The U.S. Food and Drug Administration (FDA) has recently approved two significant medical advancements: Alhemo (concizumab-mtci) for hemophilia and Sequana Medical NV's alfapump System for liver cirrhosis. These approvals mark important steps forward in treating these conditions and improving patient outcomes.
Alhemo Approved for Hemophilia
On December 20, 2024, the FDA approved Alhemo (concizumab-mtci) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors. This approval offers a new option for patients who have developed inhibitors, which can make traditional factor replacement therapy less effective.
The most common adverse reactions reported in at least 5% of patients were injection site reactions and hives (urticaria). Hypersensitivity reactions, including redness of skin (erythema), rash, itching (pruritus), and abdominal pain, have also been reported. The prescribing information provides detailed recommended dosing guidelines.
alfapump System Approved for Liver Cirrhosis
Also on December 20, the FDA approved Sequana Medical NV's alfapump System, an implanted pump indicated for the removal of excess peritoneal fluid in certain adult patients experiencing fluid buildup in the abdominal cavity due to liver cirrhosis. This system had previously received a Breakthrough Device designation, highlighting its potential to address an unmet medical need.
The alfapump System continuously drains excess fluid from the peritoneal cavity into the bladder, where it can be eliminated through normal urination. This approval offers patients an alternative to repeated doctor visits for paracentesis, a procedure to drain fluid from the abdomen. The alfapump System is intended for single-patient use only.
