Mustang Bio Inc (NASDAQ: MBIO) has received an Orphan Drug Designation from the US FDA for its CD20-targeted, autologous CAR T cell therapy, MB-106, for Waldenstrom macroglobulinemia (WM) treatment. WM is a rare type of B-cell-Hodgkin lymphoma.
Mustang is developing MB-106 in partnership with Fred Hutchinson Cancer Center
MB-106 is under development in a partnership between the company and the Fred Hutchinson Cancer Center in treating patients with chronic lymphocytic leukemia (CLL) and B-NHLs that have relapsed or are resistant to treatment.
Medicines and biologic drugs that are designed for the effective and safe treatment, diagnosis, or treatment of rare diseases or conditions that impact fewer than 200,000 persons in the US are given orphan drug designation by the FDA. When medicine is designated as an orphan, it receives benefits like tax credits towards the expense of clinical studies when it is approved and exemptions from prescription drug user fees. In addition, when the FDA grants a product Orphan Drug Status, it is eligible for seven years of commercial exclusivity for the condition for which it has been designated as an Orphan Drug. This market exclusivity is separate from intellectual property rights.
CEO Manuel Litchman said, “We are very pleased to receive Orphan Drug Designation from the FDA, as it is an important regulatory milestone for Mustang’s MB-106 program for the treatment of Waldenstrom macroglobulinemia, a rare B-NHL with a significant unmet medical need. We look forward to dosing the first patient in our multicenter Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106 for relapsed or refractory B-NHL and CLL under Mustang’s IND shortly.”
WM patient to be added to indolent lymphoma Phase 1 study
Patients with Waldenstrom syndrome will be added to the indolent lymphoma group of the Phase 1 part of this study concurrently with the addition of participants to the aggressive lymphoma and CLL cohorts.
All five dose intervals of the MB-106 findings, which were presented at the beginning of the month at the European Hematology Association 2022 Hybrid Congress, continue to show significant efficacy and an extremely favorable safety profile.