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 CNS 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. The long-acting C5 complement inhibitor is already approved for treating the indication in the EU and Japan. There are about 20,000 prevalent cases of the disease in the U.S., western Europe and Japan, according to Clarivate DRG data.

Timing everything in US Rx price negotiations

Forget location. When it comes to escaping the first round of Medicare price negotiations due to pending generic or biosimilar competition, it’s all about timing, timing, timing. If the U.S. Centers for Medicare & Medicaid Services (CMS) determines, by Aug. 1, 2024, that there will be genuine generic or biosimilar competition to one of the 10 selected drugs by 2026, no maximum fair price (MFP) will go into effect for that innovator, a CMS spokesperson told BioWorld. But if that determination comes a day later, the MFP will apply for 2026.

Amo points to clinical benefits in pivotal CDM study

Amo Pharma Ltd. is evaluating its next steps based on results from its pivotal phase II/III study testing AMO-02 (tideglusib) in children and adolescents with congenital myotonic dystrophy (CDM), a subtype of myotonic dystrophy type 1. While the trial did not meet the primary endpoint, as measured using a physician-completed rating scale, results indicated a clinically and statistically significant benefit on a range of functional and objective assessments such as cognitive performance and motor skills. Amo plans to talk to regulators about how to advance the AMO-02, a GSK3beta inhibitor, in CDM.

Time for US biosimilar path to catch up with the science?

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 (BCPIA) that created the framework for the U.S. biosimilar market. While biosimilars were largely unknown when the BPCIA was enacted in 2010, they have now tallied more than 2 billion patient days globally with no safety issues, Juliana Reed, executive director of the Biosimilars Forum, told BioWorld. In that time, she said, the analytics and science have evolved. Now, the development path needs to advance as well if the U.S. wants a robust biosimilars market.

BioWorld Insider Podcast – AI: driving drug development from effective to remarkable

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. Among the gems and eyebrow raisers is talk of dramatic reductions in the time it takes to identify the right molecule for development and how digital clinical trials in the not-too-distant future will substantially shrink study times. This episode also provides a preview of the annual Biofuture conference. Each year, a group of trailblazers, disruptors and forward-thinking executives converge to evaluate and forecast the future of health care. This year, BioWorld is a gold sponsor of the Oct. 4-6 event in New York. If you attend, you'll have the chance to hear panels and join workshops and fireside chats with key opinion leaders like Penberthy.

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