PERSPECTIVE THE ON RXPIPELINE - Visibly Different...make its way to market quickly. Currently, there are approximately 26 AD drugs in phase III of the pipeline, 17 of which are disease

1 | PERSPECTIVE ON THE RXPIPELINE • DECEMBER 2019

Understanding changes in the medication market and their impact.

EnvisionRx continuously monitors the drug pipeline. As treatment options change, we evaluate and share our perspective on the clinical benefits and impact in the market. Our Perspective on the Rx Pipeline reports provide ongoing insights from our team of clinical experts and considerations to protect and improve plan performance.

Top 10 Developments to Watch in 2020:} Aducanumab and the Advancement of Alzheimer's Disease Research

} Bempedoic Acid’s Potential Place in High Cholesterol Treatment

} Cabenuva and its Role in the Treatment of HIV

} Ervebo’s Impact on the International Prevention of Ebola

} Obeticholic Acid and the Search for a Treatment for Non-Alcoholic Steatohepatitis

} The Continued Development of Immunotherapy for Peanut Allergy Sufferers

} Self-Administered Formulations being Developed for Current IV Medications

} Approval of Trikafta™ and the Potential Impact it Could Have on Cystic Fibrosis Therapy

} The First-of-its-Kind New Indication for Vascepa® for the Prevention of Cardiovascular Events

} A New Technology for Gene Editing and How it Could Revolutionize Medical Treatment

PERSPECTIVE RXPIPELINEON THE


Clinical Pipeline

PIPELINE STAGE R & D FDA

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MarketOpen Source Alternative

An estimated 5.8 million Americans suffer from Alzheimer’s Disease (AD) and, as the population ages, that number is expected to rapidly increase.[1] This devastating condition rose from being ranked as the 12th most burdensome disease or injury in the United States in 1990 to the sixth in 2016 in terms of disability adjusted life years (DALYs). In 2016, Alzheimer’s disease was also the fourth highest disease or injury in terms of years of life lost (YLLs).[2,3] The condition also poses a financial burden, with Alzheimer’s and other dementia conditions accounting for an estimated $290 billion in healthcare, long-term care and hospice costs in 2019.[1]

Aducanumab is a monoclonal antibody that selectively targets aggregated beta amyloid plaques in patients with Alzheimer’s disease.[4] While this has been a mechanism of action used in AD research over the last couple of decades, due to multiple failures of drugs using this method to reduce AD decline, some experts have questioned if this is the right mechanism of action to pursue. This doubt initially included aducanumab, however, further analysis of aducanumab’s EMERGE trial showed that a subset of the population treated with the two higher doses of the drug appeared to have a significant reduction in clinical decline. This was as measured by the pre-specified secondary endpoints using the Mini-Mental State Examination (MMSE), the AD Assessment Scale-Cognitive Subscale 13 Items (ADAS-COG 13) and the AD Cooperative Study-Activities of Daily Living Inventory Mild Cognitive Impairment Version (ADCS-ADL-MCI) scales. The results were as follows:[5]

• MMSE - 15% versus placebo, P=0.06• ADAS-COG 13 - 27% versus placebo, P=0.01• ADCS-ADL-MCI - 40% versus placebo, P=0.001• Amyloid plaques were also reduced

While it is unknown if the FDA will find the information for aducanumab favorable or sufficient as presented by Biogen and Eisai, drugs for Alzheimer’s treatment and prevention are definitely a 2020 to watch, as any approval could be a blockbuster. If successful clinical data is supplied to the FDA, it will likely become a priority review and make its way to market quickly.

Currently, there are approximately 26 AD drugs in phase III of the pipeline, 17 of which are disease modifying and six for prevention.[6] Of the 26 in phase III development, 22 of these drugs could reach the pharmacy market in the next five years.

Additionally, a timely international approval for AD research occurred, where a Chinese biotech company won conditional approval (with the requirement for more data to support safety and efficacy) for oligomannate (GV-971), an algae-derived oligosaccharide component that alters AD patients’ gut microbiomes. This was the first AD drug approval in the world since 2003.[7]

aducanumab BIIB037Manufacturer: Biogen/Eisai Indication/Use: Alzheimer’s diseaseDosage Form: IntravenousPipeline Stage: Phase III, pending FDA filling

BLA - Biologics License Application NDA - New Drug Application PDUFA - Prescription Drug User Fee ActGlossary of Terms


Bempedoic acid is a novel non-statin product that is being developed to reduce elevated LDL cholesterol. Esperion has submitted a new drug application (NDA) to the FDA for bempedoic acid alone and in combination with ezetimibe.[8] In clinical trials, bempedoic acid reduced LDL cholesterol by 17.4% compared to placebo.[9] The combination product of bempedoic acid and ezetimibe reduced LDL by 38%.[10] High-intensity statins (i.e., atovastatin and rosuvastatin) reduce LDL cholesterol by approximately 40-50% and reduce the risk of coronary heart disease with multiple risk factors.[11]

While bempodoic acid alone reduced LDL cholesterol, there was no difference in the number of heart attacks and strokes over one year when compared to placebo.[9]

Based on the current data, bempedoic acid is unlikely to replace statin therapy and may not have widespread use. It will be used as add-on therapy or in patients who cannot tolerate statin therapy. For now, statin therapy is the most effective treatment for cholesterol reduction and risk reduction of myocardial infarction, stroke and arterial revascularization procedures in patients without clinically evident cardiovascular disease.

bempedoic acid ETC-1002Manufacturer: EsperionIndication/Use: HypercholesterolemiaDosage Form: OralPipeline Stage: PDUFA 2/21/20

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Cabenuva, if approved, would be the first-ever long-acting injectable anti-retroviral for those that are virologically suppressed (HIV-1 RNA less than 50 copies per mL) and have no known or suspected resistance to either cabotegravir (CAB) or rilpivirine (RPV).[12] This two-drug regimen contains a previously approved non-nucleoside reverse transcriptase inhibitor, RBV (marketed as Edurant®), and an investigational integrase inhibitor, CAB. These products will each be injected monthly for HIV-1 maintenance therapy. Prior to starting this long-acting injectable, patients would take an oral-lead of both medications to assess tolerability of the products prior to injection.[13]

In clinical trials the combination long-acting injectable was non-inferior to current anti-retroviral therapy and patients maintained virologic suppression. Patients tolerated the injection with minimal discontinuation rates. This product would be administered by a healthcare provider. If approved, this product would offer an alternative administration option for patients that require lifelong maintenance therapy for viral suppression of HIV-1.[13]

Cabenuva cabotegravir, rilpivirine Manufacturer: Viiv healthcareIndication/Use: HIV-1Dosage Form: InjectablePipeline Stage: PDUFA 12/29/19


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Ervebo® V920Manufacturer: Merck & Co/NewLink GeneticsIndication/Use: Prevention of the onset of Ebola ZaireDosage Form: VaccinePipeline Stage: BLA 3/14/20

Ebola is a deadly virus with a 50% fatality rate in humans.[14] V920 is a recombinant, replication-component Ebola vaccine that was originally engineered by the Public Health Agency of Canada in an effort for global health and prevention of the onset of Ebola in the Democratic Republic of Congo and other endemic areas. As of August 8, 2018, approximately 164,000 patients had been vaccinated with V920, with an estimated efficacy rate of 97.5% for prevention of disease transmission. From a global health perspective this vaccine can be used to create “vaccination rings” around individuals at risk for contracting Ebola. On November 11, 2019, the European Commission announced the granting of marketing authorization for V920, which would make it available in 31 European countries.[14-16] The FDA has accepted the Biologics License Application (BLA) for the vaccine and granted it priority review.

Obeticholic Acid OCAManufacturer: Intercept PharmaceuticalsIndication/Use: Non-alcoholic steatohepatitis (NASH)Dosage Form: OralPipeline Stage: Phase III, PDUFA March 2020

Non-alcoholic steatohepatitis (NASH) causes liver swelling and damage and is the second leading cause of liver transplant. It can be asymptomatic for many years and is only diagnosed with an invasive liver biopsy conducted and interpreted by a specialist. NASH usually begins as non-alcoholic fatty liver disease (NAFLD), a condition that could affect nearly 50% of Americans.[17] Approximately 5% to 20% of NAFLD patients progress to NASH, although significant weight loss (5% to 10%) can prevent this progression.[17] The search is still on for the first efficacious NASH-indicated FDA-approved medication. Currently, the treatment goal is to prevent fibrosis, as it may also prevent cirrhosis of the liver.

Obeticholic acid (OCA) is a synthetic version of bile acid that is currently FDA-approved for the treatment of primary biliary cholangitis (PBC) under the name Ocaliva®. OCA may reduce fibrosis in certain patients with NASH. The REGENERATE trial showed positive results in one out of two primary outcomes using the 25 mg dose of OCA, with statistically significant fibrosis reduction (greater than 1 fibrosis stage). It is notable that NASH itself was not shown to improve, but did not worsen while on OCA.

Many other NASH pipeline drugs have failed to produce robust positive results. The race is on for a drug that may improve, resolve or prevent NASH itself. Notably, the Institute for Clinical and Economic Review (ICER) is expected to publish an evaluation of NASH, and specifically obeticholic acid, in May 2020.


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Peanut Allergy UpdateIn a previous edition of Perspective on the Rx Pipeline, we discussed the development of immunotherapies for patients with peanut allergies. Immunotherapies introduce patients to trace amounts of a specific allergen, slowly increasing the dosage over time, with the hope that the patient will become desensitized to the allergen, lessening risks and healthcare costs. In that July edition, A Sensitive Alternative for Peanut Allergy Sufferers, we reviewed AR101, now known as Palforzia, an oral biologic immunotherapy in development that would reduce the risk of anaphylaxis after accidental peanut exposure in patients age four to 17. In September 2019, the FDA’s Allergenic Products Advisory Committee (APAC) voted to support Palforzia (7 to 2 on efficacy data and 8 to 1 on safety data), putting the drug on target for FDA approval in early 2020.[19]

A transdermal patch reviewed in A Sensitive Alternative for Peanut Allergy Sufferers, Viaskin Peanut, is following close behind and will be reviewed by the FDA in August of 2020. Three more products are also in early research phases:

• ANB020 – An antibody product that inhibits interleukin-33 in adult patients[20] • CA002 – Another immunotherapy oral product[21]

• PRT100 – A probiotic to be used with oral immunotherapy that may demonstrate immune modulator effects[22]

Expect FDA approvals for these peanut allergy treatments to start occurring in 2020.

Drug Name Manufacturer Indication/Use Dosage Form Pipeline Stage

Palforzia™ Aimmune Immunotherapy for peanut allergy Oral PDUFA January 2010

Viaskin Peanut DBV Technologies Immunotherapy for peanut allergy Patch PDUFA August 2020

ANB020 AnaptysBio Immunomodulatory therapy for peanut allergy Injectable Phase III TBD

CA002 Camallergy Immunotherapy for peanut allergy Oral Phase II TBD

PRT100 Prota Therapeutics Immunomodulatory probiotic for peanut allergy Oral TBD

https://visiblydifferent.envisionrx.com/blog/perspective-on-the-rx-pipeline-peanut-allergies

https://visiblydifferent.envisionrx.com/blog/perspective-on-the-rx-pipeline-peanut-allergies


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Subcutaneous Formulations of IV MedicationsThere are a number of treatments for various conditions that require patients to go to a healthcare provider for administration. Some of these are intravenous infusions, which can take a substantial amount of time. This can be an inconvenience for patients. Because of that, recently, a number of pharmaceutical manufacturers have developed self-administration options, including Fasenra for the treatment of severe uncontrolled asthma. Available since 2017, Fasenra previously required in-office administration by a healthcare professional. The new auto-injector pen, approved in October 2019, now allows for a patient or caregiver to administer an every-four-week dose of Fasenra at home.[23] Nucala, another in-office treatment for asthma, also had a self-administrated subcutaneous (SC) product approved in June 2019.

An intravenous (IV) treatment for adults with moderate to severe ulcerative colitis, Entyvio, is undergoing testing for an SC dosage form. The manufacturer, Takeda, conducted the VISIBLE 1 study to examine the efficacy and safety of Entyvio SC with 383 adult patients with moderately to severely active ulcerative colitis. Patients were given the IV induction and then 216 responders entered the maintenance phase where they received either IV, SC or placebo. The results found 46.2% of those on the SC formulation were in clinical remission at 52 weeks versus 14.3% remission in patients taking placebo (p<0. 001). With the IV drug, 42.6% were also in remission.[24] Entyvio SC was dosed every two weeks, where the current infusion is every 8 weeks.

Lastly, Janssen’s submitted for FDA approval of Darzalex SC, an oncology medication for multiple myeloma, with data from the phase III COLUMBIA study showing non-inferiority of the SC formulation to the IV formulation.[25]

Subcutaneous and/or self-administered medications have the potential to switch patients from utilizing their medical benefit over to the prescription benefit. For oncology medication, we may see patients switch to SC formulations over IV due to ease or time of administration, but remain under the medical benefit. Expect to see a continued trend of manufacturers of IV formulations looking at patient convenience and administration to gain approval of new formulations for their current drugs.

Drug Name Manufacturer Indication/Use Dosage Form Pipeline Stage

Fasenra® Pen (benralizumab) AstraZeneca Maintenance treatment

of severe asthma Subcutaneous Approved

Nucala® SC (mepolizumab) GSK

Maintenance treatment of severe asthma and eosinophilic granulomatosis with polyangiitis (EGPA)

Subcutaneous Approved

Entyvio® SC (vedolizumab) Takeda Ulcerative colitis and

Crohn’s disease Subcutaneous PDUFA March 2020

Darzalex® SC (daratumumab) Janssen Multiple myeloma Subcutaneous 3Q2020


Drug Approval

Trikafta™ ivacaftor, tezacaftor, elexacaftorManufacturer: Vertex Pharmaceuticals, Inc.Indication/Use: Cystic Fibrosis Dosage Form: OralTraditional or Specialty: Traditional

Cystic Fibrosis (CF) is caused by a gene mutation and is a rare, progressive, life-threatening disease. Patients experience formation of thick mucus build up in their lungs and digestive track, putting them at risk for severe respiratory infections and digestive problems.[26]

Granted Priority Review, in addition to Fast Track and Breakthrough designations, Trikafta was approved by the FDA for the treatment of CF five months earlier than anticipated. It is the first triple combination therapy approved for the most common cystic fibrosis mutation, F508del. It is estimated that approximately 90% (27,000 individuals) of the cystic fibrosis population has at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The efficacy of Trikafta was demonstrated in patients with CF aged 12 years and older by evaluating the percent predicted expiratory volume in one second (ppFEV1), which is an established marker of disease progression. Both clinical trials showed an improvement compared to baseline of ppFEV1. The safety profile of Trikafta is similar to other CFTR modulators. Trikafta’s drug combination has significant potential to shift current CF product utilization and offer a treatment to CF patients that previously did not have a CFTR therapy option.[26, 27]


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Vascepa® icosapent ethylManufacturer: Amarin Pharma, Inc.Indication/Use: Diet adjunct to reduce triglyceride (TG) levels in adult patients with severe (> 500mg/dL) hypertriglyceridemia Dosage Form: Oral Traditional or Specialty: Traditional Date of Original Approval: July 26, 2012

Vascepa, a medication containing eicosapentaenoic acid (EPA), which is derived from fish oil, is approved by the FDA for the treatment of severe hypertriglycermidemia.[28] Amarin Pharma recently sought an expanded indication for Vascepa to be used to prevent the first major cardiovascular (CV) event in patients with elevated triglycerides (TG), defined as TG > 135mg/dL, and other risk factors for cardiovascular disease (CVD).

On November 14, 2019, the FDA Endocrinologic and Metabolic Drugs Advisory Committee convened to review the data in the Reduction of Cardiovascular Events with EPA-Intervention Trial (REDUCE-IT) trial, which showed a relative risk reduction of 25% in the first occurrence of a major CV event with Vascepa.[29] The advisory committee voted 16-0 in favor of expanding the indication of Vascepa as an add-on to statins therapy for a reduction of the first occurrence of a major CV event. This labeling update is anticipated to be approved December 28, 2019, and could have a major impact, as the proposed indication has never been approved for any other lipid-altering drug and could change the standard of care for primary prevention of CV disease.[30]

Please note, prior to publication, on December 13, 2019, FDA granted Vascepa an expanded indication approval of use as an adjunct to maximally tolerated statin therapy to reduce the risk of myocardial infarction, stroke, coronary revascularization, and unstable angina requiring hospitalization in adult patients with elevated triglycerides (> 150mg/dL) and established cardiovascular disease OR diabetes mellitus and two or more additional risk factors for cardiovascular disease.


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Gene or genome editing is a technology that scientists use to alter an organism’s DNA for the treatment of certain conditions. There are several approaches for gene editing. A new technology, known as CRISPR, has the potential to be revolutionary. It has shown to be more accurate, efficient and cheaper than previous genome editing attempts. With CRISPR technology, a piece of RNA with a ‘guide’ is directed to target a specific segment in the DNA genome, then the Cas9 enzyme cuts the targeted location, allowing for replacement or repair of the DNA sequence.[31] The hope is that results would be substantial or even curative from CRISPR treatment.

While it is unknown if the technology will lead to treatments under the medical or prescription benefit, the FDA does consider CRISPR-Cas9 technology gene therapy, when done in humans, to be a drug and FDA regulated under the biologics license application.[32] Clinical studies using CRISPR technology began on humans in the United States in 2019 and currently, there are 20 active clinical trials.[33] Some conditions CRISPR is being studied to treat include cancer, sickle cell disease, beta thalassemia and leber congenital amarousis.

CTX001, a drug by CRISPR Therapeutics that uses the technology for the treatment of transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD), received Fast Track designation by the FDA in April 2019. Recently, CRISPR Therapeutics reported that after four months of therapy with CTX001, TDT patients were transfusion independent and had 99.8% F-cells (the number of red blood cells expressing the engineered form of hemoglobin). The SCD patient was free of vaso-occulsive crises at four months and had 94.7% F-cells.[34] In 2020, full results may start to form and we may learn more about the role of CRISPR technology in medical treatment.

Beyond medical treatment, CRISPR technology may also be used to identify and validate molecules that cause disease, identifying drug targets, which could have an important impact on drug discovery.[35] As research continues, CRIPSR-Cas9 technology could increase drug development, fueling the pipeline for years to come.

CRISPR-Cas9 Manufacturer: TBD, CRISPR Therapeutics Indication/Use: Drug development and treatmentDosage Form: TBDPipeline Stage: Early research


[1] 2019 ALZHEIMER’S DISEASE FACTS AND FIGURES. (2019). Retrieved 3 December 2019, from https://www.alz.org/media/documents/alzheimers-facts-and-figures-2019-r.pdf

[2] Reitz C, Mayeux R (2014). Alzheimer disease: epidemiology, diagnostic criteria, risk factors and biomarkers. Biochem Pharmacology; 88(4), 640-51. doi: 10.1016/j.bcp.2013.12.024. Epub 2014 Jan 4.

[3] US Burden of Disease Collaborators, Mokdad, AH, Ballestros K, et al (2018). The State of US Health, 1990-2016: Burden of Diseases, Injuries, and Risk Factors among US States. Journal of American Medical Association, 10;319(14), 1444-1472. doi: 10.1001/jama.2018.0158.

[4] Sevigny J, e. (2016). The antibody aducanumab reduces Aβ plaques in Alzheimer’s disease. - PubMed - NCBI. Nature, 537(7618), 50-6. doi: 10.1038/nature19323.o Retrieved 3 December 2019, from https://www.ncbi.nlm.nih.gov/pubmed/27582220

[5] Biogen Plans Regulatory Filing for Aducanumab in Alzheimer’s Disease Based on New Analysis of Larger Dataset from Phase 3 Studies | Biogen. (2019). Retrieved 3 December 2019, from https://investors.biogen.com/news-releases/news-release-details/biogen-plans-regulatory-filing-aducanumab-alzheimers-disease

[6] Morgan ,D., Holzapfel D, Rochelle P, Paskus J. The Current State of Alzheimer’s Drug Development. (2019). Retrieved 3 December 2019, from https://www.usagainstalzheimers.org/sites/default/files/2019-07/RAAPages_FINAL.pdf

[7] Wang, X., Sun, G., Feng, T. et al (2019). Sodium oligomannate therapeutically remodels gut microbiota and suppresses gut bacterial amino acids-shaped neuroinflammation to inhibit Alzheimer’s disease progression. Cell Research, 9, 787–803, doi:10.1038/s41422-019-0216-x . Retrieved 3 December 2019, from https://www.nature.com/articles/s41422-019-0216-x#Abs1

[8] ESPERION | BA/Ezetimibe Combination Tablet. (2019). Retrieved 25 November 2019, from https://www.esperion.com/pipeline/bempedoic-acid-ezetimibe-combo-tablet/

[9] Goldberg, A., Leiter, L., Stroes, E., Baum, S., Hanselman, J., & Bloedon, L. et al. (2019). Effect of Bempedoic Acid vs Placebo Added to Maximally Tolerated Statins on Low-Density Lipoprotein Cholesterol in Patients at High Risk for Cardiovascular Disease. JAMA, 322(18), 1780. doi: 10.1001/jama.2019.16585

[10] Ballantyne, C., Laufs, U., Ray, K., Leiter, L., Bays, H., & Goldberg, A. et al. (2019). Bempedoic acid plus ezetimibe fixed-dose combination in patients with hypercholesterolemia and high CVD risk treated with maximally tolerated statin therapy. European Journal Of Preventive Cardiology, 204748731986467. doi: 10.1177/2047487319864671

[11] Stone, N., Robinson, J., Lichtenstein, A., Bairey Merz, C., Blum, C., & Eckel, R., et al (2013). 2013 ACC/AHA Guideline on the Treatment of Blood Cholesterol to Reduce Atherosclerotic Cardiovascular Risk in Adults. Circulation, 129(25 suppl2), S-S45 doi: 10.1161/01 cir. 0000437738.63853.7a

[12] Cabenuva. Accessed on 5 December 2019 from https://www.cabenuvahcp.com/?cc=v%3Apsea%3Acbh%3A156792&gclid=CKuyr72vn-YCFaOnxQId4HoBFA

[13] ViiV HEALTHCARE SUBMITS NEW DRUG APPLICATION TO US FDA FOR THE FIRST MONTHLY, INJECTABLE, TWO-DRUG REGIMEN OF CABOTEGRAVIR AND RILPIVIRINE FOR TREATMENT OF HIV. Accessed on 5 December 2019 from https://viivhealthcare.com/en-gb/media/press-releases/2019/april/viiv-healthcare-submits-new-drug-application-to-us-fda-for-the-first-monthly-injectable-two-drug-regimen-of-cabotegravir-and-rilpivirine-for-treatment-of-hiv/

[14] Ebola Virus. Accessed 25 November 2019 fom https://www.afro.who.int/health-topics/ebola-virus-disease

[15] Ebola vaccinations in the Democratic Republic of the Congo – In the community, by the community Accessed 25 November 2019 from https://www.afro.who.int/news/ebola-vaccinations-democratic-republic-congo-community-community

[16] Ebola Vaccine. Accessed 25 November 2019 from https://www.precisionvaccinations.com/vaccines/ervebo-ebola-vaccine

[17] Rau M, Schilling, A, Meertens J (2016). et al Progression from Nonalcoholic Fatty Liver to Nonalcoholic Steatohepatitis Is Marked by a Higher Frequency of Th17 Cells in the Liver and an Increased Th17/Resting Regulatory T Cell Ratio in Peripheral Blood and in the Liver The Journal of Immunology, 196 (1) 97-105; DOI: https://doi.org/10.4049/jimmunol.1501175 https://www.jimmunol.org/content/196/1/97

[18] Weib J, Rau M, Geier A (2014). Non-alcoholic fatty liver disease: epidemiology, clinical course, investigation, and treatment. Deutsches Arzteblatt International., 111(26):447-52. doi: 10.3238/arztebl.2014.0447. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4101528/

[19] FDA Allergenic Products Advisory Committee Votes to Support the Use of Aimmune’s PALFORZIA™ (AR101) for Peanut Allergy | Aimmune Therapeutics. (2019). Retrieved 3 December 2019, from http://ir.aimmune.com/news-releases/news-release-details/fda-allergenic-products-advisory-committee-votes-support-use

[20] AnaptysBio Announces Positive Top-Line Proof-of-Concept Data For ANB020 In Moderate-to-Severe Baseline Adult Peanut Allergy Patients | AnaptysBio Inc. (2019). Retrieved 3 December 2019, from https://ir.anaptysbio.com/news-releases/news-release-details/anaptysbio-announces-positive-top-line-proof-concept-data-anb020

[21] Clinical trials — Camallergy. (2019). Retrieved 3 December 2019, from http://www.camallergy.com/clinical-trials

[22] PRT100 Mechanism of Action | Prota Therapeutics. (2019). Retrieved 3 December 2019, from https://protatherapeutics.com/index.php/aa/

[23] Fasenra approved in the US for self-administration in a new pre-filled auto-injector (2019). Retrieved 3 December 2019, from https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2019/fasenra-approved-in-the-us-for-self-administration-in-a-new-pre-filled-auto-injector-the-fasenra-pen-04102019.html

[24] Sandborn, W., Baert, F., Danese, S., Krznari, Z., D’Haens, G., & Kobayashi, T. et al. (2019). OTH-12 Efficacy and safety of vedolizumab subcutaneous formulation for ulcerative colitis: results of the visible trial. Retrieved 3 December 2019, from https://gut.bmj.com/content/68/Suppl_2/A60.1

[25] Janssen Submits Application to U.S. FDA Seeking Approval of New DARZALEX® (daratumumab) Subcutaneous Formulation | Johnson & Johnson. (2019). Retrieved 3 December 2019, from https://www.jnj.com/janssen-submits-application-to-u-s-fda-seeking-approval-of-new-darzalex-daratumumab-subcutaneous-formulation

[26] FDA approves new breakthrough therapy for cystic fibrosis. (2019). Retrieved 3 December 2019, from https://www.fda.gov/news-events/press-announcements/fda-approves-new-breakthrough-therapy-cystic-fibrosis

[27] Trikafta (elexacaftor/tezacaftor/ivacaftor) [prescribing information]. Boston, MA: Vertex Pharmaceuticals

[28] Vascepa (icosapent ethyl) [prescribing information]. Bedminster, NJ: Amarin Pharma Inc; February 2017.

[29] Bhatt, D., Steg, P., Miller, M., Brinton, E., Jacobson, T., & Ketchum, S. et al. (2019). Cardiovascular Risk Reduction with Icosapent Ethyl for Hypertriglyceridemia. New England Journal Of Medicine, 380(1), 11-22. doi: 10.1056/nejmoa1812792

[30] November 14, 2019: Meeting of the Endocrinologic and Metabolic Drugs Advisory Committee Meeting Announcement (2019). Retrieved 2 December 2019, from https://www.fda.gov/advisory-committees/november-14-2019-meeting-endocrinologic-and-metabolic-drugs-advisory-committee-meeting-announcement#event-materials

Sources


Sources Continued

Clinical efficacy and safety, balanced with a drug’s value, are always at the forefront in the EnvisionRx formulary decisions and pipeline planning. The rationale for those decisions may go beyond the use of the FDA’s labeled indication. Our clinical reviews may utilize, but are not limited to, recognized consensus guidelines, the Institute for Clinical and Economic Review (ICER), and compendium such as the National Comprehensive Cancer Network (NCCN Guidelines®) and DRUGDEX®. EnvisionRx monitors FDA updates and safety announcements daily, as well as follows guidance from the Center of Disease Control and Prevention (CDC) and the US Preventive Service Task Force (USPSTF®).

[31] What are genome editing and CRISPR-Cas9 (2019). U.S. National Library of Medicine. Retrieved 3 December 2019, from https://ghr.nlm.nih.gov/primer/genomicresearch/genomeediting

[32] Information about Self-Administration of Gene Therapy (2019). U.S. Food and Drug Administration. Retrieved December 2019, https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/information-about-self-administration-gene-therapy

[33] CRISPR (search term). Clinicaltrials.gov. Retrieved 3 December 2019, https://clinicaltrials.gov/ct2/results?cond=&term=CRISPR+&cntry=&state=&city=&dist=

[34] CRISPR Therapeutics, Vertex Report First Data from Trials of Gene-Editing Treatment CTX001 (2019). Retrieved 3 December 2019, https://www.genengnews.com/news/crispr-therapeutics-vertex-report-first-data-from-trials-of-gene-editing-treatment-ctx001/

[35] Fellmann C, Gowen BG, Lin PC, Doudna JA, Corn JE. Cornerstones of CRISPR-Cas in drug discovery and therapy. Nat Rev Drug Discov. 2017 Feb;16(2):89-100. doi: 10.1038/nrd.2016.238. Epub 2016 Dec 23.

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The Envision Clinical Steering Committee brings together leaders from across our national pharmacy care company to monitor the drug landscape, provide recommendations on how to address changes, and to ensure our clients and patients are prepared—in advance.

With any new development, we partner with our Pharmacy & Therapeutics (P&T) Committee and consult with our best-in-class specialty pharmacy, to provide a balanced perspective on the clinical effectiveness of all available options, the cost impact to our plan sponsors and patients, and the impact on the overall patient experience.

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Documents

PERSPECTIVE THE ON RXPIPELINE - Visibly Different...make its way to market quickly. Currently, there are approximately 26 AD drugs in phase III of the pipeline, 17 of which are disease