Disc is seeking accelerated approval and priority review of its NDA submissionFDA decision to accept and file the NDA for review occurs within 60 days of submission WATERTOWN, Mass., Sept. 30, 2025 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announced the submission of a New Drug Application (NDA) to the U.S. Fo ...

Core Insights - Inovio Pharmaceuticals' shares increased by 20.6% following FDA's acceptance of a rolling submission timeline for the biologics license application (BLA) for INO-3107, aimed at treating recurrent respiratory papillomatosis (RRP) in adults [1][5] - The company plans to finalize the BLA submission in the coming months and seeks priority review, targeting FDA acceptance by the end of 2025 [1][2] - INO-3107 is designed to elicit a targeted T cell response against HPV-6 and HPV-11, which cause RRP, and has received breakthrough therapy and orphan drug designations in the U.S. [3][5] Company Developments - Inovio's BLA submission is supported by data from a completed phase I/II study that demonstrated the candidate's safety, tolerability, immunogenicity, and efficacy in patients with HPV-related RRP [6] - The FDA has indicated that the completed study could support a BLA filing under the accelerated approval program, negating the need for a pivotal phase III study [6] - A confirmatory study for INO-3107 will be initiated, enrolling 100 patients across the U.S. [7] Industry Context - Precigen, Inc. recently received FDA approval for Papzimeos, a treatment for RRP, which also holds breakthrough therapy and orphan drug designations [8] - Precigen's Papzimeos was approved based on data from a pivotal phase I/II study, which met primary safety and efficacy endpoints, eliminating the need for a confirmatory study [10] - Year-to-date, Inovio's shares have risen by 34.4%, significantly outperforming the industry average growth of 3.2% [4]

Core Insights - The FDA has accepted the supplemental New Drug Application (sNDA) for Zepzelca in combination with atezolizumab for first-line maintenance treatment of extensive-stage small cell lung cancer (ES-SCLC), with a PDUFA action date set for October 7, 2025 [1][2] - The sNDA submission is based on the Phase 3 IMforte trial results, which showed statistically significant improvements in progression-free survival (PFS) and overall survival (OS) for the combination therapy compared to atezolizumab alone [2][6] - Jazz Pharmaceuticals will host an investor webcast to discuss the Zepzelca data, featuring commentary from experts in the field [3] Company Overview - Jazz Pharmaceuticals is a global biopharma company focused on developing innovative medicines for serious diseases, including a growing portfolio of cancer treatments [25][26] - The company is headquartered in Dublin, Ireland, and is dedicated to transforming the lives of patients with limited therapeutic options [25][26] Industry Context - Small cell lung cancer (SCLC) accounts for approximately 13% of lung cancers in the U.S., with around 30,000 new cases reported annually [4] - SCLC is known for its aggressive nature and rapid spread, leading to a high unmet need for effective treatment options [4]

Core Insights - The FDA has accepted Ascendis Pharma's New Drug Application for TransCon CNP for treating children with achondroplasia, granting it Priority Review status with a PDUFA goal date of November 30, 2025 [1][2] - TransCon CNP has shown significant annualized growth velocity and multiple benefits beyond linear growth in clinical trials compared to placebo, with a safety profile similar to that of placebo [1][2] - Achondroplasia affects over 250,000 people globally and leads to various complications, necessitating new treatment options [3] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [4][5] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [5] Industry Context - Achondroplasia is a rare genetic condition caused by a variant in the FGFR3 gene, leading to serious complications beyond skeletal dysplasia, including muscular, neurological, and cardiorespiratory issues [3] - Current therapies for achondroplasia are limited, and there is a significant demand for new treatments that can improve health outcomes [2][3]