The rolling BLA submission began in December 2023, with timelines pre-agreed with the FDA, based on the results of the phase 3 ZIRCON study in ccRCC (NCT03849118). In the study, all coprimary and secondary end points were met. Specifically, the agent demonstrated a sensitivity of 86%, ...
(14) The proportion of BLA approvals did not change over the past decade (Figure 1), with an average of 23% of all new drug approvals (excluding biosimilars). The lowest percentage of BLAs was approved in 2013, with 8% compared to a high of 32% in 2015. The number of biosimilar ...
The draft guidance also describes pre-BLA meeting considerations, noting that FDA “strongly recommends” scheduling one. The draft guidance states that only one 90-minute pre-BLA meeting will typically be granted for a specific product or indication planned for an original marketing application. Such...
“Trodelvy’s approval is a major milestone in our transformation from a research-based organization to a fully-integrated biopharmaceutical company, underscoring our commitment to bring innovative therapies to patients with hard-to-treat ca...
After the FDA accepts an NDA or BLA, a Prescription Drug User Fee Act (PDUFA) date is assigned. This is the FDA’s target decision date for granting approval or denial. In cases following the standard review timeline, the PDUFA date is scheduled 10 months after the NDA/BLA submissio...
该疗法已获得 FDA 授予的孤儿药资格和罕见儿科疾病( RPD )资格。 目前,评估疗效和安全性的3期临床试验正在进行中,Orchard计划在2022年初与FDA讨论其潜在的BLA申请。 2022 FDA Regulatory Outlook for Gene Therapy Since the first gene therapy approval of tisagenlecleucel (Kymriah) in the US for the treatm...
A brief history of the development program and previous relevant communications with the FDA, including a summary of all conical and nonclinical studies conducted thus far and the ones planned for NDA/BLA Any substantial changes in the product development plans, if applicable ...
该疗法已获得FDA授予的孤儿药资格和罕见儿科疾病(RPD)资格。目前,评估疗效和安全性的3期临床试验正在进行中,Orchard计划在2022年初与FDA讨论其潜在的BLA申请。 2022 FDA Regulatory Outlook for Gene Therapy Since the first gene therapy...
100 101 Pre-new drug application (pre-NDA)/pre-biologics license application (pre-BLA) 102 meetings (21 CFR 312.47). 103 104 Post-action meetings requested 3 or more months after an FDA regulatory action other 105 than an approval (i.e., issuance of a complete response letter)...
They’re looking for greater rigor in the evidentiary standards and are looking for regulatory action when post-approval trials are either not conducted, not conducted meeting timelines, or conducted and show that a drug or device doesn’t work. I would echo what we are hearing from this ...