ClinicaSpace

Mixed headlines have plagued the cell and gene therapy space of late. We believe that a renewed case of optimism is not only warranted but essential if these therapies are to reach their full potential.

Since July, several biotechs have been forced to pivot as previous agreements with the FDA around evidence required for approval were reversed, a phenomenon that, according to experts, could portend a more restrictive regulator.

A week into his tenure as head of the FDA’s Center for Drug Evaluation and Research, experts agree that Rick Pazdur is the “ideal fit” to stabilize the agency. And, according to one ex-FDA official, if his CBER counterpart Vinay Prasad tries to supersede Pazdur’s authority, “there will be hell to pay.”

Priority voucher awardees and regulators could feel pressure to “meet the moment” as FDA watchers question the intent and feasibility of the Commissioner’s National Priority Voucher program.

Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation.

One of the FDA’s potential approvals this month could break an existing monopoly in the treatment space for a rare growth disorder.

The cholesterol-lowering drug is part of a suite of medicines that also includes MariTide and that Amgen Chief Medical Officer Paul Burton hopes will make the company the “undisputed leader in the management of cardiometabolic risk for patients” by 2030.

A new generation of companies is eyeing the existing, sizeable hair loss market, hoping that better formulations and new scientific targets will finally produce treatments that are safer and more effective, as well as commercially successful.

The upheaval of the Health and Human Services workforce and leadership leaves much to be desired in terms of delivery, recently retired FDA Chief Information Officer Vid Desai tells BioSpace, but the regulatory agency is evolving to be more open to much needed change.

While the FDA continues to put out guidance documents and approve drugs, some companies are already reporting delays in dealings with the agency, while insiders warn of falling morale and a negative perception from the rest of the biopharma world.