Blood & Coagulation Disorders

The health technology assessment institute, NICE, has reversed its rejection of Sobi’s once-weekly drug for preventing and treating severe hemophilia A, meaning that the treatment has now secured reimbursement in three European countries.

Pfizer cited many reasons for ending sales of hemophilia B gene therapy Beqvez, including a lack of patient and doctor interest. The company no longer plans to develop AAV gene therapies.

This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.

Hematologists discussed the still limited treatment options for sickle cell disease at ASH, weighing the risks and benefits of disease-modifying versus curative therapies after the withdrawal of Pfizer’s Oxbryta (voxelotor).

Sanofi’s rilzabrutinib and Alvotech/Advanz Pharma’s golimumab (AVT05) are among five new entries on the European Medicines Agency’s latest monthly list of products for which marketing applications are currently under review. Both drugs are investigational and are yet to be approved anywhere in the world.

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include Hympavzi, Pfizer's treatment for adults and adolescents with severe hemophilia A or B without inhibitors.

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.