FDA Accepts NDAs for Beren's Adrabetadex and Dr. Reddy's Abatacept Biosimilar

The U.S. Food and Drug Administration has accepted for review Beren Therapeutics' New Drug Application for adrabetadex, an investigational cyclodextrin therapy designed to increase intracellular cholesterol trafficking in Niemann-Pick disease type C (NPC). The FDA assigned adrabetadex a Prescription Drug User Fee Act (PDUFA) target action date of August 17, 2026 and granted priority review.

If approved, adrabetadex would represent a first-in-class, disease-modifying approach to treat infantile-onset NPC, a rare and rapidly fatal pediatric neurodegenerative disorder. The therapy would be the only treatment to directly target the underlying pathophysiology of Niemann-Pick disease type C.

The NDA for adrabetadex is supported by data demonstrating a clinically meaningful survival benefit in infantile-onset patients. This evidence is from an externally controlled analysis intended to serve as a single adequate and well-controlled study. The comprehensive submission also includes data showing slowed disease progression, confirmatory biomarker and nonclinical findings and patient experience narratives.

Key findings were recently presented at WORLDSymposium 2026, the largest annual scientific meeting in the field of lysosomal storage disease, including survival data in infantile-onset NPC: a 71% reduction in the risk of mortality in adrabetadex-treated patients compared with matched external controls (HR 0.289; 95% CI, 0.141–0.593; P < 0.0001).

In 2025 the FDA granted adrabetadex Breakthrough Therapy Designation, a status that accelerates the development of drugs for serious or life-threatening conditions when early evidence suggests a substantial improvement over existing therapy.

Niemann-Pick disease type C is a rare, autosomal-recessive, severe, neurodegenerative disorder caused by pathologic variants in the NPC1 (~95% of cases) or NPC2 genes, leading to impaired cholesterol trafficking resulting in progressive neurological decline and premature death. Infantile-onset NPC refers to NPC in infants and children who first experience neurological symptoms between 0 and 6 years of age. Earlier neurological onset is associated with more rapid progression and poorer prognosis, with mean survival of ~5.6 years for early infantile-onset (age of neurological onset <2 years) and ~13.4 years for late-infantile onset (2 to <6 years).

Adrabetadex is a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers under investigation as a treatment for NPC. Data from clinical trials and expanded access programs suggest that adrabetadex is generally well tolerated. The main adverse events associated with adrabetadex include hearing impairment that can be managed with hearing aids when necessary, and post-dose fatigue and/or ataxia.

Separately, the U.S. Food and Drug Administration has accepted for review Dr. Reddy's Laboratories' Biologics License Application for DRL_AB, a proposed biosimilar to Orencia (abatacept) for intravenous infusion. The company submitted the 351(k) BLA in December 2025. Dr. Reddy's stated it is the first company to submit a BLA for an abatacept biosimilar.

DRL_AB is intended for treatment of adults with moderately-to-severely active rheumatoid arthritis, adults with active psoriatic arthritis, and individuals aged six years and above with moderately-to-severely active polyarticular juvenile idiopathic arthritis.

The BLA submission includes analytical, pharmacokinetic and clinical studies. Dr. Reddy's completed a Phase 1 study that achieved pharmacokinetic similarity with comparable safety and immunogenicity profiles to Orencia. A pivotal Phase 3 study comparing the efficacy and safety of DRL_AB with Orencia is ongoing.

Abatacept is a selective co-stimulation modulator that inhibits T-cell activation by binding to CD80 and CD86, thereby blocking interaction with CD28. The reference product Orencia is owned by Bristol-Myers Squibb Company.