Clinical Trial of Remdesivir to Treat COVID-19


NIH (NIAID) Clinical Trial of Remdesivir to Treat COVID-19 Begins

Randomized, controlled clinical trial to evaluate the safety and efficacy of the investigational antiviral REMDESIVIR in hospitalized adults diagnosed with COVID-19 

  • Remdesivir is an investigational broad-spectrum antiviral treatment with previous testing for treatment for Eboila, MERS and SARS
  • Enrolling hospitalized adults with COVID-19 in Nebraska, Remdesivir vs placebo
  • Participants must have laboratory-confirmed SARS-CoV-2 infection and evidence of lung involvement with need for supplemental oxygen, abnormal chest X-rays, requiring mechanical ventilation
  • Will compare (vs. placebo) participant outcomes on day 15  on seven-point scale (fully recovered – death)


Image credit: FDA and NIAID

News & Views: COVID-19 updates, Rare Disease efforts, Fraudulent DTC genetic tests & review, Stopping illicit medical products from India, Updated Insulin regulatory pathway, Directory for approved drugs (since 1985)


FDA’s Actions in Response to 2019 Novel Coronavirus at Home and Abroad

Active partner in Novel Coronavirus (COVID-19) response, working closely with  Department of Health and Human Services, as well as with international counterparts

  • Active Supply Chain Surveillance: Closely monitor domestic and global supply chain
  • Inspections and Monitoring Compliance of FDA Products Manufactured Overseas: Risk-based surveillance testing of imported products, including those from China
  • Safety of Consumer Products: Close monitoring of fraudulent products and false product claims related to COVID-19
  • Efforts to Diagnose, Treat and Prevent: Expedite development and availability of medical products needed to diagnose, treat and prevent disease
  • Next Steps: Collaborating with interagency partners, international partners, medical product developers and manufacturers to help advance response efforts combat this virus



Rare Disease Day 2020: FDA Continues Important Work on Treatments for Rare Diseases

Commemorating  Rare Disease Day by efforts to get treatments to patients with rare diseases across the thousands of identified rare diseases

  • Public meeting with stakeholders to address challenges and opportunities surrounding rare disease product development
  • New request for applications (RFA) for the Orphan Products Grants Program
  • Additional information on orphan “exclusivity protected uses.”
  • Enhanced rare disease patient website




Collaborative Review of Scientific Evidence to Support Associations Between Genetic Information and Specific Medications

Concerns with DTC genetic tests making claims about genetic test results to manage medication treatment that are NOT supported FDA-approved drug labeling or other scientific evidence

  • Steps to help ensure that claims are grounded in sound science to avoid inappropriate management of patients’ medications
  •  New web-based resource including table describing some of the gene-drug interactions for altered drug metabolism, differential therapeutic effects, differences in risks of adverse events
  • Continue to review various professional guidelines e.g. Clinical Pharmacogenetics Implementation Consortium
  • Continue participation in community-based collaborative approach for ongoing evaluation of the evolving science

Table of Pharmacogenetic AssociationsREAD


FDA Takes Action with Indian Government to Protect Consumers From Illicit Medical Products

Operation Broadsword targeted packages entering the U.S. through International Mail Facility from Jan. 28 through Jan. 30

  • Investigators from both governments examined > 800 shipments, ~50 different FDA-regulated products, many transshipped through third-party countries to conceal point of origin and avoid detection
  • Stopped approximately 500 shipments of illicit, and potentially dangerous, unapproved prescription drugs and combination medical devices from reaching American consumers
  • FDA provided consumer information :  BeSafeRx: Know Your Online Pharmacy.  and encouraged to report any unlawful sale of medical products online to FDA.



Smooth Regulatory Transition of Insulin and Other Biological Products  to Increase Patient Access and Potentially Lower Prices on Insulin

Steps to ensure a smooth transition to new regulatory pathway

  • Final Rule for Biosimilars Action Plan
  • Incorporates changes made by the Biologics Price Competition and Innovation Act of 2009 (BPCI Act) and the Further Consolidated Appropriations Act, 2020
  • Intended to balance innovation and competition and facilitate the development and approval of biosimilar and interchangeable product




Compilation of CDER New Molecular Entity (NME) Drug and New Biologic Approvals

New resource to assist external and agency researchers collecting historical information about FDA’s drug approvals 

  • Provide researchers with curated data regarding drug products approved by CDER since 1985 through 2019
  • Facilitate data accessibility, transparency, and accuracy when researchers seek information about an approved drug
  • Derived from FDA internal databases and document records, and reflects the state of each application at the time of initial regulatory approval
  • CDER will manage the compilation and will update it periodically with the latest drug approval data

Compilation of CDER NME and New Biologic Approvals 1985-2019

Compilation of CDER New Molecular Entity (NME) Drug and New Biologic Approvals – Data Dictionary

Image credit: FDA

Potential Nitrosamines in Medication


What to Know and Do About Possible Nitrosamines in Medication

Ongoing investigation of several potentially cancer-causing substances, called nitrosamines, recently found in blood pressure, heartburn, acid reflux medications

  •  Some drugs – including angiotensin II receptor blockers (ARBs), ranitidine, and nizatidine – have been recalled because of nitrosamine impurities
  • Patients need to talk to their health care professional, as risks of stopping medicine may outweigh the potential risk of exposure to nitrosamines
  • Nitrosamines not expected to cause harm when ingested at low levels


Image credit: FDA

Market Authorizations: 2019-nCoV diagnostic, ERVEBO Ebola vaccine, TEPEZZA for thyroid eye disease

Capture2019-nCoV Real-Time RT-PCR Diagnostic Panel


INTENDED USE: For the presumptive qualitative detection of nucleic acid from the 2019-nCoV in upper and lower respiratory specimens (such as nasopharyngeal or oropharyngeal swabs, sputum, lower respiratory tract aspirates, bronchoalveolar lavage, and nasopharyngeal wash/aspirate or nasal aspirate) collected from individuals who meet CDC criteria for 2019-nCoV testing. Testing is limited to qualified laboratories
designated by CDC and, in the United States, certified under the Clinical Laboratory Improvement Amendments of 1988 (CLIA), to perform high complexity tests.




  • Reverse transcriptase polymerase chain reaction (PCR) test that provides presumptive detection of 2019-nCoV from respiratory secretions, such as nasal or oral swabs
  • Positive test result indicates likely infection with 2019-nCoV and infected patients should work with their health care provider to manage their symptoms and determine how to best protect the people around them
  • Negative results do not preclude 2019-nCoV infection and should not be used as the sole basis for treatment or other patient management decisions; negative results must be combined with clinical observations, patient history and epidemiological information

REGULATORY PATHWAY: Emergency Use Authorization to address public health  emergency



ERVEBO ebola vaccine for intramuscular injection


INDICATION: Prevention of disease caused by Zaire ebolavirus in individuals 18
years of age and older

ADDRESSING UNMET NEED: First FDA-approved vaccine for the prevention of Ebola virus disease (EVD), caused by Zaire ebolavirus

MECHANISM OF ACTION: Immunization results in an immune response and protection from disease caused by Zaire ebolavirus


  • Guinea study, during 2014-2016 outbreak, n= 3,537 contacts and contacts of contacts with laboratory-confirmed EVD, “immediate” or 21-day “delayed” vaccination
  • ERVEBO 100% effective in preventing Ebola cases with symptom onset greater than 10 days after vaccination
  • No EVD cases in “immediate” cluster group vs 10 cases in 21-day “delayed” cluster group
  • Liberia study- n= 477, Sierra Leone study – n=500, Canada, Spain, US study – n=900
  • Antibody responses in Canada, Spain and U.S. study were similar to those among individuals in the studies conducted in Liberia and Sierra Leone.


  • Most commonly reported side effects were pain, swelling and redness at the injection site, as well as headache, fever, joint and muscle aches and fatigue.


  • Priority Review and Tropical Disease Priority Review Voucher, Breakthrough Therapy designation
  •  Because of public health importance, FDA worked closely with Merck and completed its BLA evaluation in less than six months



TEPEZZA (teprotumumab-trbw) injection, for intravenous use

Horizon Therapeutics

INDICATION: Treatment of Thyroid Eye Disease


  • First drug approved for the treatment of thyroid eye disease-
  • Rare condition where muscles and fatty tissues behind eye become inflamed, causing eyes to be pushed forward and bulge outwards (proptosis)

MECHANISM OF ACTION: Not been fully characterized- binds to IGF-1R and blocks  activation and signaling


  • Two studies, n=170 patients with active thyroid eye disease, TEPEZZA or placebo
  • Primary endpoint: >2 millimeter reduction in proptosis- 71% (Study 1) and 83% (Study 2) vs. 20% and 10%


  • Most common adverse reactions: Muscle spasm, nausea, alopecia, diarrhea, fatigue, hyperglycemia, hearing loss, dry skin, dysgeusia, headache
  • Should not be used if pregnant, counseling on pregnancy prevention during treatment and for 6 months following last dose


  • Priority Review, in addition to Fast Track and Breakthrough Therapy Designation 
  • Orphan Drug designation, with support from FDA Orphan Products Grants Program
  • Postmarketing commitments: Additional studies to characterize efficacy, safety, quality


Image credits: FDA, Merck, Horizon Therapeutics








News and Views: Coronavirus medical countermeasures, 2019 New Therapies, Gene Therapy innovation, Deter anti-competitive business for biologics, Heart Health education for women, HIV Drugs mobile database, Warning for nicotine toothpicks


Advance Development of Novel Coronavirus Medical Countermeasures (MCM)

FDA collaborating with interagency partners, product developers, international partners and global regulators to
  • Expedite development and availability of MCM to diagnose, treat, mitigate, prevent
  • Utilize pathways, including Emergency Use Authorization (EUA) to more rapidly make MCM available
  • Provide regulatory advice, guidance, and technical assistance to  sponsors developing investigational MCMs
  • Provide review and feedback on development proposals including design and set-up of clinical trials
  • Protect safety of nation’s blood supply and human cells, tissues,cellular/tissue-based products for transplantation
  • Enable access to investigational MCM through EUA or expanded access mechanisms
  • Protect consumers against fraudulent products by monitoring fraudulent products and false product claims and taking appropriate action

READ , 2019-nCOV website 


2019 CDER New Drug Approvals Report

Annual report entitled Advancing Health Through Innovation: New Drug Therapy Approvals

  • Variety of novel drugs for advancing patient care – never approved in US
  • Overview of other notable approvals — new uses of uses for approved drugs
  • Treat new population of patients, such as children
  • Innovative ways to enhance efficiency and expedite review and approval

Innovation and Access areas

  • Rare Diseases
  • Neurological and Psychiatric disorder
  • Infectious diseases
  • Heart, Lung, Circulatory, Endocrine diseases
  • Autoimmune conditions
  • Women’s and Men’s specific health issues
  • Cancers and blood disorders
  • Biosimilars



Strong Support of Innovation in Development of Gene Therapy Products

FDA efforts  to support innovators developing new gene therapy products, which insert new genetic material into a patient’s cells

  • 4 products approved
  • > 900 investigational new drug (IND) applications for ongoing clinical studies
  • will serve to improve therapeutic choices

Publication of guidances issued to provide recommendations for product developers



New Efforts to Further Deter Anti-Competitive Business Practices, Support Competitive Market for Biological Products to Help Americans

FDA and FTC to collaborate to curtail and discourage anti-competitive behavior to faciliate robust competition and bring down cost for biologics

  1. Promote greater competition in biologic markets
  2. Deter behavior that impedes access to samples needed for the development of biologics, including biosimilars
  3. Take appropriate action against false or misleading communications
  4. Review patent settlement agreements involving biologics, including biosimilars, for antitrust violations


CaptureHeart Health Education for Women

FDA Office of Women’s Heath (OWH) initiatives is support of American Heart Month
  • Heart health educational video ‘Getting a Beat : On what women know about heart health
  • New KNOWH the Difference initiative, which focuses on sharing important knowledge and news on women’s health (KNOWH)
  • OWH research and extramural funding opportunities

Mobile-Friendly Database with Information on Life-Saving HIV Drugs

Interactive database  on critical information about antiretrovirals (ARVs) eligible for purchase under the President’s Emergency Plan for AIDS Relief (PEPFAR) program

  • Empower public and health care providers by enhancing the amount and availability of information and data provided on each drug
  • Health care providers, consumers, procurer access to FDA-reviewed product labeling and essential scientific information for safe and effective use of drug
  • Information on pediatric drugs, where manufactured, shelf-life, storage requirements
  • Ability to export reports, collect metrics, readily access on mobile platforms


FDA Warns Maker of Nicotine-Containing Toothpicks of Several Violations, Including Illegal Sales 

  • Warning letter to Smart Toothpicks LLC,  Tempe, Arizona, for selling dissolvable tobacco products, including Peppermint Ice Nicotine Toothpicks
  • Three specific violations:
    • selling a tobacco product to a minor through the company’s website
    • selling unauthorized modified risk tobacco products
    • failing to include required nicotine warning statements on both packaging and advertising
  • Written response to this letter within 15 working days on corrective actions
    • discontinue violative labeling, advertising, sale, and/or distribution
    • plan for maintaining compliance with the FD&C Act

Action is part of the agency’s Youth Tobacco Prevention Plan, to prevent and reduce youth tobacco use 


Image credits: FDA