Approval of Biogen/Ionis’ Spinraza gives the drug development community a successful, collaborative model for a rapid pathway to full approval for a rare disease therapy; it also provides a timely, compelling rebuttal to claims that US FDA’s drug review operations are in dire need of reform.
The US FDA’s Dec. 23 approval of Biogen Inc./Ionis Pharmaceuticals Inc.’s Spinraza (nusinersen) for the rare neurological disease spinal muscular atrophy (SMA) may be the perfect antidote to the bad feelings that followed the difficult and internally divisive approval of Sarepta Therapeutics Inc.’s Exondys 51 (eteplirsen) by the same review group at FDA.
In a year marked by a steep drop in NME approvals (22 vs. 45), a jump in Complete Response letters...
The UK MHRA has emphasized the merits of the Project Orbis scheme, which has resulted in 12 new drug approvals and 14 indication extensions in the country so far. However, overall annual approval figures were lower in 2023 and 2024 than in the years prior.
The UK drug regulator has introduced several changes in recent months, such as the introduction of a decentralized manufacturing regulation. Matthew Durdy, CEO of the Cell and Gene Therapy Catapult, explains what these changes mean for the cell and gene industry.
Vinay Prasad’s exit from the US FDA reignited conversations about integrating its drugs and biologics centers under George Tidmarsh. Those plans appear to be shelved, but the leadership transition could be an opportunity to fully realize the vision for the Oncology Center of Excellence.
The European Medicines Agency is bringing back in-person oral explanation meetings with drug sponsors on a pilot basis, following industry feedback that direct engagement with its scientific committees is highly valued.
Acknowledging the risks and limitations of factorial designs, draft guidance outlines how sponsors might employ external data, such as registries or patient-level real-world data, for combination cancer therapies.
Enhancing the consistency and clarity of information requests and discipline review letters, and improving assessment milestone communications are key areas targeted by generic drug makers for user fee program negotiations.
Vinay Prasad’s exit from the US FDA reignited conversations about integrating its drugs and biologics centers under George Tidmarsh. Those plans appear to be shelved, but the leadership transition could be an opportunity to fully realize the vision for the Oncology Center of Excellence.
