Busy FDA gives rare disease sector complementary pathways, unanswered questions – 2026 snapshot on June 08, 2026 Accelerated Approval Disease Evidence Principles Fast Track Infrequent mechanism pathway Orphan Drug Designation Pediatric Disorder Rare Diseases RDEP Review Voucher United States Food and Drug Administration +
Servier Supercharges Neuro Pipeline with $450M Deal for Fragile X Syndrome Candidate on September 08, 2025 Acquisition Bioscience deal Fragile X Syndrome Infrequent Kaerus KER-0193 Orphan Drug Designation pediatric drug designation Phase 2 trial Servier +
FDA Grants Full Approval to Precigen's Papzimeos for Rare Disease RRP on August 18, 2025 Approved Episodic gene therapy Immunotherapy Infrequent Orphan Drug Designation Papzimeos Precigen Recurrent respiratory papillomatosis RRP United States Food and Drug Administration +