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Mrx CLINICAL ALERT MRx JUNE 2019 CLINICAL ALERT YOUR MONTHLY SOURCE FOR DRUG INFORMATION HIGHLIGHTS HOT TOPIC: completed 2-year START trial (n=12, GENE THERAPY APPROVED therapeutic cohort). Both studies EDITORIAL FOR SPINAL MUSCULAR enrolled patients with SMA type 1. ATROPHY Treatment with Zolgensma led to STAFF significant improvement in reaching The United States (US) Food and developmental motor milestones, Drug Administration (FDA) approved such as head control, sitting without EDITOR IN CHIEF onasemnogene abeparvovec-xioi support, and/or standing/walking ® Maryam Tabatabai (Zolgensma ) for the treatment of without assistance compared to the PharmD patients < 2 years of age with spinal natural history of the disease. A durable muscular atrophy (SMA) who have effect of nearly 4 years was reported bi-allelic mutations in the survival in the START long-term follow-up. EXECUTIVE EDITOR motor neuron 1 (SMN1) gene, which Zolgensma carries a Boxed warning Carole Kerzic instructs production of a protein critical for acute serious liver injury. RPh for motor neuron function. SMA is the leading genetic cause of infant Avexis/Novartis set a wholesale DEPUTY EDITORS mortality. The most common form, SMA acquisition cost (WAC) of $2.125 million Jill Bot type 1, occurs in about 1 in 10,000 for the 1-time Zolgensma dose, making PharmD newborns, leading to about 500 new it the world’s most expensive drug. cases per year in the US. Patients with The Institute for Clinical and Economic Stephanie Christofferson SMA type 1 experience progressive Review (ICER) published a value-based PharmD motor function decline beginning price benchmark for Zolgensma of $1.2 shortly after birth. If left untreated, million to $2.1 million to reach a cost- Jessica Czechowski permanent ventilation or death often effectiveness threshold of $100,000 to PharmD occurs by 2 years of age. Zolgensma $150,000 per life year gained (LYG). This is the only gene replacement therapy amount assumes durable effectiveness Lara Frick approved for SMA. It is administered and considers the positive results of PharmD, BCPS, BCPP as a 1-time intravenous (IV) infusion the SPR1NT trial in presymptomatic over 1 hour and is used as part of a patients (< 6 months of age). Safety Leslie Pittman multidisciplinary approach to treat SMA and efficacy of repeat administration PharmD type 1. In December 2016, the SMN2- have not been studied, nor has use in directed antisense oligonucleotide patients with advanced SMA (complete nusinersen (Spinraza®) became the limb paralysis or permanent ventilator first disease-modifying therapy to dependence). While Spinraza use after treat SMA (all types) in pediatrics and Zolgensma therapy has been reported, adults; maintenance therapy is given clinical benefits and risks have not been intrathecally every 4 months. established. Zolgensma is available as patient-specific kits through a specialty Zolgensma was granted Breakthrough pharmacy distributor. The company is Therapy and Orphan Drug designations working closely with payers to create as well as Priority Review. FDA-approval 5-year outcome-based agreements and of Zolgensma is supported by the novel pay-over-time options. Download at: ongoing STR1VE trial (n=21) and the magellanrx.com | JUNE 2019 According to the guideline, cardiovascular (CV) risk factors BIOSIMILAR CORNER should be assessed every 4 to 6 years in patients aged 20 to 39 years to determine the need for treatment. FDA GUIDANCE ON INTERCHANGEABLE The race- and sex-specific Pooled Cohort Equation (PCE) is recommended to estimate 10-year atherosclerotic BIOLOGICS CVD (ASCVD) risk for asymptomatic adults aged 40 to To date, 19 biosimilar products have been approved in 79 years. While use of PCE is best supported in non- the US, 7 of which are commercially available. Currently, Hispanic populations, other risk assessment tools, such no biosimilar is considered to be interchangeable with as the Framingham CVD risk score, Reynolds risk score, its reference product; therefore, pharmacists cannot Systematic COronary Risk Evaluation (SCORE), and QRISK/ substitute a biosimilar for the reference product JBS3, can be considered in Hispanics or populations with without involvement of the prescriber. As part of their enhanced risk (e.g., HIV infection, chronic inflammatory Biosimilars Action Plan, the FDA recently provided disease, low socioeconomic status). Use of the coronary standards for approval of interchangeable biologic artery calcium (CAC) score is reasonable to estimate agents in their final guidance titled, “Considerations ASCVD risk when uncertainty still exists. In addition, in Demonstrating Interchangeability with a Reference serum lipids should be assessed starting in childhood. Product Guidance for Industry.” Statins remain first-line therapy for hypercholesterolemia. The FDA advises that in addition to showing Due to their proven CV benefits in patients with type 2 biosimilarity, the sponsor must demonstrate that diabetes mellitus (T2DM), sodium-glucose cotransporter the proposed interchangeable biologic product is 2 (SGLT 2) inhibitors and glucagon-like peptide-1 receptor expected to yield the same clinical result compared to agonists (GLP-1RA) are recommended in patients with the reference product for all of the reference product’s T2DM and additional CVD risk factors. While low-dose FDA-approved indications. Data necessary to meet the aspirin is established for secondary prevention of ASCVD, interchangeability standards may include critical quality because of a lack of net benefit, it should only be considered attributes (e.g., safety, purity, potency), target/receptor for primary prevention in select patients aged 40 to 70 interactions, pharmacokinetics, and immunogenicity. years who are not at increased bleeding risk. Any difference between the proposed interchangeable product and the reference product should be justified and proven that it will not prohibit the same clinical BEHAVIORAL HEALTH CORNER results as the reference product in any given patient. In addition, if the product is administered more than once to a patient, alternating use between INSOMNIA TREATMENT SAFETY ALERT the proposed interchangeable biologic and the The FDA released a Drug Safety Communication reference product should not lead to increased safety regarding rare but serious injuries associated with risks or reduced efficacy. Considerations regarding prescription sedative-hypnotic agents used to treat interchangeability of container closure systems and insomnia. Over a 26-year period, a total of 66 cases delivery device components are also addressed. of complex sleep behaviors have been reported in Furthermore, insulins, historically regulated by the patients taking eszopiclone (Lunesta®), zaleplon FDA as small molecules, will be deemed biological (Sonata®), and zolpidem (Ambien®, Ambien CR®, products as of March 23, 2020. This transition will Edluar®, Intermezzo®, Zolpimist®). These included allow approval of insulins that are interchangeable sleepwalking, sleep driving, and other activities while with their brand competitors. not fully awake. Some cases resulted in death, even in patients taking the lowest recommended dose. As CVD PRIMARY PREVENTION GUIDELINES a result, the FDA is strengthening the warnings for the products listed to include a Boxed warning regarding The American College of Cardiology (ACC) and American sleep behaviors. Furthermore, a contraindication Heart Association (AHA) released a new guideline on will be added advising prescribers to avoid use in the primary prevention of cardiovascular disease (CVD). patients who have previously experienced complex The guideline emphasizes the importance of a healthy sleep behavior while taking any of these agents. lifestyle in patients with diabetes, hypercholesterolemia, Patients should stop taking their prescribed insomnia or hypertension. The ACC/AHA recommends a agent and contact their physician if they experience multidisciplinary, team-based approach and shared complex sleep behavior. decision-making between the patient and physician. 2 | JUNE 2019 DRUG INFORMATION HIGHLIGHTS • Update on consumer level recalls of generic • The American Urological Association (AUA) released antihypertension medications due to potential human consensus guidelines on the evaluation, testing, treatment, carcinogenic impurities: Vivimed Life Sciences issued and follow-up of recurrent urinary tract infections (UTI) a recall of 19 lots of losartan potassium 25 mg, 50 mg, in women. Initial treatment should be based on the and 100 mg tablets due to the detection of N-nitroso- local antibiogram. A short course (generally ≤ 7 days) N-methyl-4-amino butyric acid (NMBA) above the FDA’s of antibiotics is considered reasonable to treat acute acceptable exposure limit. Moreover, the FDA published cystitis; however, antibiotic-resistant cases may require 2 new tests, HRMS and RapidFire-MS/MS, as options for parenteral antibiotics (≤ 7 days). To decrease risk of regulators and industry to detect nitrosamine impurities. future UTIs, antibiotic prophylaxis and/or cranberry • As part of the continued effort to tackle the opioid crisis, consumption may be considered. Additionally, in peri- the FDA unveiled a new “Remove the Risk” campaign and post-menopausal women, risk of recurrent UTIs that provides a stepwise approach for patients to may be decreased with vaginal estrogen therapy if no properly dispose of unused prescription opioids. contraindications exist. Medicine take-back programs are the preferred method • ICER revised its value-based
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