The U.S. FDA said it is investigating the risk of hematologic malignancies associated with Bluebird Bio Inc.’s Skysona (elivaldogene autotemcel), approved in 2022 as a one-time gene therapy for treating early active cerebral adrenoleukodystrophy in boys, ages 4 to 17.
Following a late-cycle review meeting with the U.S. FDA in September and the agency’s decision to skip the advisory committee meeting, expectations were high heading toward the PDUFA date for Applied Therapeutics Inc.’s priority NDA for govorestat in galactosemia. So the complete response letter issued by the FDA just ahead of the Nov. 28 PDUFA date, citing deficiencies in the clinical application, caught nearly everyone off guard.
Our immune cells are not just “defenders” against deadly viruses and pathogens but also a great balancer for tissue homeostasis. For neurological disorders, understanding the neuro-immune axis could be key to treating previously untreatable conditions such as autism spectrum disorder, according to Jun R. Huh, professor of immunology at Harvard Medical School.
Arovella Therapeutics Ltd. is heading toward the clinic with its lead product, ALA-101, which consists of a chimeric antigen receptor (CAR) targeting CD19 and invariant natural killer T (iNKT) cells.
Our immune cells are not just “defenders” against deadly viruses and pathogens but also a great balancer for tissue homeostasis. For neurological disorders, understanding the neuro-immune axis could be key to treating previously untreatable conditions such as autism spectrum disorder, according to Jun R. Huh, professor of immunology at Harvard Medical School.
The U.S. District Court for the Northern District of California ordered Lorik Papyan, who pleaded guilty three years ago to one count of unlicensed wholesale distribution of prescription drugs, to pay Gilead Sciences Inc. nearly $32 million in restitution to cover lost profits due to a counterfeit HIV drug scheme he was involved in.
The FDA’s August 2024 draft guidance for predetermined change control plans (PCCPs) for all device types has provoked some misgivings among industry, with both the Medical Device Manufacturers Association and the Advanced Medical Technology Association blasting the draft’s limitations on the scope of the changes that could be included in a PCCP.
The FDA announced a new pilot program for communication of medical device recalls, but industry may be wary of a program that seems to be driven toward early notification of what the agency believes “are likely to be high-risk recalls.” The agency’s device center states that this latest effort is designed to “improve the timeliness of communications” of corrective actions undertaken by device manufacturers. This would apply when the corrective action is as significant as a device removal and when the corrective action constitutes nothing more than an update to a product’s instructions for use.
Arovella Therapeutics Ltd. is heading toward the clinic with its lead product, ALA-101, which consists of a chimeric antigen receptor (CAR) targeting CD19 and invariant natural killer T (iNKT) cells.
Outlook Therapeutics Inc. is forging onward with plans to resubmit the BLA for ONS-5010, an ophthalmic version of Avastin (bevacizumab, Roche AG), after a missed endpoint in phase III with the VEGF binder for wet age-related macular degeneration (AMD).
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