New Approved and Emerging Treatments in Oncology
In 2017, the brisk pace of drug approvals from the US Food and Drug Administration allowed an influx of new treatment options for oncology indications. These included novel breakthrough treatments for solid tumors and hematologic malignancies, new and expanded indications for existing approved therapies, and the addition of new agents in drug classes with already demonstrated success. As the rapid evolution of the oncology treatment landscape is expected to continue, it is important for oncology care providers and decision-makers to stay informed about the latest generation of treatment options as well as their impact on standard of care.
A Year of Firsts in Oncology Drug Approvals
The year 2017 was a particularly successful one for new specialty drug approvals by the US Food and Drug Administration (FDA). After a dip in in 2016, 2017 is on track for 30 approvals—more than any previous year except 2015.1 As of December 1, 2017, a total of 44 agents received new approvals from the FDA for oncology indications.2
This year also saw a lot of firsts, including several products that offered treatment options for patients with complex and previously untreatable cancers (Table).3 Orphan drugs, which treat rare conditions, make up a growing proportion of the specialty drug pipeline.1 However, even as more options enter the market, drug prices are not dropping. Similarly, orphan drugs, which constitute a growing percentage of the specialty drug pipeline, cost an average of 5 times more than nonorphan drugs. Thus, although the conditions treated with orphan drugs are rare, 30% of drugs in this category are considered “blockbusters” as they bring in $1 billion or more each year.1
When the FDA approved Mvasi (bevacizumab-awwb; Amgen) as a biosimilar to Avastin (bevacizumab) on September 14, it became the first biosimilar approved in the United States for the treatment of cancer. Since then, a second biosimilar, Ogivri (trastuzumab-dkst, Mylan) has also been approved. Biosimilars are expected to become an increasingly important part of the specialty pharmaceuticals market. Biosimilars “are really acting like competing brands,” said Aimee Tharaldson, PharmD, of Express Scripts, at the Academy of Managed Care Pharmacy 2017 Nexus meeting.1 This will become especially so as more regulatory and litigation hurdles are eliminated.1 While this year has yielded additional guidance on the naming of biosimilar drugs, the same has not been true for interchangeability requirements. Once those regulations are known and biosimilar drugs can be deemed interchangeable, it is anticipated that they will produce greater cost savings.1
Another first emerged with the US approval of the chimeric antigen receptor T-cell (CAR-T) cancer immunotherapy Kymriah (tisagenlecleucel; Novartis) in August. This landmark approval was soon followed by a second when Yescarta (axicabtagene ciloleucel; Kite Pharma) was approved in October for advanced non-Hodgkin lymphoma.1
One notable trend this past year has been the approval of new and multiple indications for existing drugs such as Opdivo (nivolumab; Bristol-Myers Squibb) and Keytruda (pembrolizumab; Merck). But it was the May 23 accelerated approval of Keytruda that gained the most attention as the drug became the first FDA-approved cancer treatment based on a common biomarker rather than tumor location.
One growing trend to keep an eye on is the drug development movement toward specific patient subpopulations and a focus on targeting cancer gene mutations and molecular pathways.4 A few emerging key areas for new therapeutic advances are discussed below.
Growth in Oral Oncolytics
Of the novel cancer drugs approved in 2017, over half were oral oncolytic therapies.5 The trend is expected to continue moving forward, as roughly 4 of 10 cancer therapies in the current pipeline are oral medications.3 The cancer pipeline for 2018 includes five oral drugs for a range of tumors: the nonsteroidal antiandrogen apalutamide (Janssen) for castration-resistant prostate cancer; entinostat (Syndax) for estrogen receptor-positive breast cancer; ivosidenib (Agios) for acute myeloid leukemia; larotrectinib (Array BioPharma) for TRK+ fusion tumors; talazoparib (Pfizer) for BRCA mutation-positive breast cancers.1 Other than apalutamide and entinostat, most cancer drugs in the 2018 pipeline are targeted therapies.1 Three antibody-drug conjugates for targeted delivery are also in the 2018 pipeline: AbbVie’s depatuxizumab for glioblastoma brain tumors and rovalpituzumab for small-cell lung cancer, and Seattle Genetics’ sacituzumab for triple-negative breast cancer.3