Sanofi

Sanofi and partner Swedish Orphan Biovitrum shared positive topline results from their ongoing Phase III trial on a candidate drug for patients diagnosed with severe hemophilia A. 

BioMarin

BioMarin updated several of the company’s gene therapy programs on February 17 following a September 2021 update, when the drug manufacturer reported the U.S. Food and Drug Administration had placed a clinical hold on the BMN 307 Phearless Phase I/II trial.

BioMarin Pharmaceutical shared positive results from the company’s ongoing Phase III GENEr8-1 trial on the viability of valoctocogene roxaparvovec as a treatment for patients diagnosed with severe hemophilia A.

Genentech’s Hemlibra (emicizumab-kxwh) showed no new safety signals and continued to deliver consistent bleeding control in the final analysis of a long-term, late-stage hemophilia A study.

After touting positive Phase III results at the American Society of Hematology’s annual meeting, the U.S. FDA placed a clinical hold on uniQure”s hemophilia B treatment AMT-061 due to a patient developing liver cancer. 

Sanofi Hits the Brakes on Its Hemophilia Program Published: Nov. 6, 2020 By Mark Terry BioSpace   Three hemophilia organizations, the World Federation of Hemophilia, the European Haemophilia Consortium and […]

The U.S. Food and Drug Administration’s approval-review schedule for August includes the Biologics License Application for Bristol Myers Squibb and Juno Therapeutics’ lisocabtagene maraleucel (liso-cel) for relapsed or refractory large B-cell lymphoma after at least two previous therapies. 

Ahead of a planned Phase III study, Pfizer and Sangamo Therapeutics released follow-up data from its Phase I/II Alta study in hemophilia A that showed an investigational gene therapy treatment provided blood-clotting activity levels for more than one year.

Denmark’s Novo Nordisk paused three clinical trials of concizumab over safety issues. The studies are evaluating the drug for hemophilia A and B.

As pharma companies bolster their pipelines via multibillion-dollar acquisitions, drug manufacturers continue to heavily invest in the oncology space.