Novartis has acknowledged seven previously unknown cases of tumours in children who received its gene therapy for spinal muscular atrophy, according to STAT. The therapy, Zolgensma, is a high-profile treatment in the rare disease sector.
This disclosure is significant for pharma and biotech leaders assessing the safety profile of gene therapies. Safety incidents can affect regulatory scrutiny, patient trust and market adoption. Companies may need to review their own risk management and communication strategies as regulators assess the situation.
