Register Now: From Clinical to Commercial: Accelerating ADC Late-Stage Development with Technology Innovation and Integrated Platform
Logo Image
News|Podcasts|August 20, 2026 (Updated: August 20, 2026)

The BioPharm Brief: Rare Disease, New Options

Listen
0:00 / 0:00

This edition of The BioPharm Brief covers FDA approvals for Pasatru in fibrodysplasia ossificans progressiva and Genglycos in glycogen storage disease type Ia, along with a new bispecific CAR-T entering clinical development for large B-cell lymphoma.

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry.

The FDA has approved Regeneron’s Pasatru, or garetosmab-grts, for adults with fibrodysplasia ossificans progressiva, or FOP, an ultra-rare genetic disorder in which bone forms abnormally in muscles and other connective tissues. Pasatru is the first and only FDA-approved treatment shown in a placebo-controlled trial to reduce new heterotopic ossification lesions and clinician-assessed flare-ups in adults with FOP. The approval was supported by the Phase 3 OPTIMA trial, where Pasatru demonstrated a 90% or greater reduction in new heterotopic ossification lesions at 56 weeks. The treatment targets Activin A, a signaling protein involved in the abnormal bone formation associated with FOP.

Another rare disease has a new treatment option with the FDA’s accelerated approval of Ultragenyx’s Genglycos, or pariglasgene brecaparvovec-opnr, for patients eight years and older with glycogen storage disease type Ia. Genglycos is a one-time AAV8 gene therapy designed to address the underlying genetic cause of the disease and reduce patients’ daily dependence on cornstarch as an adjunct to nutritional management. The approval is based on reduction in daily cornstarch intake, and continued approval is contingent on verification of clinical benefit in confirmatory studies. Genglycos is the first FDA-approved gene therapy for GSDIa and also represents Ultragenyx’s first gene therapy approval.

And in oncology, AbelZeta Pharma has regained global rights to prizloncabtagene autoleucel, or Prizlon-cel, from Janssen and received FDA clearance for its IND application in large B-cell lymphoma. The therapy is an anti-CD20/CD19 bispecific CAR-T designed for patients with large B-cell lymphoma, including patients whose disease has relapsed after prior CAR-T treatment. AbelZeta is now working with the FDA to finalize clinical development protocols, with plans to evaluate Prizlon-cel in third-line or later patients previously treated with CAR-T as well as in second-line patients who have not received CAR-T therapy.

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

Key takeaways:

  • Pasatru becomes a new treatment option for adults with the ultra-rare bone-forming disorder FOP.
  • Genglycos is the first FDA-approved gene therapy for glycogen storage disease type Ia.
  • AbelZeta is moving a bispecific CAR-T into US clinical development for large B-cell lymphoma.

Related to this article

View more
The BioPharm Brief: An AI-Designed Multibody ADC Enters the Clinic, Blackstone Backs Pfizer’s Monthly GLP-1, and enGene Readies a Bladder Cancer BLA
The BioPharm Brief: An AI-Designed Multibody ADC Enters the Clinic, Blackstone Backs Pfizer’s Monthly GLP-1, and enGene Readies a Bladder Cancer BLA
FDA clears the IND for Biolojic Design’s BD200, an AI-designed Trop2/Nectin-4 multibody ADC; Blackstone Life Sciences agrees to provide Pfizer up to $1.5 billion to develop its monthly GLP-1 receptor agonist berobenatide; and enGene Therapeutics plans a year-end BLA for detalimogene voraplasmid in BCG-unresponsive non-muscle invasive bladder cancer.
Read more
The BioPharm Brief: FDA Designations for a Bispecific CAR-T, a BLA for Friedreich’s Ataxia, and Phase 2b MASH Data for Efinopegdutide
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.
Read more
The BioPharm Brief: argenx Bets on Peptides, a Two-Bispecific Regimen in Prostate Cancer, and a New Take on T-Cell Engagers
The BioPharm Brief: argenx Bets on Peptides, a Two-Bispecific Regimen in Prostate Cancer, and a New Take on T-Cell Engagers
argenx agrees to acquire macrocyclic peptide developer Unnatural Products for up to about $1.15 billion, Johnson & Johnson’s pasritamig and JNJ-9401 bispecific regimen achieves a 73% PSA response in phase 1 in metastatic castration-resistant prostate cancer, and Janux Therapeutics’ TRBV6-targeted T-cell engager JANX011 shows deep B-cell depletion in healthy volunteers.
Read more
The BioPharm Brief: A Bispecific Approval in Head and Neck Cancer, TCR Bispecifics Head to the Clinic, and a New Antibody Target in Essential Thrombocythemia
The BioPharm Brief: A Bispecific Approval in Head and Neck Cancer, TCR Bispecifics Head to the Clinic, and a New Antibody Target in Essential Thrombocythemia
FDA grants accelerated approval to Johnson & Johnson’s subcutaneous EGFR/MET bispecific Rybrevant Faspro for previously treated head and neck squamous cell carcinoma, T-Therapeutics raises a $133 million Series B to take TCR-CD3 bispecifics into clinical trials, and Alethio Therapeutics raises a $90 million Series A to advance its first-in-class G6B antibody for essential thrombocythemia.
Read more
The BioPharm Brief: Ivonescimab Priority Review in China, Stargardt Gene Therapy Moves Toward Phase 3, and Longer Dosing Intervals for an IBD Bispecific
The BioPharm Brief: Ivonescimab Priority Review in China, Stargardt Gene Therapy Moves Toward Phase 3, and Longer Dosing Intervals for an IBD Bispecific
China’s NMPA grants priority review to Akeso’s PD-1/VEGF bispecific ivonescimab with chemotherapy for first-line biliary tract cancer, VeonGen Therapeutics aligns with FDA on a phase 3 design for its dual-AAV Stargardt gene therapy VG801, and Caldera Therapeutics’ IL-23p19×TL1A bispecific CLD-423 shows a half-life of more than 40 days in a phase 1a trial.
Read more
The BioPharm Brief: A $7 Billion Oncology Option Deal, a TL1A Pivot, and Oral Adalimumab Heads to the Clinic
The BioPharm Brief: A $7 Billion Oncology Option Deal, a TL1A Pivot, and Oral Adalimumab Heads to the Clinic
Boehringer Ingelheim licenses six preclinical ADC and T-cell engager programs from Sino Biopharmaceutical in a deal worth up to $7 billion, Spyre Therapeutics drops anti-TL1A antibody SPY072 in rheumatic diseases after phase 2 misses and turns to hidradenitis suppurativa, and Biora Therapeutics gets ethics approval for a first-in-human trial of its BioJet oral adalimumab capsule.
Read more