Blood and Clotting
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.
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.
US FDA approval of Niktimvo for third-line chronic GVHD, a competitor to Sanofi’s Rezurock, came less than two months ahead of anticipated approval of Syndax’s leukemia drug revumenib.
The company’s giroctocogene fitelparvovec, partnered with Sangamo, could compete with BioMarin’s hemophilia A gene therapy Roctavian, but longer-term data may be needed.
2seventy bio sold full rights to a hemophilia A program and in vivo gene editing technology to partner Novo Nordisk.
The Phase III ESLIM-01 study with HUTCHMED’s Syk inhibitor sovleplenib met its primary endpoint, demonstrating a 48.4% durable response rate for chronic immune thrombocytopenia, significantly higher than placebo. Global development appears to be accelerating and a multinational dose-finding Phase Ib study has also been initiated.
Having already posted Phase III success in non-transfusion dependent patients, Agios’s Pyrukynd now has shown the ability to reduce transfusions in alpha and beta thalassemia patients.
Cargo Therapeutics has got an early boost from its abstract to be presented the European meeting, while Shattuck has suffered on safety doubts for its CD47 inhibitor.
The company expects to have data from transfusion-dependent and non-transfusion dependent patients this year, with a potential expanded US approval in 2025 for Pyrukynd.
Mim8 looks good in hemophilia A but Hemlibra’s lead is all but unassailable.
The drug maker said the program would allow for continued reimbursement when patients change insurance plans. A Sangamo-partnered gene therapy for hemophilia A is also in the works.
The company announced positive topline results from a Phase II study of mezagitamab and expects to have five programs in Phase III development this fiscal year.
Gilead said the FDA requested a partial clinical hold on the studies as well, just over a week after the drug maker said it would not develop the magrolimab further for blood cancers.
The Phase III trial of BT524 produced positive topline results in patients with acquired fibrinogen deficiency, potentially making it the first fibrinogen concentrate to win US FDA approval for that indication.
The anti-C5 inhibitor sets an example of swift global trial and approval in China while fiercer competition awaits in the US, Europe and Japan.
The pricing watchdog said 94% of Fabhalta’s price comes from offsetting the cost of AstraZeneca’s Ultomiris, while Novartis said ICER’s analysis has “substantial challenges.”
The company’s new blockbuster eye treatment helped it maintain growth in 2023, while 2024 will see more strategic updates as the company broadens its disease area focus.
The biopharma industry’s fate lies largely in its R&D success and failures. Here, Scrip looks at five clinical trial failures that got readers clicking in 2023. Some were more comprehensive than others, highlighting the nuance that can be injected into clinical data in different settings and subgroups.
The company hopes to position mitapivat as an oral treatment for patients across the thalassemia spectrum. A Phase III trial in non-transfusion-dependent alpha- or beta-thalassemia was positive.