With four complete response letters behind it and seven months ahead of its April 2023 PDUFA date, Mallinckrodt plc’s Terlivaz (terlipressin) has been approved by the U.S. FDA for treating hepatorenal syndrome. Terlivaz had a decade-long series of obstacles before getting the long-awaited approval. The vasopressin analogue selective for V1 receptors was approved, in part, on results from the phase III CONFIRM trial of 300 patients, which met its primary endpoint of renal function improvement, avoidance of dialysis and short-term survival (p=0.012).
With the science on aging advancing, it’s time for the U.S. to modernize its regulatory approval path for new longevity treatments, members of a House Science, Space and Technology subcommittee were told Sept. 15.
From the beginning of the monkeypox outbreak in the U.S. in May, the federal government has bungled the response, according to both Democratic and Republican members of the Senate Health, Education, Labor and Pensions (HELP) Committee.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Akouos, Atyr, Biontech, Clovis, Cstone, H. Lundbeck, Hansa, I-Mab, Innocare, Merck, Novavax, Otsuka, Pfizer, TC.
The toxicity associated with oncology therapies is the stuff of legend with clinicians and patients, and thus the U.S. FDA and manufacturers have been working to fine tune these dosing regimens. However, the FDA's Oncology Center of Excellence (OCE) includes a programmatic effort to optimize dosing regimens, which led to an editorial by FDA officials that calls on industry to consider whether the maximum tolerated dose paradigm is really the optimal approach to oncology drug development.
Pricing new drugs for the U.S. market, especially those treating rare diseases, is getting a lot more complex now that the Medicare inflation rebate is in play. The rebate provision in the newly enacted Inflation Reduction Act incentivizes companies to set higher launch prices for drugs that will be used by Medicare beneficiaries since their future price increases will be limited to the rate of inflation. Although some of the other drug pricing measures included in the new law won’t kick in for a few years, the Medicare inflation rebate is to become effective next year.
More than four years after Right to Try legislation was enacted in the U.S., the FDA is issuing a final rule, Annual Summary Reporting Requirements Under the Right to Try Act, specifying the deadline and content for the annual reports the law requires participating drug sponsors or manufacturers to submit.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Acadia, Biolinerx, BMS, Chiesi, Mersana, Vaxxinity.
Bristol Myers Squibb Co. (BMS) dodged a black-box warning on the label of just-approved Sotyktu (deucravacitinib), but hurdles lie ahead for the first-in-class, oral, allosteric tyrosine kinase 2 inhibitor. Designed to treat adults with moderate to severe plaque psoriasis who are candidates for systemic therapy or phototherapy, Sotyktu is priced as $75,000 per year, and will become available during September, BMS said.
RSS
