From breakthrough innovative treatments to blockbuster sales, drug approvals have the ability to transform the future of pharmaceutical companies. In 2024, the FDA approved a range of new drugs that will be of significant interest to investors and patients alike.
Among the drugs approved this year, two stood out: Janssen’s daratumumab for multiple myeloma and Lilly’s Alzheimer’s hopeful donanemab. Both drugs improve upon the first-generation myeloma drug Trikafta, demonstrating better efficacy and patient tolerability and expanding the eligibility to transplant-eligible patients. Despite the strong performance of these two drugs, Evaluate estimates that their combined global sales will be less than $8 billion.
Another notable development was the FDA’s expansion of its accelerated approval program to include rare diseases. This is an attempt to reduce the time it takes for the FDA to approve a new drug from a company, with the goal of reviewing these medications in just a few months rather than ten. FDA Commissioner Marty Makary also suggested that the FDA should be willing to waive its scientific requirements for certain drugs targeting rare conditions that are considered life-threatening.
Interestingly, this year’s approvals show that bio/pharmaceutical giants and small/mid-sized firms have become more evenly split in terms of their drug approvals with each recording eight of the 26 new medications approved by FDA’s CDER. The TIDES class of drugs dominated with nine approvals, including five peptides and four oligonucleotides (the peptide tirzepatide contains two mini PEGs, which may signal the return of pegylation as a strategy to increase half-lives following the withdrawal of peginesatide in 2013). A new trend we noted was that more FDA approvals this year stated whether the drugs were only indicated for adults or for adult and pediatric populations, reflecting the FDA’s recent focus on this issue.