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1493 on Drugs and Therapeutics In Brief: New Recommendations for Use of Metformin in Renal Impairment ...p 51 Cariprazine Vraylar for Schizophrenia and Bipolar I Disorder ...p 51 Maestr

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IN THIS ISSUE

ISSUE

1433

Volume 56

Published by The Medical Letter, Inc • A Nonprofi t Organization

ISSUE No

1493

on Drugs and Therapeutics

In Brief: New Recommendations for Use of Metformin in Renal Impairment p 51

Cariprazine (Vraylar) for Schizophrenia and Bipolar I Disorder p 51

Maestro Rechargeable System for Weight Loss p 54

Mifepristone (Mifeprex) Label Changes p 55

In Brief: Cholic Acid (Cholbam) for Bile Acid Synthesis Disorders p 56

In Brief: Jadenu – A New Formulation of Deferasirox for Iron Overload online only

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51

on Drugs and Therapeutics

Take CME Exams

ISSUE

1433

Volume 56

ISSUE No

1493 Maestro Rechargeable System for Weight Loss Mifepristone (Mifeprex) Label Changes p 54p 55

In Brief: Cholic Acid (Cholbam) for Bile Acid Synthesis Disorders p 56

In Brief: Jadenu – A New Formulation of Deferasirox for Iron Overload online only

ALSO IN THIS ISSUE

IN BRIEF

New Recommendations for Use of

Metformin in Renal Impairment

The FDA has required labeling changes that replace

serum creatinine (SCr) with estimated glomerular

fi ltration rate (eGFR) as the parameter used to determine

the appropriateness of treatment with the biguanide

metformin (Glucophage, and others) in patients with renal

impairment These changes will allow more patients with

mild to moderate renal impairment to receive metformin,

which is generally the fi rst drug prescribed for treatment

of type 2 diabetes.

Metformin was previously contraindicated in women with

a SCr level ≥1.4 mg/dL and in men with a SCr level ≥1.5

mg/dL, but use of SCr as a surrogate indicator tends to

underestimate renal function in certain populations (e.g.,

younger patients, men, black patients, patients with

greater muscle mass) The calculation of eGFR takes into

account age, race, and sex, as well as SCr level, providing a

more accurate assessment of kidney function A literature

review summarized in an FDA Drug Safety Communication

concluded that, based on eGFR, metformin is safe to use in

patients with mild renal impairment and in some patients

with moderate renal impairment 1

The eGFR should be calculated before patients begin

treatment with metformin and at least annually thereafter

Metformin is now contraindicated in patients with an

eGFR <30 mL/min/1.73 m 2 , and starting treatment with

the drug in patients with an eGFR between 30 and 45 mL/

min/1.73 m 2 is not recommended If the eGFR falls below

45 mL/min/1.73 m 2 in a patient already taking metformin,

the benefi ts and risks of continuing treatment should be

assessed Metformin should be not be administered for

48 hours after an iodinated contrast imaging procedure in

patients with an eGFR <60 mL/min/1.73 m 2 or a history

of liver disease, alcoholism, or heart failure, or in those

receiving intra-arterial contrast, and the eGFR should be

re-evaluated before treatment is restarted ■

1 FDA Drug Safety Communication: FDA revises warnings

regard-ing use of the diabetes medicine metformin in certain patients

with reduced kidney function Available at: www.fda.gov/Drugs/

DrugSafety/ucm493244.htm Accessed April 14, 2016.

Cariprazine (Vraylar) for

Schizophrenia and Bipolar I Disorder

The FDA has approved cariprazine (Vraylar – Actavis), an

oral, once-daily, second-generation antipsychotic, for treat-ment of schizophrenia and for acute treattreat-ment of manic or mixed episodes associated with bipolar I disorder

Pronunciation Key Cariprazine : kar ip' ra zeen Vraylar : vray' lar

STANDARD TREATMENT — With the exception of clozapine

(Clozaril, and others), second-generation antipsychotics

are not clearly more effective than fi rst-generation

antipsychotics for treatment of schizophrenia, but in

usual doses they are much less likely to cause tardive dyskinesia Clozapine is usually reserved for refractory disease because of its potential for serious toxicity, including seizures and agranulocytosis Olanzapine

(Zyprexa, and generics) may be the most effective of the

other second-generation antipsychotics, but it is also the most likely to cause metabolic adverse effects Patients who do not respond to one antipsychotic may respond

to another Long-acting injectable formulations may be useful when adherence is a problem

For treatment of manic or mixed episodes associated

with bipolar I disorder, lithium, valproate, and

second-generation antipsychotics generally have similar effi cacy, but individual patients may have greater improvement with one or another, and adverse effects may vary widely

Table 1 Pharmacology

Class Second-generation antipsychotic Mechanism Dopamine (D 2 ) and serotonin (5-HT 1A ) partial

of action agonist; 5-HT 2A antagonist Formulation 1.5, 3, 4.5, 6 mg capsules

Metabolism Hepatic, primarily by CYP3A4 and (for

caripra-zine and DCAR 1 ) to a lesser extent by CYP2D6 Elimination Urine (21%)

Half-life Cariprazine: 2-4 days

1 DCAR and DDCAR are active metabolites equipotent to cariprazine.

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Table 3 Some Relative Adverse Effects of Second-Generation Antipsychotics

* Limited experience

Table 2 Results of Some Cariprazine Clinical Trials

Mean

Acute Exacerbation of Schizophrenia

n=732 4 Cariprazine 1.5 mg/d -19.4 -1.0

Cariprazine 3 mg/d -20.7 -1.1 Cariprazine 4.5 mg/d -22.3 -1.3 Risperidone 4 mg/d 5 -26.9 -1.5

n=604 6 Cariprazine 3 mg/d -20.2 -1.4

Cariprazine 6 mg/d -23.0 -1.5 Aripiprazole 10 mg/d 5 -21.2 -1.4

n=439 7 Cariprazine 3-6 mg/d -22.8 -1.4

Cariprazine 6-9 mg/d -25.9 -1.6

Acute Manic/Mixed Bipolar Episodes

n=492 8 Cariprazine 3-6 mg/d -18.6 -1.9

Cariprazine 6-12 mg/d -18.5 -1.9

n=235 9 Cariprazine 3-12 mg/d -15.0 -1.6

n=310 10 Cariprazine 3-12 mg/d -19.6 -1.9

1 All trials were randomized and double-blind Schizophrenia trials were 6 weeks in duration Bipolar I disorder trials were 3 weeks in duration.

2 The primary endpoint in the schizophrenia trials was mean change in the 181-point Positive and Negative Syndrome Scale (PANSS) The primary endpoint in the bipolar I disorder trials was mean change in the 61-point Young Mania Rating Scale (YMRS) Higher scores indicate more severe disease Each active treatment caused signifi cantly more improvement than placebo in each trial

3 Clinical Global Impressions of Severity of Illness; a 7-point scale with higher scores indicating more severe disease Each active treatment caused signifi cantly more improvement than placebo in each trial.

4 S Durgam et al Schizophr Res 2014; 152:450

5 The trial was not designed to compare cariprazine to this active control, which was included to ensure assay sensitivity.

6 S Durgam et al J Clin Psychiatry 2015; 76:e1574

7 JM Kane et al J Clin Psychopharmacol 2015; 35:367

8 JR Calabrese et al J Clin Psychiatry 2015; 76:284.

9 S Durgam et al Bipolar Disord 2015; 17:63

10 GS Sachs et al J Affect Disord 2015; 174:296.

DRUG INTERACTIONS — Cariprazine and its active

metabolites are substrates of CYP3A4 Concurrent use of a CYP3A4 inducer such as carbamazepine may reduce the effi cacy of cariprazine and is not recommended Unlike aripiprazole and brexpiprazole, cariprazine pharmacokinetics are not signifi cantly affected by inhibitors of CYP2D6.9

Both lithium and valproate can take days to weeks to

have a full therapeutic effect; patients with mania often

require adjunctive treatment with an antipsychotic drug

or temporary treatment with a benzodiazepine.1

PHARMACOLOGY — Cariprazine is metabolized by

CYP3A4 and, to a lesser extent, by CYP2D6 to its

equipotent active metabolites desmethylcariprazine

(DCAR) and didesmethylcariprazine (DDCAR) At

steady state, DDCAR serum concentrations are 300%

higher than those of cariprazine

Like aripiprazole and brexpiprazole, cariprazine and its

metabolites are partial agonists at dopamine D2

recep-tors and serotonin 5-HT1A receptors, and antagonists at

5-HT2A receptors Uniquely among second-generation

antipsychotics, cariprazine has about 10-fold greater

affi nity for the D3 receptor than for the D2 receptor; the D3

receptor may play a role in mood modulation.2

CLINICAL STUDIES — Randomized, double-blind trials

comparing cariprazine to placebo for treatment of

schizo-phrenia over 6 weeks or treatment of acute manic or mixed

episodes associated with bipolar I disorder over 3 weeks

are summarized in Table 2 Cariprazine was signifi cantly

more effective than placebo for both indications.3-8

ADVERSE EFFECTS — Adverse effects occurring in

≥5% of patients taking cariprazine and more frequently

than in those taking placebo in at least one clinical trial

have included extrapyramidal symptoms, akathisia,

dyspepsia, vomiting, somnolence, and restlessness

Mean weight gain over 6 weeks was 0.5-0.7 kg greater

with cariprazine than with placebo

PREGNANCY — There are no adequate studies of

cariprazine in pregnant women Administration of the

drug to pregnant rats at doses lower than the maximum

recommended human dose caused fetal malformations,

developmental delays, and decreased pup survival

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DOSAGE AND ADMINISTRATION — Cariprazine should

be taken once daily Patients beginning treatment

should take 1.5 mg on day 1 Increasing the dose

to 3 mg on day 2 is recommended for patients with

bipolar I disorder and permitted for patients with

schizophrenia The daily dose can then be adjusted in

1.5- or 3-mg increments up to a maximum of 6 mg If a

strong CYP3A4 inhibitor must be coadministered with

cariprazine, dosage adjustments should be made as

described in the package insert

CONCLUSION — In short-term trials, cariprazine

(Vraylar) was more effective than placebo in treating

acute exacerbations of schizophrenia and acute

manic or mixed episodes associated with bipolar I

disorder It appears to have relatively mild metabolic

adverse effects and is long-acting, but extrapyramidal

symptoms can be a problem and its long-term safety

is unknown Aripiprazole (Abilify, and generics), a

similar drug that has a longer record of effi cacy and

safety and is now available generically, may be a

better choice ■

Table 4 Some Oral Second-Generation Antipsychotics

Usual Adult Dosage

Aripiprazole 2 – generic 2, 5, 10, 15, 20, 30 mg tabs 10-30 mg once/d 15-30 mg once/d $598.30

Asenapine – Saphris (Forest) 2.5, 5, 10 mg sublingual tabs 5-10 mg bid 5-10 mg bid 915.50

Brexpiprazole – Rexulti (Otsuka/Lundbeck) 0.25, 0.5, 1, 2, 3, 4 mg tabs 2-4 mg once/d N/A 934.70

Cariprazine – Vraylar (Actavis) 1.5, 3, 4.5, 6 mg caps 1.5-6 mg once/d 3-6 mg once/d 1006.10

Iloperidone – Fanapt (Novartis) 1, 2, 4, 6, 8, 10, 12 mg tabs 6-12 mg bid N/A 1139.20

Lurasidone – Latuda (Sunovion) 20, 40, 60, 80, 120 mg tabs 40-160 mg once/d N/A 921.90 Olanzapine 2 – generic 2.5, 5, 7.5, 10, 15, 20 mg tabs 10-20 mg once/d 10-20 mg once/d 15.60

Quetiapine – generic 25, 50, 100, 200, 300, 150-750 mg/d divided 400-800 mg/d divided 44.40

extended-release – Seroquel XR 50, 150, 200, 300, 400 mg ER tabs 400-800 mg once/d 400-800 mg once/d 750.30 Risperidone 2 – generic 0.25, 0.5, 1, 2, 3, 4, mg tabs; 4-8 mg/d divided 1-6 mg/d divided 29.70

N/A = Not FDA-approved for this indication; ODT = orally disintegrating tablets; ER = extended-release

1 Approximate WAC for 30 days’ treatment at the lowest usual adult dosage in schizophrenia WAC = wholesaler acquisition cost or manufacturer’s published price to wholesalers; WAC represents a published catalogue or list price and may not represent an actual transactional price Source: AnalySource® Monthly April 5, 2016 Reprinted with permission by First Databank, Inc All rights reserved ©2016 www.fdbhealth.com/policies/drug-pricing-policy

2 Also available parenterally.

3 Clozapine can cause seizures and agranulocytosis and should be reserved for patients with schizophrenia who fail to respond to other drugs

1 Drugs for psychiatric disorders Treat Guidel Med Lett 2013; 11:53.

2 JM Beaulieu et al Regulation of Akt signaling by D2 and D3 dopamine receptors in vivo J Neurosci 2007; 27:881.

3 S Durgam et al An evaluation of the safety and effi cacy of cariprazine in patients with acute exacerbation of schizophre-nia: a phase II, randomized clinical trial Schizophr Res 2014; 152:450.

4 S Durgam et al Cariprazine in acute exacerbation of schizo-phrenia: a fi xed-dose, phase 3, randomized, double-blind, placebo- and active-controlled trial J Clin Psychiatry 2015; 76:e1574.

5 JM Kane et al Effi cacy and safety of cariprazine in acute exacerbation of schizophrenia: results from an international, phase III clinical trial J Clin Psychopharmacol 2015; 35:367.

6 JR Calabrese et al Effi cacy and safety of low- and high-dose cariprazine in acute and mixed mania associated with bipolar I disorder: a double-blind, placebo-controlled study J Clin Psy-chiatry 2015; 76:284.

7 S Durgam et al The effi cacy and tolerability of cariprazine in acute mania associated with bipolar I disorder: a phase II trial Bipolar Disord 2015; 17:63.

8 GS Sachs et al Cariprazine in the treatment of acute mania in bipolar I disorder: a double-blind, placebo-controlled, phase III trial J Affect Disord 2015; 174:296.

9 Inhibitors and inducers of CYP enzymes and P-glycoprotein Med Lett Drugs Ther 2016; 58:e46.

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Table 1 Procedures for Weight Loss 1

Excess

Adjustable gastric An adjustable band squeezes the stomach 47% 3 Band slippage and erosion, excess vomiting,

Biliopancreatic Combines a restrictive procedure with a ≥70% 1 Higher risk of complications and death, diversion with procedure that bypasses about three quarters protein and nutrient defi ciencies

duodenal switch of the small intestine

Gastric balloon device Silicone balloon(s) placed in stomach endoscopic- 27-28% 4,5 Gastric ulceration, abdominal pain,

ally and inflated with a sterile solution nausea, vomiting, abdominal distention Roux-En-Y gastric Proximal 20-30 mL pouch of stomach is anasto- 66% 6 Perioperative mortality rate of 0.2-2.1%, bypass mosed to a limb of the jejunum, bypassing most nutrient defi ciencies, dumping syndrome

of the stomach, all of the duodenum, and the fi rst (nausea, bloating, colic, diarrhea), hyper-

Sleeve gastrectomy Most of the greater curvature of the stomach is 59% 7 Dumping syndrome, aggravation of

Vagus nerve blockade Device delivers intermittent electrical pulses to 24% 9 Pain at neuroregulator site, heartburn,

block vagus nerve signals between the stomach nausea, belching, abdominal pain and the brain

1 Med Lett Drugs Ther 2015; 57:21.

2 Defi ned as the weight in kilograms above the weight at a BMI of 25.

3 ≥10 years follow-up PE O'Brien et al Ann Surg 2013; 257:87.

4 At 24 weeks J Ponce et al Surg Obes Relat Dis 2015; 11:874.

5 At 9 months Results of an unpublished trial summarized in the Orbera package insert Excess weight loss was defi ned as weight above ideal body weight lost

6 ≥2 years follow up N Puzziferri et al JAMA 2014; 312:934.

7 ≥5 years follow up T Diamantis et al Surg Obes Relat Dis 2014; 10:177.

8 R Peterli et al Ann Surg 2013; 258:690.

9 At 12 months S Ikramuddin et al JAMA 2014; 312:915

Maestro Rechargeable System for

Weight Loss

The FDA has approved the Maestro Rechargeable

System (EnteroMedics), a subcutaneously implanted

device, for use in adults who have not been able to lose

weight with a weight loss program within the past 5 years

and who have a body mass index (BMI) of 40 to 45, or a

BMI ≥35 and at least one obesity-related comorbidity

PROCEDURES FOR WEIGHT LOSS — Surgical treatment

for obesity is generally limited to patients with a BMI ≥40,

or a BMI ≥35 with an obesity-related comorbidity such

as diabetes, hypertension, or hypercholesterolemia

Procedures that cause malabsorption (Roux-en-Y

gastric bypass and biliopancreatic diversion) result

in greater weight loss and more adverse effects than

purely restrictive procedures such as adjustable gastric

banding or sleeve gastrectomy.1 Gastric balloon devices

are inserted endoscopically and can be left in place for up

to 6 months; when they are removed, patients generally

regain a substantial fraction of the weight they had lost.2

THE NEW DEVICE — The Maestro Rechargeable System,

which consists of a rechargeable neuroregulator disc,

two wire leads, and two electrodes, utilizes

high-frequency electrical pulses to block vagus nerve

signals between the stomach and the brain According

to the manufacturer, vagal blocking therapy reduces

appetite by enhancing satiety and ultimately decreases

food intake The 2.75 x 3.5 inch neuroregulator

is implanted subcutaneously in the thoracic side

wall; it delivers intermittent electrical pulses to the electrodes, which are placed laparoscopically on the anterior and posterior vagal nerve trunks above the gastroesophageal junction

The manufacturer recommends that the device

be programmed to deliver intermittent electrical pulses over 13 hours while the patient is awake The neuroregulator is recharged by a mobile battery and radiofrequency transmitter that is held or strapped over the neuroregulator while charging The battery level should be checked daily and charged for about 30-60 minutes when low The neuroregulator is designed to operate for up to 8 years According

to the manufacturer, the list price of the Maestro Rechargeable System is $19,000.

CLINICAL STUDIES — In a double-blind trial (EMPOWER),

294 patients were randomized to active treatment with

the Maestro Rechargeable System for 9-16 hrs/day or

to a sham device (delivering a low frequency of electrical impulses, but not enough to affect vagus nerve function)

in addition to weight loss counseling sessions Patients controlled the amount of time per day that the device delivered impulses; more than half of the patients in the active treatment group used the device ≤9 hrs/day After 12 months, there was no signifi cant difference between the 2 groups in the percent of excess weight lost ("excess weight" is the weight in kilograms above the weight at a BMI of 25).3 A subgroup analysis found that the amount of weight lost was linearly related to the time the device was used

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In another double-blind trial (ReCharge), 239 patients with

a BMI of 40 to 45 or a BMI ≥35 and at least one

obesity-related comorbidity were randomized to treatment with

the Maestro Rechargeable System or to a sham device in

addition to weight loss counseling sessions The devices

were programmed to deliver a charge for ≥12 hours daily,

but the sham device had no leads At 12 months, the

actively-treated group had a mean excess weight loss of

24.4% compared to 15.9% in the sham group; although the

difference was statistically signifi cant, the primary effi cacy

endpoint of a ≥10% superiority margin was not met At 12

months, 52% of actively-treated patients achieved ≥20%

excess weight loss and 38% achieved ≥25% excess weight

loss, compared to 32% and 23%, respectively, with sham

therapy4; among the 84 patients who were moderately

obese (BMI 35-40), those receiving active treatment

achieved a 33% excess weight loss, compared to 19%

with sham therapy.5 Weight loss was sustained through

18 months in patients treated with the active device, while

those treated with the sham device regained >40% of the

weight lost between 12 and 18 months.6

An open-label study in 28 obese patients with type

2 diabetes found that mean excess weight loss was

9% at 1 week and 25% at 12 months with the Maestro

Rechargeable System At 12 months, HbA1c levels

had decreased by 1% and, among 15 patients with

elevated blood pressure, mean arterial pressure

had decreased by 8 mm Hg.7 Reductions in weight,

HbA1c, and blood pressure were sustained through

24 months.8

ADVERSE EFFECTS — In the ReCharge trial, serious

adverse events related to vagal nerve blockade and

intra-abdominal surgery occurred in 8.6% of patients.4

Pain at the neuroregulator site occurred in about 40% of

patients, whether they received the active or sham device

Heartburn/dyspepsia, nausea, dysphagia, eructation,

and abdominal pain occurred more frequently with the

active device

Use of the Maestro device is contraindicated

in patients with cirrhosis, portal hypertension,

esophageal varices, or a hiatal hernia that cannot be

corrected by surgery, and in those who have another

implanted device such as a pacemaker or defi brillator

Patients with the device should not undergo magnetic

resonance imaging (MRI) scans

CONCLUSION — Vagal blocking therapy with the

Maestro Rechargeable System is less effective than

bariatric surgery for treatment of obesity How it

compares to less invasive procedures such as gastric

balloon devices is not clear ■

Mifepristone (Mifeprex) Label

Changes

The FDA has approved several signifi cant changes in

the labeling of mifepristone (Mifeprex – Danco), an oral

antiprogestin that has been used in the US for more than

15 years for termination of intrauterine pregnancy.1 It has generally been used with the prostaglandin analog

misoprostol (Cytotec, and generics)

TIMING OF USE — Under the new labeling, mifepristone

and misoprostol can now be used to terminate pregnancy for up to 70 days, rather than 49 days, after the fi rst day of the last menstrual period Multiple studies have shown that the combination is >96.5% effective through 63 days of gestation; data on its effectiveness between 64 and 70 days are much more limited.2 In a retrospective cohort study of data from more than 30,000 women undergoing either medical

or surgical abortion before 64 days of gestation, effi cacy was >99.5% in both groups with no difference

in major adverse events.3

DOSAGE AND ADMINISTRATION — The new labeling

recommends a 200-mg oral dose of mifepristone (rather than 600 mg) followed by 800 mcg of misoprostol (rather than 400 mcg) administered buccally (rather than orally) 24-48 hours later (rather than 48 hours later) Additionally, in-offi ce administration of misoprostol is no longer required; women can now self-administer misoprostol at home.4 Follow-up with the provider to confi rm

1 Diet, drugs, and surgery for weight loss Med Lett Drugs Ther 2015; 57:21.

2 ReShape and Orbera – two gastric balloon devices for weight loss Med Lett Drugs Ther 2015; 57:122

3 MG Sarr et al The EMPOWER study: randomized, prospec-tive, double-blind, multicenter trial of vagal blockade to induce weight loss in morbid obesity Obes Surg 2012; 22:1771

4 S Ikramuddin et al Effect of reversible intermittent intra-ab-dominal vagal nerve blockade on morbid obesity: the ReCharge randomized clinical trial JAMA 2014; 312:915

5 JM Morton et al Effect of vagal nerve blockade on moderate obesity with an obesity-related comorbid condition: the Re-Charge Study Obes Surg 2016 April 5 (epub).

6 SA Shikora et al Sustained weight loss with vagal nerve block-ade but not with sham: 18-month results of the ReCharge trial

J Obes 2015 July 12 (epub).

7 S Shikora et al Vagal blocking improves glycemic control and elevated blood pressure in obese subjects with type 2 diabetes mellitus J Obes 2013 July 30 (epub)

8 SA Shikora et al Intermittent vagal nerve block for improve-ments in obesity, cardiovascular risk factors, and glycemic control in patients with type 2 diabetes mellitus: 2-year results

of the VBLOC DM2 study Obes Surg 2015 October 15 (epub).

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Table 1 Mifeprex Label Changes

▶ Can be used later in pregnancy, up to 70 days from fi rst day

of last menstrual period (vs 49 days)

▶ Lower 200-mg PO dose of mifepristone (vs 600 mg)

▶ Higher 800-mcg buccal dose of misoprostol (vs 400 mcg PO)

▶ Misoprostol can be taken at home 24-48 hours later (vs 48

hours later in-offi ce), reducing the number of total offi ce

visits from 3 to 2

▶ Follow-up visit 7-14 days after mifepristone administration

(vs 14 days)

1 Mifepristone (RU 486) Med Lett Drugs Ther 2000; 42:101.

2 MJ Chen and MD Creinin Mifepristone with buccal misoprostol

for medical abortion: a systematic review Obstet Gynecol 2015;

126:12.

3 LD Ireland et al Medical compared with surgical abortion for

effective pregnancy termination in the fi rst trimester Obstet

Gynecol 2015; 126:22.

4 K Iyengar et al Home use of misoprostol for early medical

abor-tion in a low resource setting: secondary analysis of a

random-ized controlled trial Acta Obstet Gynecol Scand 2016; 95:173.

5 T Middleton et al Randomized trial of mifepristone and

buc-cal or vaginal misoprostol for abortion through 56 days of last

menstrual period Contraception 2005; 72:328.

complete termination of pregnancy and evaluate the

degree of bleeding is recommended between 7 and

14 days after mifepristone administration (rather

than 14 days) The new regimen reduces the number

of offi ce visits from 3 to 2

Use of the lower dose of mifepristone has been

common practice for many years Misoprostol tablets

have been administered orally, sublingually, buccally,

and intravaginally The oral route has been less

effective and caused more adverse effects (nausea,

diarrhea) than intravaginal administration Buccal

administration has been as effective as intravaginal

administration and more effective than oral

administration, with only a slightly higher incidence

of adverse effects than the intravaginal route.5-7

REMS PROGRAM — Mifepristone will continue to be

available only through a REMS program to certifi ed

prescribers who meet specifi ed qualifi cations and

agree to follow the guidelines for appropriate use

of the drug Patients are also required to sign an

agreement form The drug must be dispensed in

a clinic, medical offi ce, or hospital by or under the

supervision of a certifi ed prescriber, and ultrasound

must be available

CONCLUSION — The FDA’s changes to the labeling

of mifepristone (Mifeprex) recommend use of a lower

dose of mifepristone, use of the drug later in the fi rst

trimester, self-administration of misoprostol (Cytotec,

and generics) at a higher dose and by the more

effective buccal route, and fewer offi ce visits ■

IN BRIEF

Cholic Acid (Cholbam) for Bile Acid

Synthesis Disorders

The FDA has approved oral cholic acid (Cholbam –

Retrophin) for treatment of children and adults with bile acid synthesis disorders caused by single enzyme defects and for adjunctive treatment of peroxisomal disorders such as Zellweger spectrum disorders in patients who have liver disease, steatorrhea, or complications from fat-soluble vitamin malabsorption Patients with these rare inborn errors of bile acid metabolism cannot synthesize primary bile acids such as cholic acid, resulting in reduced bile flow, decreased absorption of fat and fat-soluble vitamins, and development of liver disease, which can be

fatal Cholbam is the fi rst drug to be approved in the US for

these indications

FDA approval was based on a long-term, single-arm clinical trial, an extension of that trial, and case reports in a total of 77 patients with bile acid synthesis disorders and

34 patients with peroxisomal disorders Administration

of cholic acid appeared to decrease hepatic injury and increase height and weight; 67% of patients with bile acid synthesis disorders and 42% of those with peroxisomal disorders treated in the clinical trials survived for more than 3 years 1 Some of these survivors have been treated successfully for more than 20 years Diarrhea has been the most common adverse effect.

Cholbam is available in 50- and 250-mg capsules The

usual dosage is 10-15 mg/kg taken once daily or in two divided doses The cost of 30 days’ treatment for a 50-kg patient at a daily dose of 10 mg/kg is about $50,000 2 ■

1 FDA Medical review: cholic acid (Cholbam) Available at: www.accessdata.fda.gov/drugsatfda_docs/nda/2015/ 205750Orig1s000MedR.pdf Accessed April 14, 2016.

2 Approximate WAC WAC = wholesaler acquisition cost or man-ufacturer’s published price to wholesalers; WAC represents a published catalogue or list price and may not represent an ac-tual transactional price Source: AnalySource® Monthly April

5, 2016 Reprinted with permission by First Databank, Inc All rights reserved ©2016 www.fdbhealth.com/policies/drug-pricing-policy

6 B Winikoff et al Two distinct oral routes of misoprostol in mife-pristone medical abortion: a randomized controlled trial Obstet Gynecol 2008; 112:1303.

7 R Kulier et al Medical methods for fi rst trimester abortion Co-chrane Database Syst Rev 2011; 11:CD002855.

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In Brief: Jadenu – A New Formulation of Deferasirox for

Iron Overload www.medicalletter.org/TML-article-1493f

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on Drugs and Therapeutics

IN BRIEF

Jadenu – A New Formulation of

Deferasirox for Iron Overload

The FDA has approved an oral tablet formulation of

deferasirox (Jadenu [ jade' new] – Novartis) for

once-daily treatment of chronic iron overload due to blood

transfusions (transfusional hemosiderosis) in patients ≥2

years old or chronic iron overload in patients ≥10 years old

with non-transfusion-dependent thalassemia syndromes

A once-daily, oral tablet for suspension formulation of

deferasirox (Exjade) was approved in 2005 for the same

indications 1 Jadenu and Exjade are the only once-daily

oral formulations for iron chelation available in the US

Table 1 Deferasirox Products

Available Usual

(Novartis) for oral suspension PO once/d 3-5

1 Dosage for transfusional iron overload Dosage adjustments are recommended

for hepatic or renal impairment.

2 Approximate WAC for 30 days' treatment at the lowest usual dosage for a

70-kg patient WAC = wholesaler acquisition cost or manufacturer’s published

price to wholesalers; WAC represents a published catalogue or list price

and may not represent an actual transactional price Source: AnalySource®

Monthly April 5, 2016 Reprinted with permission by First Databank, Inc All

rights reserved ©2016 www.fdbhealth.com/policies/drug-pricing-policy.

3 Jadenu dosages of 14, 21, and 28 mg/kg/day are equivalent to Exjade dosages

of 20, 30, and 40 mg/kg/day, respectively.

4 Recommended dosage for patients ≥2 years old.

5 Take on an empty stomach at least 30 minutes before food Must be dispersed

in liquid before administration.

6 Can be taken on an empty stomach or with a low-fat meal For patients who

have diffi culty swallowing tablets, it can be crushed and sprinkled on soft food,

such as yogurt or applesauce.

No new clinical trials were required for approval of Jadenu,

which was based on earlier clinical trials with Exjade

The most common adverse effects reported with use

of deferasirox in clinical trials were diarrhea, vomiting,

nausea, abdominal pain, rash, neutropenia, and increases

in serum creatinine Severe skin reactions, including

Stevens-Johnson syndrome and erythema multiforme,

have been reported rarely Hearing loss and ocular

disturbances, including cataracts, have been reported

with deferasirox; patients taking the drug should have

annual auditory and ophthalmic exams Gastrointestinal

ulceration and hemorrhage and renal and hepatic toxicity

have also occurred.

1 Deferasirox (Exjade): a new iron chelator Med Lett Drugs Ther

2006; 48:35.

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1 Discuss the new recommendations for use of metformin (Glucophage, and others) in patients with renal impairment.

2 Review the effi cacy and safety of cariprazine (Vraylar) for treatment of schizophrenia and bipolar I disorder.

3 Review the effi cacy and safety of the Maestro Rechargeable System for weight loss

4 Discuss the new recommendations for use of mifepristone (Mifeprex) for termination of pregnancy.

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Issue 1493 Questions

(Correspond to questions #81-90 in Comprehensive Exam #74, available July 2016)

6 Your colleague is considering using either cariprazine or aripiprazole for treatment of acute mania in a 24-year-old woman with bipolar l disorder She is concerned about side effects, especially weight gain You could tell her that:

a weight gain appears to be about the same with either aripiprazole or cariprazine

b cariprazine has been shown to be more effective than aripiprazole

c cariprazine is less likely than aripiprazole to cause diabetes

d all of the above

Maestro Rechargeable System for Weight Loss

7 The Maestro Rechargeable System reduces excess weight by:

a causing malabsorption

b increasing GI transit time

c reducing stomach capacity

d blocking vagus nerve signals between the stomach and the brain

8 In the ReCharge trial, the Maestro Rechargeable System reduced

excess weight at 12 months by about:

a 25%

b 35%

c 55%

d 65%

Mifepristone (Mifeprex) Label Changes

9 Under the new labeling, mifepristone and misoprostol can now

be used to terminate pregnancy for up to how many days after the fi rst day of the last menstrual period?

a 49

b 56

c 63

d 70

10 Which route of administration is now recommended for misoprostol?

a oral

b buccal

c vaginal

d all of the above

New Recommendations for Use of Metformin in Renal Impairment

1 You are deciding on initial therapy for a 45-year-old black

man with type 2 diabetes He weighs 225 lbs and has a serum

creatinine of 1.5 mg/dL and an eGFR of 57 mL/min/1.73 m 2 Which

of the following is true about the use of metformin in this patient?

a it is contraindicated because his serum creatinine is

≥1.5 mg/dL

b it is contraindicated because his eGFR is <60 mL/min/1.73 m 2

c he can take metformin, but the dosage should be reduced

d metformin is considered safe for use in this patient

2 The above patient is being scheduled for an iodinated contrast

imaging procedure Which of the following should you

recommend?

a continue metformin without interruption

b do not administer metformin until 48 hours after the

procedure is completed and eGFR is reassessed

c reduce the dose of metformin by 50% until 72 hours after

the procedure

d none of the above

Cariprazine (Vraylar) for Schizophrenia and Bipolar I Disorder

3 The only second-generation antipsychotic that is clearly more

effective than fi rst-generation antipsychotics for treatment of

schizophrenia is:

a cariprazine

b clozapine

c olanzapine

d aripiprazole

4 In clinical trials, cariprazine was more effective than:

a placebo

b aripiprazole

c risperidone

d all of the above

5 Compared to cariprazine, aripiprazole has:

a a longer record of effi cacy

b a longer record of safety

c generic availability

d all of the above

ACPE UPN: Per Issue Exam: 0379-0000-16-493-H01-P; Release: April 25, 2016, Expire: April 25, 2017 Comprehensive Exam 74: 0379-0000-16-074-H01-P; Release: July 2016, Expire: July 2017

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