For over two decades, 1996 has stood out as a successful year for drug approvals, with a whopping 53 approvals following a new user fee program. Until 2018, that is. The total number of FDA approvals for 2018 was 59, with 55 of those new molecular entities (NMEs) coming over the first 11 months of the year.
The 59 novel drugs and biologics leapt over the previous year’s number of drugs approved, which was 46 in 2017. The number of new products approved by the Center for Drug Evaluation and Research (CDER) in 2018 was also nearly double the center’s 10-year average of 33 approvals per year.
It wasn’t just the total number of approvals that makes 2018 stand out, either. For the first time, more than half (58%) of approved NMEs target orphan indications. Further, one-third (32%) of the drugs were first-in-class molecules and one-quarter had breakthrough designations. Additionally, three-fourths of the NMEs benefitted from priority review.
2018 also brought the first approval under the recently created limited population pathway for antibacterial and antifungal drugs (LPAD) pathway, Insmed’s Arikayce (amikacin liposome inhalation suspension), which is approved to treat nontuberculous mycobacterial lung disease.
“While CDER approved 59 novel drugs in 2018, more than in any prior year, CDER, as always, focuses on the medical value of the approvals, not the number. We also approved new uses, new formulations, and new dosage forms for many already FDA-approved drugs that will help to advance patient care,” FDA says.
Some of the novel approvals in 2018 include: the first drug to treat smallpox, SIGA Technologies’ Tpoxx (tecovirimat); the first drug to treat a rare inherited form of rickets known as x-linked hypophosphatemia, Ultragenyx’s Crysvita (burosumab-twza); and the first cannabidiol-based drug, GW Pharmaceuticals’ Epidiolex (cannabidiol).
The question now becomes whether 2018 will be an outlier, similar to the way 1996 was, or if the level of drug development and approval activity has truly moved up a notch. By looking at the pipeline of application, there remain encouraging signs for the sustainability of the activity seen last year.