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Here are the top drugs expected to be approved this year.
According to the FDA, 48 drugs have been approved in 2019, bringing more treatment options to the market for patients.1 Vizient, a health care performance improvement company, released its 2019 Drug Price Forecast for health systems, with an expected 4.57% increase for pharmaceutical purchases made from January 1, 2020 to December 31, 2020.2
Here are drugs in the pipeline expected to be approved in 2020 and their financial impact.
Pipeline drugs for 2020
It can sometimes be difficult to predict which medications will be approved, as there can be drug safety issues that the FDA needs more clarification on and further studies may be warranted, according to Erin Fox, PharmD, BCPS, FASHP, senior director, Drug Information and Support Services, University of Utah Health, Salt Lake City. Fox, who provides budget forecasting in her clinical practice setting, discussed how the budget is generally set after drugs receive FDA approval.
According to Fox, the pharmacy and therapeutics committee reviews the medications, and drug monographs are created to determine whether the new drugs will be added to the hospital formulary.
The FDA approved Eli Lilly’s Reyvow (lasmiditan) for acute treatment of migraine with or without aura, and it is expected to be available in January 2020. Reyvow is unique in that it is the first FDA-approved medication in a new class of acute migraine treatments known as serotonin (5-HT)1F receptor agonists.3
Adakveo (crizanlizumab-tmca) received FDA approval on November 15, 2019, which is 2 months ahead of schedule. Adakveo is manufactured by Novartis and is the first targeted therapy to treat patients with sickle cell pain. Additionally, Adakveo received orphan drug designation, which provides incentives to the manufacturer.4 Alder Biopharmaceuticals submitted the Biologics License Application (BLA) for eptinezumab for migraine prevention, and the expected approval date is February 21, 2020. Eptinezumab is a monoclonal antibody that has demonstrated positive results for migraine prevention in the PROMISE 1 and PROMISE 2 phase 3 studies.5
Seattle Genetics and Astellas have submitted the BLA for enfortumab vedotin for advanced or metastatic bladder cancer, and the target approval date is March 15, 2020. Enfortumab vedotin is considered a novel investigational antibody-drug conjugate that targets Nectin-4, which is a protein that is prevalent in bladder cancer. The BLA submission was based on positive results from the first cohort of patients in the EV-201 pivotal phase 2 clinical trial.6
Merck submitted the BLA for the Ebola vaccine (V920), which was granted priority review with an expected approval date of March 14, 2020.7 Biohaven Pharmaceuticals is studying rimegepant for acute treatment and prevention of migraine. Rimegepant is a calcitonin gene-related peptide receptor antagonist with a long half-life and good oral bioavailability, and the New Drug Application (NDA) has been submitted to the FDA with an expected approval of early 2020. Data presented at the International Headache Conference demonstrated that rimegepant decreased disability by about 41% and reduced lost productivity time by approximately 50% over a 1-year treatment period.8
Alnylam Pharmaceutical’s Givlaari (givosiran) was approved November 20, 2019, earlier than the expected date of February 4, 2020, for the treatment of acute hepatic porphyria, a rare genetic disease that can resemble other conditions and affect quality of life. Givlaari is the first drug approved for this rare disease, and it comes with a high wholesale price of $575,000. Alnylam plans to offer patient assistance and work with insurance companies to help provide coverage.
The ENVISION phase 3 study demonstrated that Givlaari improved quality of life measures compared with placebo.9 Epizyme’s tazemetostat was granted priority review for patients with epithelioid sarcoma who cannot receive curative surgery, and approval is expected January 2020.10 Intercept has submitted an NDA for obeticholic acid for patients with fibrosis due to nonalcoholic steatohepatitis (NASH). Approval is expected in 2020 based on positive results from the REGENERATE phase 3 study showing obeticholic acid improved liver fibrosis without worsening NASH at 18 months.11
Correction: In the corresponding table, Biohaven’s investigational migraine compound rimegepant is listed as expecting FDA approval for the acute treatment and prevention of migraine. This is to clarify that the PDUFA date for early 2020 is for the acute indication only, not prevention.
1. Novel Drug Approvals for 2019. FDA’s website. https://www.fda.gov/ drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2019.
2. Vizient Drug Price Forecast Projects 4.57 Percent Increase in Hospital Drug Spending for 2020 [news release]. Vizient’s website. Irving, Texas. https://newsroom.vizientinc.com/press-release/clevel-leader/vizient-drug-price-forecast-projects-457-percent-increase-hospital-drug.
3. FDA approves new treatment for patients with migraine [news release]. FDA’s website. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-patients-migraine.
4. FDA approves first targeted therapy to treat patients with painful complication of sickle cell disease [news release]. FDA’s website. https://www.fda.gov/news-events/press-announcements/ fda-approves-first-targeted-therapy-treat-patients-painful-complication-sickle-cell-disease
5. Alder Biopharmaceuticals Submits Biologics License Application to the US Food and Drug Administration for Eptinezumab [news release]. Alder’s website. https://investor.alderbio.com/ news-releases/news-release-details/alder-biopharmaceuticalsr-submits-biologics-license-application
6. Seattle Genetics and Astellas Announce US FDA Grants Priority Review for Enfortumab Vedotin Biologics License Application in Locally Advanced or Metastatic Urothelial Cancer [news release]. Astellas’ website. https://www.astellas.com/en/news/15091
7. FDA Accepts Merck’s Biologics License Application (BLA) and Grants Priority Review for V920, the Company’s Investigational Vaccine for Ebola Zaire Virus [news release]. https://investors.merck.com/news/ press-release-details/2019/FDA-Accepts-Mercks-Biologics-License-Application-BLA-and-Grants-Priority-Review-for-V920-the-Companys-Investigational-Vaccine-for-Ebola-Zaire-Virus/default.aspx
8. Rimegepant. Biohaven Pharmaceuticals’ website. https://www. biohavenpharma.com/science-pipeline/cgrp/rimegepant
9. Biohaven Presents Data Demonstrating Reduction in Migraine-Related Disability and Improvement in Patient Reported Outcomes After Oral Treatment With Rimegepant at the International Headache Conference Late Breaking Session [news release]. https:// www.biohavenpharma.com/investors/news-events/press-releases/09-09-2019
10. FDA approves first treatment for inherited rare disease [news release]. FDA’s website. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-inherited-rare-disease
11. Epizyme Announces Positive Pre-NDA Meeting for Tazemetostat for Follicular Lymphoma, Pipeline Updates and Third Quarter 2019 Results [news release]. Epizyme’s website. https://epizyme. gcs-web.com/news-releases/news-release-details/epizyme-announces-positive-pre-nda-meeting-tazemetostat
12. Intercept Submits New Drug Application to the US FDA for Obeticholic Acid in Patients with Fibrosis Due to NASH [news release]. Intercept’s website. http://ir.interceptpharma.com/news-releases/news-release-details/intercept-submits-new-drug-application-us-fda-obeticholic-acid