Rare Disease Treatment

Stockholm – September 24, 2026 -- Sobi (STO: SOBI) will present new clinical data on its rare-disease drug Gamifant (emapalumab) at the 2026 Annual Meeting of the Histiocyte Society (HS2026), running September 26-29 in Rotterdam, the Netherlands, with a pooled survival analysis nominated for the meeting's top poster prize.

Pooled transplant-survival analysis earns Robert J. Arceci Prize nomination

The accepted abstract set includes a new pooled analysis of pre- and post-transplant survival outcomes in primary haemophagocytic lymphohistiocytosis (HLH), drawing on data across three prospective trials. Sobi said the analysis was selected as one of the ten highest-scoring poster abstracts at HS2026 and has been nominated for the Robert J. Arceci Prize for Best Poster.

Suzhou – – September 16, 2026 -- Accro Bioscience said AC-101 tablets have been included in the National Medical Products Administration's Center for Drug Evaluation (CDE) "Caring Plan-Extension" pilot program, targeting Blau syndrome as the proposed indication, with the public notice period closing September 11, 2026.

NMPA pilot program aims to accelerate rare disease drug development in China

The Caring Plan-Extension pilot, run by China's CDE, is designed to encourage innovative drug development for rare diseases. Accro Bioscience confirmed the inclusion took effect September 14, 2026, China Standard Time (UTC+8).

FDA granted AC-101 Rare Pediatric Disease designation three months earlier

The U.S. Food and Drug Administration awarded AC-101 Rare Pediatric Disease (RPD) designation for Blau syndrome in June 2026, ahead of the Chinese regulatory step.