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Breakthrough Therapies for a Range of Conditions
While the number of drugs in development is rising, what is more impressive than the sheer number of candidates — and approvals — is the kinds of drugs being approved. Breakthrough Therapies, in particular, are once again making up a significant portion of new drugs coming to market — up from 20 percent of approvals in 2015 to 41 percent in 2017.
Here's an early look at some key Breakthrough Therapies expected to be reviewed by the U.S. Food and Drug Administration (FDA) in 2020.
NASH: Obeticholic Acid
Non-alcoholic steatohepatitis (NASH), is a severe form of fatty liver disease that is marked by inflammation and damage to liver cells — and in the most serious cases advanced fibrosis, cirrhosis, liver failure or liver cancer.
This fall, Intercept Pharmaceuticals filed a New Drug Application for obeticholic acid (OCA) to treat fibrosis in patients with NASH.
In Phase III trials, 23.1 percent of patients on a high-dose of OCA showed improvement in fibrosis with no worsening of NASH after 18 months, compared to 11.9 percent of the placebo group.
An FDA decision is expected by May 2020. If approved, OCA will likely be covered under the pharmacy benefit.
Non-Hodgkin’s Lymphoma: Lisocabtagene Maraleucel
One potentially promising avenue for cancer treatment is the use of chimeric antigen receptor T-cell therapies (CAR T-cell), in which T-cells (the immune system’s hunter-killers) are removed from the patient’s body, genetically engineered to target a specific cancer, and then reinjected.
They may be joined in 2020 by a third CAR T-cell therapy for lymphoma: Celgene’s lisocabtagene maraleucel (liso-cel) for the treatment of relapsed or refractory B-cell non-Hodgkin's lymphoma, including diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma, and follicular lymphoma.
Liso-cel is expected to have a price tag similar to that of Yescarta and Kymriah — $400,000 for a one-time dose. If approved, lisocabtagene maraleucel will likely be covered under the medical benefit.
Hemophilia Gene Therapy: Valoctocogene Roxaparvovec
It’s estimated that more than 20 cell- and gene-based treatments will come up for, or receive, FDA approval over the next year and a half.
|More than 20 cell- and gene-based treatments will come up for, or receive, FDA approval over the next 1.5 years
One particularly significant gene therapy candidate is BioMarin’s valoctocogene roxaparvovec, a therapy for severe hemophilia A in patients aged 18 and older, which is expected to be approved sometime between the second and late third quarter of 2020.
Hemophilia A occurs about once per 5,000 live male births, with an overall U.S. prevalence of about 16,000.
In preliminary data from a Phase III trial, 16 patients in the high-dose cohort had an annualized bleed rate reduction of 85 percent from baseline and their use of factor VIII fell by an annualized rate of 94 percent.
The company plans to file for approval in the United States and Europe in the fourth quarter of 2019, and the drug could be available as early as mid-2020.
Peanut Allergy: Palforzia and Viaskin Peanut
Peanut allergy is the most common of childhood food allergies. Currently, there are no FDA-approved treatments for peanut allergy and it is commonly managed with rescue epinephrine. However, two new products in the market could be the beginning of what is expected to be a $4.5 billion global market in the category.
The first drug likely to come to market is Aimmune’s Palforzia, which is pending approval for use to reduce the incidence and severity of allergic reactions after accidental exposure to peanut in patients aged 4 to 17 years, expected to be approved in January 2020. It is not a drug in a traditional sense but defatted, carefully measured doses of peanut flour that are administered orally to patients over a period of months. Dosing starts at half a milligram and builds up over six months to 300 milligrams — roughly the protein content of a single peanut — after which patients continue to receive maintenance doses of Palforzia.
Viaskin Peanut, by DBV Technologies, for children with peanut allergy aged 4 to 11 years is the second drug in development to protect against accidental exposure to peanuts. Viaskin delivers small quantities of the allergen via a skin or dermal patch containing 250 micrograms of peanut protein (about a quarter of a peanut), applied daily.
Palforzia is expected to cost between $3,000 and $20,000 a year, according to the company, while analysts estimate Viaskin Peanut to cost about $6,500.
*Unless otherwise specified, these values represent total potential U.S. candidates based on anticipated indication and disease prevalence calculations; they do not reflect drug market share vs. competitors.
This document contains references to brand-name prescription drugs that are trademarks or registered trademarks of pharmaceutical manufacturers not affiliated with CVS Caremark.
Image source: Licensed from Getty Images, 2019.
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