FDA
Analysts at Guggenheim Partners expect Voyxact to see “broad commercial uptake” given its relatively broad label compared with previous accelerated approvals for IgA nephropathy.
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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.
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The regulator has received reports that a group of patients treated with Adzynma had neutralizing antibodies against the protein the therapy replaces.
After revoking Sarepta’s award in July and awarding one to Krystal last month, the FDA’s platform technology designation program appears to be back on track. These six biotechs could be on the regulator’s radar.
A new analysis from Jefferies shows that drugs receiving breakthrough designations sail through the regulatory process more quickly, on top of frequently winning approval.
The nod brings Bayer face-to-face in the market with Boehringer Ingelheim and AstraZeneca, each of which has its own HER2 blocker for non-small cell lung cancer.
The approvals come as a pleasant surprise for Regeneron, analysts say, helping bolster the overall product profile of high-dose Eylea despite outstanding manufacturing concerns.
The FDA approval of the siRNA drug Redemplo caps off a tumultuous 12 months for Arrowhead, whose partnership with Sarepta caused its own stock to drop during the gene therapy maker’s safety troubles this summer.
Sarepta must also run a post-marketing study for Elevidys to better assess the risk of serious liver injury in patients dosed with the gene therapy.
Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation.
Unpredictable communication and a lack of transparency are eroding the industry’s and the public’s trust. The FDA, experts agree, needs to take control of the narrative.
The plausible mechanism pathway “could accelerate gene therapy/editing development,” analysts at William Blair said Thursday, while adding that additional clarity is needed.