Pulse Newsletter | Volume 7 - Issue 2

On December 20, 2024, the U.S. Food and Drug Administration (FDA) approved Alhemo^® (concizumab-mtci) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years and older with hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors. Hemophilia A and hemophilia B are rare, inherited bleeding disorders in which the blood does not clot properly because it does not have enough blood-clotting proteins, known as clotting factors. Patients with hemophilia may bleed for a longer time than normal after injury or surgery and may have spontaneous bleeding in muscles, joints and organs, which can be life-threatening. Patients with hemophilia can be treated with the clotting factors they are missing, but they may develop inhibitors to these factors over time, causing these treatments to no longer be effective. Alhemo^® can be used in these patients to bypass this inhibitor effect.  

The efficacy and safety of Alhemo^® were evaluated in a multi-national, open-label phase 3 trial with 91 adults and 42 adolescent male patients with hemophilia A or B who have developed inhibitors. Efficacy was evaluated by comparing the number of treated bleeding episodes between the Alhemo^® treatment group and a group that did not use Alhemo^®. A ratio of the annualized bleeding rates (ABR) was estimated to 0.14, corresponding to a reduction in ABR of 86% for the Alhemo^® treatment group compared to the group without. Common side effects are injection site reaction and hives. Patients should be advised that treatment with Alhemo^® may increase the risk of blood clots.

On December 21, 2024, Alcon Laboratories announced a voluntary nationwide recall of one lot of Systane Ultra PF lubricant eye drops. The product is being recalled due to the presence of foreign material inside a sealed single-use vial that was determined to be fungal in nature. The fungal contamination of an ophthalmic product has the potential to cause eye infections, which could threaten the consumer’s vision.

Systane Ultra PF lubricant eye drops are used for temporary relief of burning and irritation in persons experiencing dry-eye symptoms. The affected product is packaged in a cardboard carton containing 25 sterile, single-use plastic vials and are labeled with lot number 10101 and an expiration date of September 2025. It was distributed nationwide to retail and internet outlets. To date, Alcon laboratories has not received any reports of adverse events related to this recall. Consumers should contact their physician or health care provider if they have experienced any problems that may be related to using this product and report it to the FDA’s MedWatch Adverse Event Reporting program.

On December 20, 2024, the FDA approved Zepbound^® (tirzepatide) for the treatment of moderate to severe obstructive sleep apnea (OSA) in adults with obesity, to be used in combination with a reduced-calorie diet and increased physical activity. OSA is a disorder in which a person’s upper airway becomes blocked during sleep, causing pauses in breathing. It is more common in people who are overweight or obese. Zepbound^® works by activating receptor of hormones secreted within the intestine to reduce appetite and food intake, leading to improvements in OSA symptoms by reducing body weight.

The approval for Zepbound^® in OSA was based on two randomized, double-blind and placebo-controlled studies of 469 adults without type 2 diabetes. In one study, participants enrolled were using positive airway pressure (PAP), the standard care for moderate to severe OSA. The other study enrolled participants unable or unwilling to use PAP. In both studies, participants randomly received either 10 or 15 milligrams of Zepbound^® or placebo once weekly for 52 weeks. After 52 weeks of treatment, participants who received Zepbound^® experienced a clinically meaningful reduction in events of apnea and significant decrease in body weight compared with those receiving placebo. With this, it is likely that the benefit for OSA is associated with the reduction in body weight for members who were in the treatment group.  

New Drug: Alyftrek™ (vanzacaftor, tezacaftor, and deutivacaftor)

Oral combination tablet containing vanzacaftor, tezacaftor and deutivacaftor, indicated for the treatment of cystic fibrosis, a rare genetic disease, in patients 6 years and older who have at least one responsive mutation in the CFTR gene. 

Motegrity^® (prucalopride)

Indication: For the treatment of chronic constipation.

Dosage Form/Strength: 1mg and 2mg tablets 

Average Wholesale Price (AWP): Generic = $572| Brand = $669