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The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.
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With the biopharma industry performing better of late, analysts, executives and other industry watchers are “cautiously optimistic”—a term heard all over the streets of San Francisco at the J.P. Morgan Healthcare Conference earlier this month.
Bristol Myers Squibb, GSK and Merck are contributing drug ingredients as part of their deals with the White House but are keeping many of the terms of their agreements private.
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
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The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
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Following an initial report from Reuters, Merck KGaA confirmed that it is in talks with SpringWorks for a potential acquisition, though details of its offer have yet to be revealed.
Eli Lilly inked two collaborations on Monday, one focused on cardiometabolic diseases with South Korea’s OliX Pharmaceuticals and another for cancer therapies with Australia’s AdvanCell.
Vertex expects to make the newly approved non-opioid pain medicine Journavx available by the end of February.
Nerandomilast, an oral phosphodiesterase 4B inhibitor, hit the primary endpoint in a Phase III trial in progressive pulmonary fibrosis—nearly six months after achieving a similar feat in idiopathic pulmonary fibrosis. Boehringer plans to submit for FDA and other global approvals in both indications.
The intravenous antibiotic Emblaveo, a combination of aztreonam and avibactam approved in Europe last year, was developed in partnership with Pfizer. AbbVie acquired its share of the asset as part of its $63 billion buyout of Allergan.
Adding a new indication for the CAR T cell therapy could help BMS offset the loss-of-exclusivity headwinds it faces in the coming years.
The carve-out acquisition will allow Bain to capitalize on the “promising signs for growth” in the Japanese life sciences market, recently revitalized by more industry-friendly policy changes from the government.
It is unclear why an independent data safety monitoring board recommended the suspension of Pliant’s Phase IIb/III BEACON-IPF study in idiopathic pulmonary fibrosis.
AceLink is advancing a small-molecule treatment option for Fabry disease that could provide a more convenient alternative to the current enzyme replacement therapy standard.
Novo Nordisk’s bispecific antibody Mim8 prevented bleeding events and caused no adverse safety events in the Phase III FRONTIER3 trial. The company plans to file for approval this year, hoping to compete in the hemophilia A space with Roche’s blockbuster Hemlibra.