Ultragenyx disclosed a 10% workforce reduction—about 130 roles—while refocusing resources after two failed late‑stage trials in osteogenesis imperfecta and continuing FDA scrutiny of its gene therapy program. The company resubmitted its BLA for UX111 (Sanfilippo A) after earlier manufacturing deficiencies; the FDA has now asked for additional supportive documentation tied to those manufacturing questions. CEO Emil Kakkis framed the restructuring as an effort to align spending with highest‑value programs and to preserve a path to profitability by 2027. The moves underscore the financial and operational strain that late‑stage trial failures and regulatory manufacturing hurdles can impose on specialty gene‑therapy companies.
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