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News|Podcasts|November 10, 2026

The BioPharm Brief: FDA Designations for a Bispecific CAR-T, a BLA for Friedreich’s Ataxia, and Phase 2b MASH Data for Efinopegdutide

FDA grants RMAT and Fast Track designations to AbelZeta’s CD20/CD19 bispecific CAR-T prizlon-cel in relapsed or refractory large B-cell lymphoma, Larimar Therapeutics completes a BLA seeking accelerated approval of nomlabofusp for Friedreich’s ataxia, and Merck’s GLP-1/glucagon receptor agonist efinopegdutide meets its primary endpoint in a phase 2b MASH trial.

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. Today: two FDA designations for a dual-targeting CAR-T, a protein replacement therapy heads toward review, and a dual agonist posts phase 2b liver data.

First, FDA has granted RMAT and Fast Track designations to AbelZeta’s prizlon-cel, a CAR-T therapy that targets both CD20 and CD19, for relapsed or refractory large B-cell lymphoma. Approved CAR-Ts in this disease target CD19 alone. Hitting two antigens is meant to reduce relapses that occur when tumors lose CD19. In a 48-patient study in China, 85% of patients had a complete response. AbelZeta’s US phase 2 trial will enroll patients whose disease progressed after an approved CAR-T.

Next, Larimar Therapeutics has completed its rolling BLA for nomlabofusp, a recombinant fusion protein that delivers frataxin to mitochondria in people with Friedreich’s ataxia. Larimar is seeking accelerated approval based on increases in frataxin levels in skin, which would be a new surrogate endpoint. The company expects FDA to accept the filing in early 2027 and is targeting a potential approval by mid-2027. The confirmatory phase 3 FORWARD-FA trial is enrolling.

And finally, Merck’s efinopegdutide met its primary endpoint in a phase 2b trial in MASH with moderate to advanced fibrosis. The once-weekly drug activates both GLP-1 and glucagon receptors. At 52 weeks, MASH resolved without worsening fibrosis in 56% to 63% of patients across three doses, versus 13% with placebo. Nausea and vomiting were common, especially at the highest dose. Merck has not said which dose it will take into phase 3.

That’s today’s BioPharm Brief. For analysis and expert insights, please visit BioPharmInternational.com.

Key takeaways

  • Dual-antigen CAR-T: Prizlon-cel’s designations support US development in patients who relapse after CD19 CAR-T, though it hasn’t been shown that dual targeting beats single targeting.
  • Surrogate endpoints: Larimar’s filing tests whether FDA will accept skin frataxin as a basis for accelerated approval in a rare neurodegenerative disease.
  • Dose selection: Efinopegdutide’s strong resolution rates come with tolerability tradeoffs that will shape Merck’s phase 3 plans.

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