10 new drugs expected to make waves in 2024

The landscape of pharmaceuticals continues to evolve each year. As a pharmacist, it’s always a good idea to stay in the know on upcoming FDA approvals. That way, you can avoid any confusion or surprise if a patient inquires about a new medication for their condition or a healthcare provider recommends something different.

As the new year begins, industry professionals are closely monitoring the Food and Drug Administration (FDA) calendar for decisions that could bring new drugs to the market. These upcoming FDA approvals could mean advanced treatment options and improved quality of care for patients with various medical conditions.

10 potential upcoming FDA approvals for 2024

Pharmacists are on the frontline when it comes to getting patients the right medications for their treatment plan. Therefore, you’ll want to prepare and adapt to potential 2024 FDA approvals, which may include the following drugs currently in the pipeline.

1. Lebrikizumab for atopic dermatitis

Lebrikizumab is on the cusp of FDA approval for treating moderate-to-severe atopic dermatitis. Phase 3 trials have shown that lebrikizumab is effective in treating moderate-to-severe atopic dermatitis, with about one-third to nearly half of patients experiencing clear or almost clear skin after 16 weeks. It works as an interleukin-13 (IL-13) inhibitor that blocks a key cytokine driving inflammation in the skin. As a result of these trials, lebrikizumab could become a potential first-line option for atopic dermatitis. 

2. Tolebrutinib for multiple sclerosis

Tolebrutinib has shown promise as a novel oral treatment for multiple sclerosis (MS), and it could receive the FDA green light in 2024. It works by inhibiting Bruton’s tyrosine kinase, an enzyme involved in the function of certain immune cells that are believed to play a role in inflammation in multiple sclerosis. A Phase 2b trial demonstrated that high-dose tolebrutinib (60 mg) significantly reduced brain lesions by at least 85% in patients with relapsing MS over a 12-week treatment period compared to a placebo.

3. Danicopan for paroxysmal nocturnal hemoglobinuria

Danicopan may be a potentially new add-on option to ravulizumab or eculizumab for patients with paroxysmal nocturnal hemoglobinuria (PNH) who experience clinically significant extravascular hemolysis (EVH). It works by inhibiting Factor D, which may help reduce the overactive immune response that causes the destruction of red blood cells in PNH. A Phase 3 trial showed that danicopan added to ravulizumab or eculizumab resulted in a significant hemoglobin increase of 2.94 g/dL versus 0.50 g/dL with a placebo, and no new safety concerns were reported.

4. Crovalimab for paroxysmal nocturnal hemoglobinuria

Crovalimab is another agent on the horizon for PNH treatment. It potentially offers alternative dosing options, as it’s a subcutaneous injection given every four weeks. The medication could ease patient burden by allowing for potential at-home self-administration. Based on data from the COMMODORE 3 study, crovalimab achieved hemolysis control in 78.7% of PNH patients and significantly reduced transfusions without serious adverse events.

5. Resmetirom for nonalcoholic steatohepatitis

Resmetirom, a thyroid hormone receptor-beta agonist, has been making its way through the approval process as a treatment for nonalcoholic steatohepatitis (NASH). It works by regulating fat metabolism and inflammation in the liver, which is key in reversing or slowing the progression of NASH. In the MAESTRO-NAFLD-1 trial, resmetirom was found to reduce LDL-C by up to 12.6% and hepatic fat by up to 38.6% over 52 weeks, compared to a placebo. This therapy could be a safe and effective treatment for a condition that currently lacks FDA-approved medications.

6. Fordadistrogene movaparvovec for Duchenne muscular dystrophy

Fordadistrogene movaparvovec is a single-infusion gene therapy that could revolutionize treatment for Duchenne muscular dystrophy. It works by introducing a functional dystrophin gene to patients’ muscle cells, which may help tackle the genetic root of the disease rather than just managing symptoms. In a Phase 1b trial of DMD patients, treatment with fordadistrogene movaparvovec led to a significant increase in muscle volume, with a mean change from baseline of 9.0% at one year and 7.4% at two years.

7. Lifileucel for advanced melanoma

Lifileucel is a tumor-infiltrating lymphocyte (TIL) cell therapy that may be effective in treating patients with advanced melanoma. By using the patient’s own immune system, lifileucel could be a personalized medicine strategy that could greatly improve outcomes. Lifileucel showed an objective response rate of 31.4% in a Phase 2 trial involving patients with advanced melanoma who had progressed after standard treatments. An ongoing Phase 3 trial is determining the effectiveness and safety of lifileucel with pembrolizumab for untreated advanced melanoma. 

8. Sotatercept for pulmonary arterial hypertension

Sotatercept stands out as a potential treatment for pulmonary arterial hypertension (PAH) that targets the transforming growth factor-beta pathway. This pathway may be involved in the thickening of pulmonary arteries, which is a hallmark of PAH. This new drug may improve symptoms and could potentially reverse or halt disease progression. In a Phase 3 trial, sotatercept significantly improved exercise capacity in patients with PAH, with an increase of 34.4 meters in a six-minute walk distance over a placebo.

9. Atidarsagene autotemcel for metachromatic leukodystrophy

Atidarsagene autotemcel is a gene therapy that may help slow the progression of metachromatic leukodystrophy, a devastating genetic disorder. It works by inserting a functional copy of the ARSA gene directly into patient cells. In a study spanning almost seven years, atidarsagene autotemcel successfully preserved motor and cognitive abilities in 94% of children with early MLD, a great improvement over untreated patients who typically lose mobility by around 3 years old. 

10. Firsocostat for nonalcoholic steatohepatitis

Firsocostat, an investigational therapy for NASH, is an acetyl-CoA carboxylase (ACC) inhibitor that works by blocking an enzyme involved in lipid synthesis. Its main goal is to reduce liver fat content, a key factor in the progression of NASH. By targeting liver fat accumulation, firsocostat could slow or even reverse disease progression. Currently, the drug is being studied in combination with other drugs like cilofexor and semaglutide. 

How to stay up-to-date on new approvals

As a pharmacist, it’s important to stay up-to-date on new approvals, and there are a number of different ways to do so. For example, the FDA often posts novel drug approvals for the past year in December. Their database includes all recently approved medications, including new molecular entities and biological products. You can also learn more about their drug approval process, including priority reviews and accelerated approvals. 

Numerous free and paid resources also provide calendars and information about recent or upcoming FDA decisions. These resources, such as FDA Tracker, Benzinga, and BioPharm Catalyst, may offer a streamlined view into the immediate future of pharmaceuticals. Email alerts can be set up on platforms like RTTNews to receive notifications about pending approvals or regulatory actions.

Bottom line: New drugs mean better patient care

Pharmacists may need to prepare for an influx of new drugs into the market in 2024, as the FDA is expected to approve a range of new medications for conditions like atopic dermatitis, multiple sclerosis, paroxysmal nocturnal hemoglobinuria, and more. While these drugs may provide new options for treatment, it’s often wise to assess each medication’s complete profile. That means understanding their mechanisms of action and potential side effects or drug interactions. By staying up-to-date and prepared for upcoming FDA approvals, pharmacists can make the most of new advancements and continue to provide the highest level of patient care.