Key drugs to keep an eye on in 2025
A deep dive into seven drugs with the potential for FDA approval in 2025, spanning from pain management to rare diseases.
Welcome back to Biotech Blueprint and Happy New Year!
📢 What’s new:
I’ve officially launched subscriptions, but plan to keep most of my articles free for the foreseeable future. I’ve been creating content for a while now, and if you enjoy what you’re reading, consider subscribing for just the price of a Starbucks coffee each month. If that’s not your thing, sharing the blog with your friends is equally appreciated!
I’m excited to announce that I’ll be launching the Biotech Blueprint website very soon! It will feature a ton of resources, including consulting information, a ton of data, courses I’m developing, and much more. Stay tuned for all the materials I’ve been working on!
In this deep dive, I’ll outline the drugs and companies expected to make headlines in 2025. From new therapies to rising leaders, this article highlights what to watch for this year. The list is in no particular order of importance, and of course, it reflects my own biases on what I consider interesting.
1. A non-opioid treatment for acute pain
In late January 2025, Vertex Pharmaceuticals is expecting FDA decision for suzetrigine, an investigational non-opioid treatment for moderate-to-severe acute pain. Suzetrigine, a selective NaV1.8 pain signal inhibitor, has shown promise in phase 3 trials. The FDA granted the drug priority review, with a target action date of Jan. 30. If approved, suzetrigine would become the first new class of pain medication in over 20 years, offering a non-addictive alternative to opioids. The drug has the potential to address the significant unmet need for safer pain management options, particularly for those suffering from acute pain, chronic pain, and conditions like diabetic neuropathy.
Phase 3 data revealed significant efficacy in reducing pain and an excellent safety profile with 1.8% of patients experiencing severe side effects. Despite these positive findings, analysts have mixed views on the commercial potential of suzetrigine. BMO Capital Markets analysts are confident that suzetrigine will play an important role in treating acute pain, particularly in post-surgical settings. However, analysts from William Blair have raised concerns about the challenges of entering the acute pain market, noting that suzetrigine may face competition from more affordable alternatives.
Vertex remains optimistic about the potential of suzetrigine to become a significant player in pain management, especially given the priority review status given by the FDA, emphasizing the need for non-addictive pain treatments.
2. GLP-1s for sleep apnea and chronic kidney disease
When I started outlining this article, there were no FDA-approved GLP-1 drugs for sleep apnea, but clinical trial data suggested that approval could be just around the corner. On Dec. 20, 2024, Eli Lilly’s Zepbound (tirzepatide) was FDA-approved as the first treatment for moderate-to-severe obstructive sleep apnea (OSA) in patients with obesity. GLP-1s, originally developed for obesity and diabetes, target the underlying weight issues that often worsen OSA. Clinical trials demonstrated a significant reduction in OSA symptoms, allowing many patients to discontinue CPAP therapy (steady stream of pressurized air through a mask worn while sleeping).
The approval of Zepbound for OSA underscores both promise and challenges. While it offers hope for those with obesity-related sleep apnea, its effectiveness may be limited for others. Cost and supply issues remain significant obstacles, especially as demand for GLP-1 drugs rises. However, experts anticipate that this approval will spark further innovation, with future treatments likely combining GLP-1s with traditional therapies like CPAP for more personalized care.
In 2025 and beyond, GLP-1 medications are expected to gain approval for a variety of new indications beyond diabetes, obesity, and cardiovascular disease. Ongoing research highlights their potential in treating conditions such as Parkinson’s disease, Alzheimer’s, osteoarthritis, and even alcohol use disorder. Promising early data also suggests GLP-1s could be effective in managing chronic kidney disease, with Novo Nordisk’s Ozempic likely to receive approval for this use in 2025. In a phase 3 trial, the drug reduced the risk of kidney disease-related events by 24% in adults with type 2 diabetes and chronic kidney disease.
3. Novel treatment for acute migraines
In September 2024, Axsome Therapeutics announced that the FDA has accepted its resubmitted New Drug Application for AXS-07, a treatment for acute migraine, with a final decision expected by Jan. 31, 2025. This follows an earlier complete response letter in April 2022, which raised issues related to chemistry, manufacturing, and controls. No new safety or efficacy data were required for the resubmission, which was completed in Q2 2024. The potential approval of AXS-07 would diversify Axsome’s portfolio, which currently includes Auvelity for major depressive disorder and Sunosi for narcolepsy.
AXS-07 is a novel oral medication that enables rapid absorption of meloxicam while maintaining a long plasma half-life. This multi-mechanistic drug works by inhibiting CGRP release, a key factor in migraine pain, and provides sustained relief for several hours. Clinical trials demonstrated significant efficacy, with AXS-07 achieving greater headache pain freedom and reducing the need for rescue medication compared to placebo, while maintaining a favorable safety profile.
4. Nasal spray for rapid heartbeats
In March 2025, Milestone Pharmaceuticals is expecting FDA decision for Cardamyst (etripamil), a nasal spray developed for the management of paroxysmal supraventricular tachycardia (PSVT). Cardamyst could provide a novel, self-administered, rapid-response treatment for patients suffering from this type of arrhythmia, a condition that causes unpredictable episodes of rapid heartbeats, which can be debilitating. It affects about 2 million people in the U.S., especially younger adults.
This product represents a significant advancement in the field, as it allows patients to manage acute PSVT episodes independently, reducing reliance on emergency department visits or invasive procedures like cardiac ablation. Cardamyst is designed to provide rapid relief at home, improving patient convenience and control over their condition.
5. New class of antibiotic for UTIs
GSK’s investigational oral antibiotic, gepotidacin, was accepted for priority review by the FDA for treating uncomplicated urinary tract infections (UTIs) in adult and adolescent females back in October 2024. If approved, it could be the first new class of oral antibiotics for UTIs in over 20 years.
Supported by positive results from phase 3 EAGLE-2 and EAGLE-3 trials, gepotidacin demonstrated non-inferiority to the current standard, nitrofurantoin, and showed superiority in EAGLE-3. The novel aspect of gepotidacin lies in its unique mechanism of action: it inhibits bacterial DNA replication by targeting a distinct binding site on two type II topoisomerase enzymes, making it effective against a broad range of uropathogens, including drug-resistant bacterial strains. This could address the growing challenge of antibiotic resistance, offering a promising solution for patients with UTIs, particularly as resistance to current treatments rises.
FDA decision is expected by late March 2025.
6. Novel hemophilia treatment
Sanofi’s fitusiran, an investigational treatment for hemophilia A and B, is currently under FDA review with a decision expected by Mar. 28, 2025. Fitusiran works by lowering antithrombin, a protein that inhibits clotting, to reduce bleeding risks in hemophilia patients. Data presented at the 2024 ISTH Congress highlighted fitusiran’s success in managing bleeds during major surgeries, showing no safety concerns and excellent blood control in 90% of cases. Additionally, the therapy demonstrated a strong safety profile.
Unlike traditional therapies, which replace missing clotting factors, fitusiran works by reducing antithrombin, a natural inhibitor of blood clotting, thereby enhancing thrombin generation and improving clotting. This mechanism is effective even in patients with inhibitors, who often face challenges with conventional treatments. Additionally, fitusiran offers a low treatment burden, requiring as few as six injections per year, and does not need refrigeration, making it a more convenient and accessible option compared to other therapies.
7. First treatment for rare post-transplant cancer linked to EBV
Last summer, Tabelecleucel (tab-cel) was granted priority review by the FDA for the treatment of Epstein-Barr virus (EBV)-positive post-transplant lymphoproliferative disease (PTLD), a serious condition with no approved therapies in the U.S. If approved, tab-cel would be the first FDA-approved treatment for this rare disease. Tab-cel is an allogeneic, EBV-specific T-cell immunotherapy that targets and eliminates EBV-infected cells. Atara Bio, the developer of tab-cel, is expecting to hear from the FDA by Jan. 15, 2025. Their biologics license application (BLA) is supported by data from phase 3 ALLELE trial, which demonstrated a statistically significant 48.8% objective response rate with a favorable safety profile.
Tab-cel has already received breakthrough therapy and orphan drug designations and has been approved in Europe under the name Ebvallo. It has also been granted marketing authorizations in the U.K. and Switzerland. Atara is preparing for a potential U.S. launch in early 2025.
Thanks for reading Biotech Blueprint and I hope you have a relaxing weekend.
DISCLAIMER: This content is for informational purposes only. It should not be taken as legal, tax, investment, financial, or other advice. The views expressed here are my own and do not reflect the opinions of any company or institution.
DISCLOSURE: I have no business relationships with any company mentioned in this article.
Very informative