The FDA granted orphan drug designation to a CAR T-cell remedy for recurrent glioblastoma and high-grade astrocytomas.
The U.S. Meals and Drug Administration (FDA) has granted orphan drug designation to Mustang for MB-101 (IL13Ra2-targeted CAR T-cells) for the remedy of recurrent diffuse and astrocytomas (anaplastic astrocytoma) and glioblastoma (GBM), in accordance with a information launch from Mustang Bio.
The FDA grants orphan drug designation to remedies developed for uncommon ailments — these affecting fewer than 200,000 individuals within the U.S.
As beforehand reported, preclinical knowledge introduced on the American Affiliation for Most cancers Analysis Annual Assembly in 2022 supported the potential of mixing MB-108 with MB-101 to reinforce outcomes for sufferers with recurrent glioblastoma. MB-108, an oncolytic virus, is designed to switch the tumor microenvironment (TME) by changing chilly tumors — those who evade immune detection — into sizzling tumors that entice immune exercise. This course of might assist improve the effectiveness of MB-101.
Moreover, separate early-phase knowledge from Metropolis of Hope and the College of Alabama at Birmingham have proven that MB-101 and MB-108 had been each properly tolerated in sufferers with recurrent glioblastoma. In a 2024 Nature Medication paper from Metropolis of Hope, two sufferers with excessive ranges of intratumoral CD3+ T cells — labeled as having sizzling tumors — skilled full responses after receiving MB-101 alone, lasting 7.5 months and greater than 66 months, respectively. These two sufferers had been amongst simply three within the research with the best pre-treatment immune exercise, as per the discharge.
Each section 1 trials — MB-101 at Metropolis of Hope and MB-108 on the College of Alabama at Birmingham — stay open and proceed to enroll sufferers.
In line with the Nationwide Most cancers Institute, chilly tumors describe a tumor that doesn’t set off a robust immune response. These tumors are sometimes surrounded by cells that block immune exercise, stopping T-cells from attacking the tumor. Chilly tumors normally resist immunotherapy.
Moreover, sizzling tumors describe a tumor that triggers a robust immune response. These tumors usually show floor markers that assist T-cells acknowledge and assault them. Sizzling tumors are extra doubtless to reply to immunotherapy.
“We’re thrilled that MB-101 acquired orphan drug designation on time and with a designation that’s broader than the indication proposed,” Dr. Manuel Litchman, president and chief government officer of Mustang Bio, mentioned within the information launch. “The orphan drug designation for MB-101, coupled with the orphan drug designation granted beforehand for MB-108, is robust validation for our science, as we hope to advance MB-101, together with MB-108, as a possible remedy possibility for sufferers dwelling with malignant glioma, together with sufferers with recurrent GBM and high-grade astrocytomas.
Litchman continued, “This progress demonstrates our dedication to exploring new potentialities for enhancing outcomes in sufferers with challenging-to-treat cancers.”
The longer term improvement of the MB-109 program for recurrent glioblastoma and high-grade astrocytomas will rely upon securing extra funding or getting into a strategic partnership.
This designation is supposed to encourage analysis and improvement in areas the place there could also be restricted choices for sufferers. It comes with advantages like tax credit to assist offset medical trial prices and waivers for sure FDA charges. If the drug is ultimately authorized, it could additionally obtain seven years of market exclusivity for that uncommon situation, which helps shield entry to the remedy no matter patent standing.
Reference:
“Mustang Bio Granted Orphan Drug Designation by U.S. FDA for MB-101 (IL13Ra2-targeted CAR T-cells) to Deal with Astrocytomas and Glioblastoma,” Mustang Bio, Inc., GlobeNewswire, July 7, 2025.
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