Top 10 Developments to Watch in 2020
EnvisionRx continuously monitors the drug pipeline. Our team evaluates changing treatment options in order to educate and inform our clients on the most impactful medications in development, new drugs recently approved, generics being launched and current medications receiving new indications.
- Aducanumab and the Advancement of Alzheimer's Disease Research
An estimated 5.8 million Americans suffer from Alzheimer's Disease (AD) and, as the population ages, that number is expected rapidly increase. However, there has not been a new drug approval for AD in the world since 2003. Aducanumab is a monoclonal antibody that selectively targets aggregated beta amyloid plaques in patients with Alzheimer’s disease, a mechanism of action that has been questioned due to multiple drug failures. This doubt initially included aducanumab, however, further analysis 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. Currently, there are approximately 26 AD drugs in phase III of the pipeline and a Chinese biotech company recently won conditional approval in China for an AD medication. Drugs for Alzheimer’s treatment and prevention are definitely on the 2020 watch list, as any approval could be a blockbuster.
- Bempedoic Acid's Potential Place in High Cholesterol Treatment
Bempedoic acid is a novel non-statin product that is being developed to reduce elevated LDL cholesterol alone or in combination with ezetimibe. While studies have shown a reduction in LDL cholesterol, there was no difference in the number of cardiovascular events. Based on the current data, bempedoic acid is unlikely to replace statin therapy and may not have widespread use, except as add-on therapy or in patients who cannot tolerate statin therapy.
- Cabenuva and its Role in the Treatment of HIV
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 of the drugs that compose the medication. Clinical trials showed the injectable to be non-inferior to current anti-retrovial therapies and that it was well tolerated. If approved, it could offer an alternative administration option for patients that require lifelong maintenance therapy for viral suppression of HIV-1.
- Ervebo's Impact on the International Prevention of Ebola
Ervebo 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. It has seen high success rates and was recently approved by the European Commission for use in the European Union. It is currently under priority review by the FDA.
- Obeticholic Acid and the Search for a Treatment for Non-Alcoholic Steatohepatitis
Non-alcoholic steatohepatitis (NASH) causes liver damage and is the second leading cause of liver transplant. It can be asymptomatic for many years and usually begins as non-alcoholic fatty liver disease (NAFLD), a condition that could affect nearly 50% of Americans. The current 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 trade name Ocaliva®. It is hypothesized that OCA may reduce fibrosis in certain patients with NASH. In clinical trials there were positive results in the reduction of fibrosis, however, the drug did not improve or worsen NASH. The race is on for a drug that may improve, resolve or prevent NASH itself.
- The Continued Development of Immunotherapy for Peanut Allergy Sufferers
In 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, which recently received a favorable vote from the FDA's Allergenic Products Advisory Committee (APAC) and is on target for FDA approval in early 2020. Viaskin Peanut, also reviewed in that issue, will be reviewed by the FDA in August of 2020 and three more products are in early research phases. Expect FDA approvals for these peanut allergy treatments to start occurring in 2020.
- Self-Administered Formulations being Developed for Current IV Medications
There are a number of treatments for various conditions that require patients to go to a healthcare provider for administration, which can be inconvenient. Because of this, recently, a number of pharmaceutical manufacturers have developed self-administration options. Two subcutaneous (SC) and/or self-administered asthma treatments were recently approved and SC formulations are currently being developed for ulcerative colitis and Crohn's disease and multiple myeloma. Subcutaneous and/or self-administered medications have the potential to switch patients from utilizing their medical benefit over to the prescription 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.
- Approval of Trikafta™ and the Potential Impact it Could Have on Cystic Fibrosis Therapy
Granted Priority Review, in addition to Fast Track and Breakthrough designations, Trikafta was approved by the FDA for the treatment of Cystic Fibrosis (CF) five months earlier than anticipated. It is the first triple combination therapy approved for the most common cystic fibrosis mutation, F508del, which is estimated to affect approximately 90% of the cystic fibrosis population. Trikafta’s drug combination has significant potential to shift current CF product utilization and offer a treatment option to CF patients that previously wasn't available.
- The First-of-its-Kind New Indication for Vascepa® for the Prevention of Cardiovascular Events
Vascepa, a medication containing eicosapentaenoic acid (EPA), which is derived from fish oil, is approved by the FDA for the treatment of severe hypertriglycermidemia. 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 (>/= 135 mg/dL) and other risk factors for cardiovascular disease (CVD). The medication received a favorable vote from the FDA Endocrinologic and Metabolic Drugs Advisory Committee for expanding the indication as an add-on to statins therapy for a reduction of the first occurrence of a major CV event. This indication has never been approved for any other non-statin lipid-lowering drug and, if approved, could change the standard of care for primary prevention of CV disease.
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.
- A New Technology for Gene Editing and How it Could Revolutionize Medical Treatment
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, as it has shown to be more accurate, efficient and cheaper than previous genome editing attempts. Clinical studies using CRISPR technology began on humans in the United States in 2019 and currently, there are 20 active clinical trials. 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. As research continues, CRIPSR-Cas9 technology could increase drug development, fueling the pipeline for years to come.
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