If in 2017, the FDA was trying to make up for lost time, the agency went into overdrive in 2018, approving more than 50 new molecular entities. The strong showing in 2018 followed a more-than-respectable 46 NMEs the previous year, which came after an anemic 2016 that saw only 22 approvals.
By the FDA’s count, it approved 59 NMEs in 2018, topping its previous record of 53 in 1996. If you’re wondering why our count is different, there are multiple reasons. We included two biologics—Andexxa and Jivi—and Inbrija, a new formulation of levodopa, in our list. We omitted other drugs, such as Akynzeo. The combination of netupitant and palonosetron first earned the FDA’s OK as an oral drug for the prevention of nausea in patients undergoing chemotherapy in 2014. The FDA approved an injectable version of Akynzeo that uses a prodrug of netupitant for the same indication in 2018 and listed it as an NME. We also omitted Omegaven, the active ingredient of which is fish oil triglycerides, and Annovera, a contraceptive ring that releases hormone medications that are already available on the market. And though the FDA listed cancer drugs Braftovi and Mektovi separately, we considered them a combination treatment, like Zepatier (elbasvir/grazoprevir) in 2016.
With that out of the way, let’s get down to what happened in 2018.
Predictably, cancer drugs came out tops with 16 approvals,with blood cancer treatments coming out slightly ahead of those for solid tumors. AstraZeneca’s Lumoxiti and Verastem’s Copiktra were both approved as third-line treatments for blood cancers—the former for hairy cell leukemia, a rare, slow-growing cancer, and the latter for chronic lymphocytic leukemia, follicular lymphoma and small lymphocytic lymphoma.
Several drugs were indicated for genetically defined cancers: Array BioPharma's Mektovi/Braftovi for BRAF-mutated melanoma and Agios' Tibsovo for relapsed or refractory acute myeloid leukemia with an IDH1 mutation, as well as a trio of drugs from Pfizer, namely Vizimpro for EGFR-mutated non-small cell lung cancer, Lorbrena for ALK-positive NSCLC, and Talzenna for BRCA-mutated, HER2-negative breast cancer. And who could forget Bayer and Loxo’s Vitrakvi, the "tissue-agnostic" drug that won approval to treat patients whose tumors feature a neurotrophic receptor tyrosine kinase gene fusion?
The number of rare disease approvals almost matched that of cancer approvals. Thrombocytopenia saw two new approvals in Doptelet and Tavalisse, as did hereditary transthyretin amyloidosis (hATTR) in Alnylam’s Onpattro and Akcea/Ionis’ Tegsedi. Onpattro also had the distinction of being the first approved drug for polyneuropathy stemming from hATTR, as well as the first small interfering RNA, or siRNA, medicine.
GW Pharma’s Epidiolex became the first cannabis-based medicine to get past the FDA. Approved for two rare forms of epilepsy, Dravet and Lennox-Gastaut syndromes, Epidiolex’s active ingredient is cannabidiol, a Schedule I drug with "no currently accepted medical use," which had to be rescheduled by the Drug Enforcement Administration (DEA) before GW could roll it out. While the DEA only rescheduled cannabidiol in the form of Epidiolex, GW Pharma’s historic approval could pave the way for other companies working on cannabis-based treatments.
Hepatitis C, the indication that seemed to keep on giving, at least these past few years, was absent from 2018’s crop, while HIV and migraine logged three new drugs apiece. Gilead’s HIV combo drug, Biktarvy, is expected to garner as much as $10 billion in sales, arriving in time to help stave off pressure on the Big Pharma’s hepatitis C franchise. And TaiMed’s Trogarzo, approved for multidrug-resistant HIV-1, is the first HIV therapy with a new mechanism of action approved in more than a decade.
Migraine was also a hot area, with several players racing to get a first-in-class nod for their CGRP inhibitors. Amgen and Novartis won that distinction with Aimovig, which was approved in May. Teva’s Ajovy and Eli Lilly’s Emgality followed suit in September, but there are still more to come. Allergan and Biohaven are both working on oral CGRP drugs, which represent a more attractive option for patients.
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