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Pulse Newsletter

Your source for the latest industry trends and drug information news.

Volume 6 | Issue 1
January 1, 2024

Chief Authors: Rebecca Waite, PharmD
Contributing Authors: Gabrielle Graca, PharmD/MBA Candidate and Salome Mawi, PharmD/MBA Candidate

Clinical Pharmacy Update

FDA Approves Novel Gene Therapies for the Treatment of Sickle Cell Disease

On December 8, 2023, the U.S. Food and Drug Administration (FDA) approved Casgevy™ and Lyfgenia™, the first cell-based gene therapies developed and approved for the treatment of sickle cell disease (SCD) in patients age 12 and older. SCD is a rare genetic disorder occurring most commonly in African Americans. The disease involves a mutation in hemoglobin, the protein in red blood cells that carries oxygen throughout the body, causing red blood cells to form a crescent or “sickle” shape, subsequently restricting blood flow in vessels. This restriction in blood flow, along with a lower capacity to carry oxygen, leads to diminished oxygen delivery to tissues throughout the body. Patients can experience pain, organ damage, life-threatening disabilities, early death and a greatly reduced quality of life. Both newly approved SCD therapies work by genetically modifying the patient’s blood stem cells. The modified stem cells are then transplanted back into the patient to multiply. Casgevy™ uses CRISPR/Cas9 technology to edit the mutated DNA to produce HbF, a type of hemoglobin that is effective in facilitating oxygen delivery and preventing the sickling of red blood cells. In an ongoing clinical trial, all patients treated with Casgevy™ achieved successful engraftment, meaning there were no failures or rejections of the edited blood stem cells infused back into the body. The most common side effects were nausea, abdominal pain, fever with low white blood cell count, headache, itching and reduced level of platelets. Lyfgenia™ also edits the DNA of blood stem cells, modifying them to produce HbAT87Q. This form of hemoglobin functions similarly to hemoglobin A, the normal functioning hemoglobin in adults not affected by SCD. In a clinical trial, 88% of participants treated with Lyfgenia™ achieved complete resolution of vaso-occlusive events (VOE-CR), indicating its effectiveness in reversing the blockage of blood vessels by sickle cells. The most common side effects included mouth sores and reduced levels of platelets, white blood cells and red blood cells. Lyfgenia™ has a black box warning for an increased risk of blood cancer which has occurred in some study participants. The approval of these two new drugs represents medical advancements of cell-based gene therapies as well as hope for improved quality of life in patients suffering from SCD. Both Casgevy™ and Lyfenia™ received Orphan Drug Fast Track, Priority Review and Regenerative Medicine Advanced Therapy designations.

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Drug Safety

FDA Requires Updated Prescribing Information for Promethazine Hydrochloride Injection  

To reduce the risk of severe chemical irritation and damage to tissues, the FDA has issues labeling updates related to the administration of promethazine hydrochloride injection. Promethazine hydrochloride injection is indicated to help manage certain allergic reactions, motion sickness, post-operative nausea and vomiting, and as a sedative or adjunct to analgesics. The FDA recommends health care professionals administer promethazine hydrochloride injection by deep intramuscular administration, but if the injection must be administered intravenously, health care professionals should dilute promethazine hydrochloride injection and administer by intravenous infusion to reduce the risk of severe tissue injury. The FDA has also provided further instruction for promethazine hydrochloride injection in the event that intramuscular injection is not possible. Health care professionals, caregivers, and patients should report adverse events or side effects related to the use of this product to FDA's MedWatch Safety Information and Adverse Event Reporting Program.  

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From the Pharmaceutical Industry

Food Storage Tips That Help Prevent Foodborne Illness  

Practicing proper food storage in your refrigerator, freezer and cupboards is an important step in keeping yourself and others from being sickened by microorganisms such as Salmonella, E. Coli, and C. botulinum, which causes botulism. Adhering to the proper temperature for both food and kitchen appliances is one of the best ways to prevent or slow the growth of harmful bacteria. Foods that require refrigeration should not exceed two hours spent at room temperature, and refrigerators should be monitored weekly to ensure temperatures are kept at or below 40° F (4° C) while freezers should be 0° F (-18° C). Additionally, food should be stored in covered containers and monitored for spoilage regularly. Food that appears moldy, presents a change in color or texture, or otherwise looks questionable should be discarded. Storing food in the freezer is an excellent option to stop the growth of bacteria indefinitely without reducing the food’s nutrient value. Proper storage of non-refrigerated items such as canned goods is equally important; these foods should be stored away from cleaning products and chemicals and inspected for damage or leakage prior to consumption.  

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FDA Approvals

New FDA Approvals

New Drug: Fabhalta® (iptacopan) 

Oral capsules approved for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria, a rare blood disorder in which red blood cells and platelets are damaged by the immune system [12/05/2023 – NOVARTIS]

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New Generics

New Generics Entering the Marketplace

Risperdal Consta® (risperidone) 

Indication: Long-acting injectable antipsychotic used for the treatment of schizophrenia and for the maintenance treatment of bipolar 1 disorder.

Dosage Form/Strength: 12.5mg, 25mg, 37.5mg, and 50mg intramuscular injection

Average Wholesale Price (AWP): Generic = $1,400 | Brand = $1,475