Only a handful of gene therapies for inherited diseases are approved worldwide. Bluebird, holding two of them, has been one of the leading developers.
So Bluebird’s struggles in Europe are notable for the dozens of other biotech companies advancing gene-based treatments for uncommon diseases like cerebral adrenoleukodystrophy or severe beta thalassemia.
The company’s decision also reflects the differences in how therapies approved in Europe are paid for, with decisions on reimbursement left up to the governments of individual EU member states. Compared to the U.S., European countries can be more aggressive in demanding lower prices and, as many have single-payer healthcare systems, are better able to negotiate for larger discounts.
While Bluebird set a $1.8 million price for Zynteglo, the company proposed having countries reimburse for treatment over five years. Payments were linked to continued patient benefit. READ MORE
by Ned Pagliarulo
Source: biopharmadive.com
Novartis will acquire Mariana’s lead candidate MC-339, a radioligand therapy (RLT) designed to target small-cell lung cancer. Last year, Mariana had raised $175m in a Series B round from several funds and pharma giant Eli Lilly.
The company’s aspiration to expand the use of its obesity products to cardiovascular indications has been successful. In March, its blockbuster drug Wegovy was approved by the US Food and Drug Administration (FDA) for reducing the risk of cardiovascular diseases in obese or overweight adults.
Massachusetts-based Deciphera brings to the table an extensive kinase inhibitor pipeline, kinase drug discovery expertise, and a strong commercial and sales platform in the US and European markets that is meant to advance Ono’s capabilities and presence in the oncology space.
