While the U.S. FDA didn’t ask for more study data or have safety or efficacy concerns, it does want modifications to Alexion, Astrazeneca Rare Disease’s sBLA for Ultomiris (ravulizumab-cwvz) to treat adults with the rare central nervous system disease neuromyelitis optica spectrum disorder. The agency has issued a complete response letter (CRL) requesting changes to Ultomiris’ Risk Evaluation and Mitigation Strategy (REMS) to better validate patients’ meningococcal vaccination status or prophylactic administration of antibiotics before being treated. Read More

Forget location. It’s timing, timing, timing when it comes to escaping the first round of U.S. Medicare price negotiations due to pending biosimilar competition. Under the Inflation Reduction Act (IRA), only single-source drugs that have been approved for a specific length of time are subject to the forced negotiations, which focus on drugs with the biggest Medicare spend, not necessarily the highest price tag. Since the IRA gives biologics a 12-year safe harbor from negotiations, which aligns with the biologic exclusivity provided by the Biologic Price Competition and Innovation Act, it creates a scenario in which the innovator could be facing negotiations just as its first biosimilar competition prepares to launch. That creates a lot of what-ifs. Read More

While an “unexpected placebo effect” marred its primary endpoint, the pivotal phase II/III study testing AMO-02 (tideglusib) showed clinically significant benefits across a range of functional and objective assessments, according to developer Amo Pharma Ltd., which is prepping to meet with regulators to discuss potential approval for use in children and adolescents with congenital myotonic dystrophy type 1 (CDM1), an ultra-rare subtype of myotonic dystrophy type 1 for which no treatment options are available. Read More

Now that the U.S. FDA has nearly 15 years of experience with developing and implementing a biosimilar pathway, it’s time for that regulatory path to catch up with the science, according to experts that have been involved in biosimilar development even before Congress passed the Biologics Price Competition and Innovation Act that created the framework for the U.S. biosimilar market. Read More

Google’s Scott Penberthy joins the podcast for a visionary discussion that scans the horizon for startling changes artificial intelligence will bring to drug development in the relatively near future. Read More

New hires and promotions in the biopharma industry, including: Allay, Alx Oncology, Biogen, C4, Capricor, Convergent, Imvax, Invivyd, Mersana, Nanobiotix, Novartis, Nucleome, Oncoresponse, Phanes, Purespring, Quince, Siga, Trisalus. Read More

Biopharmas raising money in public or private financings, including: Anvesana, Apollo, Aptose, Hanmi, Imvax, Jiangsu Nhwa, Medexus, Nimbus, Nurexone, Rejuvenation, Tentarix, Trevena. Read More

Clinical updates, including trial initiations, enrollment status and data readouts and publications: ABM, ACM, Addex, Biohaven, Cantargia, Eledon, Escocap, Genmab, Iliad, Insmed, Invlos, Mindbio, Nuvalent, Oncopeptides, Oncorena, Plus, Rallybio, Sareum, Seagen, Stoke, Tonix, Tris. Read More

Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Aphios, Ascendis, Biondvax, Bloomsbury, Cidara, Eterna, Giiant, Idorsia, Imidomics, J&J, Kriya, KSQ, Lineage, Manaolana, National Resilience, Novo Nortisk, Orion, Palisade, Proteros, Royalty Pharma, Scinai, Tnax, Tramontane. Read More

Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Arcturus, Aspargo, Astrazeneca, CSL, Inmune, Italfarmaco, Krystal, Nykode, Uniqure, Viatris, Wave, X4. Read More