremely rare type of blood cancer.

Almost four decades ago, the United States government passed the Orphan Drug Act in an effort to incentivize more pharmaceutical companies to develop drugs to treat patients with rare diseases. If a drug is granted an orphan drug designation, the pharmaceutical company developing the drug is eligible for certain incentives including tax credits for qualified clinical trials.

The CAR-T cell therapy, MB-106, is currently being investigated in a group of patients with various types of high-risk B-cell non-Hodgkin lymphoma. Previous findings from a study presented at a recent medical conference showed that treatment with the novel CAR-T cell therapy was associated with favorable efficacy and safety in patients with follicular lymphoma.

In this group of 18 patients, treatment with MB-106 induced an overall response rate (percentage of patients whose disease partially or completely responds to treatment) of 94%. What was noteworthy was that 14 patients achieved a complete response (meaning there were no traces of the disease). Of those 14 patients, two had Waldenstrom macroglobulinemia and both experienced a complete response.

There is currently no FDA-approved CAR-T cell therapy indicated for the treatment of Waldenstrom macroglobulinemia, according to the agent’s manufacturer Mustang Bio.

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