Blood & Coagulation Disorders

Concizumab sponsor Novo Nordisk and a number of other companies could soon learn whether or not the European Medicines Agency will give their respective drugs the marketing thumbs up.

The US FDA approved the drug for hemophilia A and B, and while it may struggle to compete in hemophilia A against Roche’s Hemlibra, it has a big convenience advantage in hemophilia B.

Once the deal with Sanofi closes, the rare disease drug will become one of Recordati’s fastest forecast sellers.  

Hemgenix has now secured reimbursement in several European markets, with more talks ongoing. While the path to reimbursement has not always been easy, innovative access deals have helped to smooth the way.

The withdrawal amid concerns about the risk-benefit ratio reduces treatment options for sickle cell disease, but it is not expected to have much effect on the SCD market, including the gene therapies.

The withdrawal amid concerns about the risk-benefit ratio reduces treatment options for sickle cell disease, but it is not expected to have much effect on the SCD market, including the gene therapies.

The company’s decision to withdraw the drug was based on “the totality of clinical data that now indicates the overall benefit of Oxbryta no longer outweighs the risk in the approved sickle cell patient population.”

The European Medicines Agency has recommended the pan-EU approval of three rare disease drugs: Pfizer’s Hympavzi, Henlius Biotech’s Hetronify and ImmunoGen’s Elahere in addition to five other new medicines.

The European Medicines Agency is this week expected to decide whether to recommend for pan-EU marketing approval for Pfizer’s marstacimab, Henlius Biotech’s serplulimab and ImmunoGen’s mirvetuximab soravtansine.

Vadadustat's review overlapped with two other oral HIF-PH class drugs. Safety issues with FibroGen/AstraZeneca’s roxadustat drew attention during vadadustat's first-cycle review, while GSK’s Jesduvroq labeling and postmarketing requirements informed the second-cycle approval.