BioMarin has finally brought its hemophilia A gene therapy Roctavian market and is determined prove naysayers wrong, but must work with its limitations and a confusing US label.
BioMarin has gained US approval for Roctavian, the first ever hemophilia A gene therapy, and the company is confident it can prove doubters wrong about how popular the one-time treatment will be with patients and payers.
However, the high $2.9m price tag on Roctavian (valoctocogene roxaparvovec), a money-back guarantee for payers, and a lack of clarity...
Create an account to read this article
Already a subscriber?
The US FDA approved Insmed’s second product, the DPP1 inhibitor Brinsupri (brensocatib) for non-cystic fibrosis bronchiectasis. The company launched the drug immediately with a list price of $88,000 per year.
The US FDA approved Hernexeos for previously treated HER2-mutant NSCLC, making it the first oral option in a setting where drugs like AstraZeneca/Daiichi Sankyo’s Enhertu dominate.
Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
The company will start shipping its newly launched phenylketonuria drug within two weeks and has bought back global net sales obligations for the treatment.
Plus deals involving Basilea/Venatorx, CorMedix/Melinta, SERB/Y-mAbs, XOMA/Lava, XOMA/Hillevax, MaxCyte/Adicet/Anocca and Bausch Health/DURECT.
Lilly said prices governments pay in developed countries, including those in Europe, need to increase to make US drug prices lower, rebalancing costs as CEO David Ricks recently advocated.
The drug is one of multiple candidates in the class that have been pulled from development, though it is not the company’s lead candidate.
