VIENNA – There's a new term gaining attention in the health care sector. Defined as the "constellation of chronic noncommunicable diseases of aging," comprising cardiovascular disease, diabetes, obesity, cancer and neurodegenerative disease, "metabesity" represents a clear opportunity in the quest to extend healthspan, at least if biopharma can succeed in getting investors, partners and regulators on board.
Hailing it as the "first new mechanism of action in decades" to treat major depressive disorder (MDD), specifically treatment-resistant depression, Johnson & Johnson's Janssen unit disclosed the FDA's approval of Spravato (esketamine), a drug that works on the N-methyl-D-aspartate (NMDA) receptor. While a win for Janssen, the agency's nod might portend even better for other NMDA-targeting drugs that can be administered without all of Spravato's restrictions.
Novavax Inc. execs tried to shift the focus to promising secondary and exploratory endpoint data showing efficacy of its Resvax respiratory syncytial virus (RSV) vaccine on the severe impacts of RSV infection, but Wall Street refused to be diverted Thursday, sending company shares (NASDAQ:NVAX) to a 52-week low on news of the primary endpoint miss in the phase III Prepare maternal immunization study.
NEW YORK – Contrary to what the flurry of recent headlines might suggest, FDA approvals of the first gene and cell therapies didn't lead to the invention of value-based pricing. The model has circulated in the U.S. health care system over the years in various forms – risk-sharing arrangements and managed entry agreements, for example – but the introduction of potentially curative treatments, combined with accelerated approvals based on limited durability data, is putting the pressure on companies to reconsider how they view product launches and reimbursement.
NEW YORK – As the U.S. House Ways and Means Committee was hearing testimony from a handful of experts Tuesday morning on how to tackle the rising cost of drug prices, a few hundred miles away panelists at the BIO CEO & Investor Conference also were discussing the need to cut patients' out-of-pocket expenses. Addressing a roomful of biopharma execs and investors in the New York Marriott Marquis in Times Square, the panel members all agreed that the Trump administration's proposed International Pricing Index (IPI) payment model, aimed at reducing reimbursement levels for the Centers for Medicare & Medicaid Services, is not the way to do it.
NEW YORK – These days "it's almost mandatory to have a session" on immune-oncology, noted Roth Capital's Jotin Marango, kicking off the 21st annual BIO CEO & Investor meeting with a panel on reshaping the tumor microenvironment via immunotherapies. But, despite the excitement generated by what's happening in the field, there's "so much nuance and few people actually understand and appreciate it all," he added.
Sanofi SA's $4.8 billion bet on Nanobody developer Ablynx NV gained further validation Wednesday, with the pharma winning an FDA nod for Cablivi (caplacizumab-yhdp) in rare and sometimes fatal blood disorder acquired thrombotic thrombocytopenic purpura (aTTP). U.S. approval comes about six months after the first commercial nanobody – a single-domain antibody that offers the specificity with the advantages of a smaller-sized molecule – gained approval in Europe for the same indication.
Just ahead of its Nov. 13 PDUFA date, revefenacin won FDA approval, becoming the first once-daily nebulized bronchodilator cleared for chronic obstructive pulmonary disease in the U.S.
Briefing documents released ahead of Friday's meeting of the FDA's Anesthetic and Analgesic Drug Products Advisory Committee (AADPAC) suggest Acelrx Pharmaceuticals Inc. might have better luck the second time around with its sublingual 30-mcg tablet version of sufentanil given by way of a non-invasive, single-dose applicator.
Top-line results from the phase III PREVENT study testing Soliris (eculizumab) in rare disease neuromyelitis optica spectrum disorder (NMOSD) "far exceeded our expectations," said Ludwig Hantson, CEO of Alexion Pharmaceuticals Inc., which is now moving to engage regulators on submissions to expand use of the complement inhibitor in NMOSD, a complement-mediated disorder for which there currently are no approved therapies.
