The multibillion-dollar market potential for obesity medications that analysts expect in the next decade signifies change is afoot for a patient population historically plagued with unsafe options and generally dismissed by investors and insurers. While glucagon-like peptide 1 receptor agonists have paved the way – offering hope-filled patients the first significant non-surgical weight loss benefit upwards of 15% – only a small portion of the obesity population and certain overweight patients are treated with medication, and even a smaller percentage receive insurance reimbursement. Still, the potential market size is astronomical. The U.S. CDC said the obesity rate in the U.S. alone has climbed from 30.5% of the population in 2000 to 42% by 2020.
Biogen now has the first FDA-approved biosimilar to Roche’s Actemra
The U.S. FDA has approved the first biosimilar to the Roche Group AG’s Actemra (tocilizumab). Tofidence (tocilizumab-bavi), a monoclonal antibody from Biogen Inc., was greenlighted Sept. 29 for treating rheumatoid arthritis in adults, and for treating polyarticular and systemic juvenile idiopathic arthritis in those ages 2 and older. It’s the first biosimilar approved to treat systemic juvenile idiopathic arthritis. Biogen has not said when Tofidence will launch. Actemra’s global sales in 2022 were $2.9 billion, a decline of 22% from the previous year as demand continued to drop. It was first approved in January 2010.
Syndax halts leukemia trial early for efficacy, plans FDA filing for revumenib this year
Syndax Pharmaceuticals Inc. is gearing up for a U.S. FDA filing on the back of positive data from a pivotal phase I/II study testing revumenib in adult and pediatric patients with relapsed/refractory KMT2A-rearranged acute myeloid leukemia and acute lymphoid leukemia. The trial met its primary endpoint with a complete response (CR) or CR with partial hematological recovery rate of 23% at an interim analysis, prompting the independent data monitoring committee to recommend stopping the trial. Should revumenib win FDA approval, it would be the first menin inhibitor to reach market, though Kura Oncology Inc.’s ziftomenib is hot on its heels.
Novo’s Rivfloza approved in US as new option for rare disease PH1
Three years after Alnylam Pharmaceuticals Inc. gained U.S. FDA approval of the first treatment for rare disease primary hyperoxaluria type 1 (PH1), Novo Nordisk A/S solidified its own marketing clearance for RNAi therapy Rivfloza (nedosiran). It is approved as an injection at 80-mg, 128-mg or 160-mg doses that can be given once per month to lower oxalate levels in children 9 and older and adults with PH1 and relatively preserved kidney function. Novo, with headquarters in Bagsvaerd, Denmark, and U.S. operations in Plainsboro, N.J., acquired Rivfloza through its $3.3 billion buyout in late 2021 of Dicerna Pharmaceuticals Inc.
With no injunction, US Rx price negotiations begin
Even as the court challenges continue, the first round of U.S. government price negotiations for selected Medicare Part D drugs officially began yesterday, Oct. 1, with manufacturers of those drugs having to sign agreements to participate in the process. Although nine constitutional challenges have been filed in various federal courts, only the Chamber of Commerce filed a motion for a preliminary injunction to stop the negotiations before they began. The U.S. District Court for the Southern District of Ohio denied that motion Friday, Sept. 29, at the same time it denied the Department of Health and Human Services’ motion to dismiss the Chamber’s complaint.
Perseverance on mRNA therapy research wins the 2023 Nobel Prize in Medicine after saving millions of lives
Researchers who follow their instincts and achieve slow results while trying to break barriers have little support. They replace it with persistence. This is the story of Katalin Karikó and Drew Weissman. What was once a dream in their minds was later a success. Their work together for decades was essential to achieving mRNA vaccines, and their perseverance was rewarded today, Oct. 2, with the 2023 Nobel Prize in Medicine.
US FDA approves Cyclopharm’s Technegas combo product for visualizing pulmonary ventilation
After a long and bumpy path to approval, the U.S. FDA has finally given the green light to Cyclopharm Ltd.’s Technegas combination product a day after the Sept. 29 PDUFA date. Technegas is a nuclear medicine functional lung ventilation imaging agent that produces a gas-like substance that is inhaled, allowing clinicians to see functional ventilation through the lungs. It uses radio-labeled carbon produced by using dried technetium-99 in a carbon crucible.