After a previous phase III failure, Savara Inc. kept at it and found success with molgramostim for the rare lung disease autoimmune pulmonary alveolar proteinosis. A second attempt, the pivotal phase III Impala-2 study of molgramostim, an inhaled form of recombinant granulocyte macrophage colony-stimulating factor for adults, hit its primary endpoint and left participants breathing easier. The results led Savara to say it would complete a BLA submission sometime in the first half of 2025, with filings in Europe and Japan to follow.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Abbvie, Astrazeneca, Botanix, Capricor, Carisma, Dermavant, Excellthera, Ionis, Janssen, Junshi, Otsuka, Roche.
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Astrazeneca, BenevolentAI, CDR-Life, Harmony, Medicure, Novo Nordisk, Nervgen, Rigel.
The industry is again raising concerns that new EU health technology assessment rules coming into force on Jan. 12, 2025, will have the opposite of the desired effect and slow down access to innovative therapies.
Novo Nordisk A/S’ CEO Lars Jørgensen is set to be the next executive in the U.S. Senate Health, Education, Labor and Pensions (HELP) Committee’s pharma parade of shame. HELP Chair Bernie Sanders (I-Vt.) announced June 24 that Jørgensen will testify before the committee Sept. 24 about his company’s U.S. pricing of its blockbuster semaglutide drugs, Ozempic and Wegovy.
After receiving a complete response letter (CRL) in 2023 related to device issues, North Chicago-based Abbvie Inc. received another CRL for ABBV-951 (foscarbidopa/foslevodopa) for treating motor fluctuations in advanced Parkinson’s disease, this time for issues at a third-party manufacturer.
Exsilio Therapeutics emerged from stealth mode on June 25, 2024, with $82 million from a series A financing that was co-led by Novartis Venture Fund and Delos Capital. The company plans to use naturally occurring, mobile genetic elements to integrate therapeutic genes at a defined location in the genome, making it safer than random integration, which can cause tumor formation.
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