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Pulse Newsletter

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

Volume 5 | Issue 7
May 1, 2023

Chief Authors: Rebecca Waite, PharmD, Bonnie Broczkowski, PharmD & Colleen Peehler, PharmD
Contributing Authors: Yi (Eve) Zheng, PharmD/MBA Candidate, Gabrielle Graca, PharmD/MBA Candidate

Clinical Pharmacy Update

Joenja® Approved for Ultra-Rare Disease

On March 24, 2023, the U.S. Food & Drug Administration (FDA) announced their approval of Pharming’s Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in patients 12 years and older. APDS is an ultra-rare genetic disorder caused by mutations in the PIK3CD or PIK3R1 genes, which impact the development and function of white blood cells in the body. These mutations lead to lower production of the white blood cells that are used to fight infections. This impairment in the immune system results in patients experiencing recurrent infections, usually in the sinuses, ears and respiratory tract. Their lymph nodes get larger and can obstruct normal swallowing and breathing. They are also at greater risk for blood cell cancers, such as lymphoma. Joenja® is the first FDA-approved therapy specifically for the treatment of APDS, receiving orphan drug and rare pediatric disease designations and priority review. Joenja® inhibits portions of the mutated PIK3 gene in patients with APDS, improving white blood cell production and reducing the size of lymph nodes. It was evaluated in a placebo-controlled 12-week study of adult and pediatric patients 12 years and older with APDS and a documented variant in either PIK3CD or PIK3R1. Patients receiving Joenja® saw a reduction in lymph node size and 37% improvement in white cell counts compared to the placebo group. These markers indicate a correction of the underlying immune defect in patients with this disease. Joenja® was well-tolerated, with headache, sinusitis and eczema as the most reported side effects. It may also cause fetal harm and should not be used during pregnancy.

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Drug Safety

Dabigatran Etexilate Recall Due to Detection of N-nitroso-dabigatran (NDAB) Impurity

On March 22, 2023, the FDA announced Ascend Laboratories’ voluntary recall of dabigatran etexilate capsules, a medication used as an oral anticoagulant to lower the risk of stroke and blood clots. The recall of 75 and 150 mg capsules is due to the presence of a nitrosamine, N-nitroso-dabigatran, above the established Acceptable Daily Intake (ADI) level. Small amounts of nitrosamines are commonly present in food and water, but long-term exposure to higher levels may increase the risk of cancer. This recall is specific to certain lot numbers and packaging configurations. The full list of impacted products can be found on the FDA recall website.

Ascend Laboratories has notified wholesalers/distributors and pharmacies with existing impacted inventory to stop distribution and quarantine the product immediately. Wholesalers/distributors have been advised to recall the distributed product. Patients who have received the impacted lots of dabigatran etexilate 75 or 150 mg capsules are advised to continue taking their medication and contact their physician for advice regarding alternative treatment. Consumers can call Ascend Laboratories at 1-877-272-7901 with questions. They are advised to contact their health care provider if they have experienced any reactions. Adverse reactions relating to the recall should also be reported to the FDA’s MedWatch Adverse Event Reporting Program. To date, no cases of adverse events have been reported in association with the recalled products.

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From the Pharmaceutical Industry

OTC Naloxone Approval

On March 29, 2023, the FDA announced the approval of Narcan 4mg nasal spray to be used for over-the-counter (OTC) availability. Narcan (naloxone hydrochloride) is used in the reversal of opioid overdoses and is considered a life-saving medication. Previously, Narcan was only available as a prescription product acquired through a pharmacy. The manufacturer of Narcan provided the FDA with data showing that consumers understand and have demonstrated their ability to use the medication safely and effectively. This approval will allow Narcan to be purchased at convenience and grocery stores, gas stations, the pharmacy counter and online. The FDA granted OTC approval to Emergency BioSolutions and will be working with the manufacturer to make Narcan available as nonprescription as soon as possible.

In the United States, drug and opioid overdoses are still a major health issue, with nearly 102,000 fatal overdoses reported from November 2021 through October 2022. Many of these are due to the presence of synthetic opioids, such as fentanyl. Timely administration of Narcan nasal spray could have prevented many of these fatalities, reversing the effects of the opioids and allowing patients to receive medical care. The OTC approval of Narcan will increase accessibility throughout communities in the U.S. Along with this approval, the FDA is still implementing its Overdose Prevention Framework, focusing on appropriate opioid prescribing and utilization and decreasing overdose risks.

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FDA Approvals

New FDA Approvals

Novel Drug Approval: ZYNYZ™ (retifanlimab-dlwr)

Intravenous injection for the treatment of adult patients with metastatic or recurrent locally advanced Merkel cell skin cancer. [3/22/2023 – INCYTE CORP]

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New Generics

New Generics Entering the Marketplace

Noxafil® (posaconazole)

Indication: Treatment of yeast infection in the mouth or throat, including infections that have not responded to other treatments.

Dosage Form/Strength: 40mg/mL oral suspension

Average Wholesale Price (AWP): Generic = $1,641 | Brand = $1,727

Aubagio® (teriflunomide)

Indication: The treatment of relapsing forms of multiple sclerosis (MS).

Dosage Form/Strength: 7mg and 14mg tablets

Average Wholesale Price (AWP): Generic = $7,224 | Brand = $10,836