Taiwanese clinical-stage drug development company AnnJi Pharmaceutical Co., Ltd. (TWSE: 7754) announced on Thursday that the US Food and Drug Administration has granted Fast Track Designation for AJ201, its first-in-class therapy for spinal and bulbar muscular atrophy (SBMA), also known as Kennedy's disease.

Combined with prior Orphan Drug Designations from both the FDA and the European Medicines Agency, the recognition underscores AJ201's potential to address significant unmet needs in SBMA.

SBMA is a rare, inherited neuromuscular disease affecting approximately one in 40,000 males worldwide. The condition results from mutations in the androgen receptor gene that lead to progressive muscle and neuron degeneration and currently lacks an FDA-approved therapy.

AJ201 is a novel compound designed to reduce mutant androgen receptor toxicity and improve motor function. It acts by promoting degradation of pathogenic proteins and enhancing cellular defence mechanisms, potentially slowing disease progression.

AnnJi plans to advance AJ201 into Phase 3 clinical development and work closely with regulators to bring forward the first potential treatment for SBMA in more than 20 years. The company aims to out-licence its drug candidates after achieving proof-of-concept in Phase II trials to accelerate global market access and drive sustainable growth.