Rare diseases
Novo’s latest investment comes just days after the U.S. FTC greenlit the highly contentious acquisition of Catalent, which analysts expect will help the pharma expand its production capacity.
Incyte is abandoning its ALK2 blocker zilurgisertib, which it was trialing for myelofibrosis-associated anemia, while iTeos will deprioritize the development of inupadenant after it failed to meet the biotech’s clinical bar in a Phase II study of metastatic non-small cell lung cancer.
Ocaliva recently failed to secure the FDA’s traditional approval for primary biliary cholangitis due to safety concerns.
The company had said in October said that it was looking to license out its ex vivo editor renizgamglogene autogedtemcel to focus its resources on its in vivo platform.
Vertex unveiled long-term durability data for Casgevy, while Beam presented Phase I/II findings for its investigational base editor BEAM-101, building up to a BLA by late 2026.
The FDA’s year-end rush includes nine target action dates, mostly for rare disease and cancer therapies.
By speeding lifesaving drugs’ way to market and focusing on the underlying causes of disease, the pathway has helped save many lives.
The payment scheme will tie gene therapy payments to improvements in health outcomes—and could potentially boost the uptake of these sickle cell disease treatments.
The agency’s warning letter outlines Applied Therapeutics’ failure to provide adequate information regarding the clinical trial—including a dosing error for govorestat—claims that the company said it had already addressed.
The cancers were diagnosed 19 to 92 months after Skysona treatment.
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