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The US Food and Drug Administration (FDA) has released a final guidance document with immediate effect advising sponsors on chemistry, manufacturing, and controls (CMC) flexibilities for cell and gene therapy (CGT) products developed for biologics license applications (BLAs).

The US pharmaceutical industry expressed support for the US Food and Drug Administration’s (FDA) proposed framework for increasing access to treatments for ultra-rare diseases yet raised questions concerning various aspects of the program.

The US Food and Drug Administration (FDA) has released draft guidance on how sponsors can utilize next-generation sequencing (NGS) methods in nonclinical studies to assess the safety risks associated with gene therapy products. This guidance aims to support the submission of investigational new drug applications (INDs) and biologics license applications (BLAs).

US Food and Drug Administration (FDA) officials credited recent advancements in regulatory science and flexibilities with enabling growth in the cell and gene therapy sector during a meeting on Thursday; however, a cell therapy expert said there is still reluctance from FDA and industry to enroll pediatric patients in clinical trials for such treatments.

During a meeting to commemorate Rare Disease Day on Monday, officials from the US Food and Drug Administration (FDA) highlighted the importance of collaboration among stakeholders, while FDA Commissioner Marty Makary touted recent FDA initiatives to speed the development of new drugs for rare diseases.

The European Medicines Agency (EMA) has announced that its pilot program for the stepwise pediatric investigation plan (sPIP) has successfully supported timely pediatric development while maintaining scientific rigor. This program will be incorporated into the existing pediatric regulatory landscape.

The Medicines and Medical Devices Safety Authority (Medsafe) is holding a consultation on plans to extend the abbreviated procedure for new medicine applications and certain change notifications.

The European Medicines Agency (EMA) has published a revised guideline on the development of drugs aimed at treating peripheral arterial occlusive disease (PAOD) in the lower extremities. It will take effect on 30 June 2026.

The US Food and Drug Administration's (FDA) Center for Drug Evaluation and Research (CDER) approved 46 novel drugs in 2025, representing a slight decrease from the novel approvals in recent years. More than half of these approvals were for rare disease indications, with 25 drugs with orphan designation approved last year.