The BioPharm Brief: Rare Disease, New Options

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.