Orchard Therapeutics secured the FDA’s first approval for an autologous gene therapy to treat the rare metabolic disease metachromatic leukodystrophy in children.
Following in the footsteps of Bristol Myers Squibb and Eli Lilly, AstraZeneca on Tuesday jumped into the radiopharmaceuticals space by acquiring Fusion Pharmaceuticals in a deal worth $2.4 billion.
Bluebird CFO Chris Krawtschuk in a statement said that the debt financing agreement with Hercules “underscores the value bluebird offers as a standalone gene therapy leader,” while also extending the biotech’s runway and “bolstering our ability to bring transformative treatments to patients and their families.”
The appellate court sided with Regeneron, agreeing with the pharma that a district court had wrongly dismissed its antitrust claims against Novartis because anti-VEGF therapies sold in pre-filled syringes (PFS) constitute their own and distinct market versus therapies sold in vials.
Bayer stated that it would request regulatory approval for its menopause drug after the daily pill was shown to ease hot flashes in a third late-stage trial, providing some relief for its pharmaceuticals unit that is reeling from a recent development setback.
New late-stage trial results for GSK’s Jemperli show improved overall and progression-free survival in a broader range of endometrial cancer patients, which could lead to a potential label expansion.
The foundation receives its funds from the wholly owned subsidiary Novo Holdings, through which it controls Wegovy maker Novo Nordisk as well as other companies.
The company stated it would cap out-of-pocket costs for its inhaled respiratory products at $35 per month in the United States, following a similar move by rival Boehringer Ingelheim earlier in the month.
Contineum Therapeutics joined the 2024 initial public offering class on Friday with an SEC filing. The biotech will use the IPO proceeds to complete a Phase II trial for its most mature candidate targeting multiple sclerosis.
The approval of Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) for metabolic dysfunction-associated steatohepatitis is expected to open doors for future therapies, while also setting the efficacy and safety bar for upcoming candidates.