At first glance, the number of drugs that received accelerated approval from the U.S. FDA’s Center for Drug Evaluation and Research (CDER) in 2023 was nothing to write home about. Yes, CDER granted nine accelerated approvals last year, up from six in 2022. But the proportion of novel drugs with accelerated approval was 16% both years. And when compared with the 12 drugs in 2020 and the 14 that received accelerated approval in 2021, last year’s crop was a little lackluster. However, a deeper look at the 2023 class of accelerated approvals shows a historic milestone. For the first time since the path was created in 1992, the number of novel biologics getting accelerated approval at CDER outpaced the number of small-molecule drugs.
Patients with amyotrophic lateral sclerosis (ALS) have a median survival of 2 to 5 years. There are 3 FDA-approved drugs for ALS (riluzole, edaravone and Relyvrio [phenylbuturate/taurursodiol]), but they only lead to modest benefit. There are several pathways involved in the disease, but all of them lead to neuroinflammation.
In 2023, the BioWorld Neurological Diseases Index (BNDI) closed with a 4.36% increase, outperforming the Nasdaq Biotechnology Index (up 3.74%) but falling short of the surge seen in the Dow Jones Industrial Average (up 13.7%). The rebound marked a significant turnaround from November, when BNDI recorded an 18.37% year-to-date decline.