What pharmacists should know about the new drugs approved in 2024

In this article:

• Notable FDA approvals
• The bottom line

In this article

• Notable FDA approvals
• The bottom line

2024 has been a busy year for new drugs. Over 40 new medications have been approved by the FDA, giving patients more options for treating various health problems. These new drugs cover a wide range of conditions, from urinary tract infections to rare genetic disorders.

Notable new drugs approved in 2024

By staying up-to-date on the latest drug approvals, pharmacists can give new advice when working with patients and other healthcare providers. Therefore, it may be helpful to know how these new drugs work, their effectiveness, and what they’re used for.

Zelsuvmi (berdazimer)

Zelsuvmi received the green light from the FDA on January 5, 2024. It’s a new topical treatment for molluscum contagiosum in patients aged one and older. In clinical trials, Zelsuvmi showed strong results, with about 30% of patients completely clearing their skin of molluscum contagiosum after 12 weeks, compared to around 20% of those using the placebo treatment.

The gel works by releasing nitric oxide, which has antiviral properties. Patients apply it once daily for up to 12 weeks. Zelsuvmi’s approval is a big deal because it’s only the second FDA-approved treatment for this condition. It gives doctors and patients more choices to fight this viral skin infection.

Exblifep (cefepime/enmetazobactam)

The FDA approved Exblifep in February 2024 for treating complicated urinary tract infections in adults. This new drug combines cefepime, a fourth-generation cephalosporin, with enmetazobactam, a beta-lactamase inhibitor.

Exblifep is typically administered through an IV drip every 8 hours. The usual dose is 2.5 grams, which includes 2 grams of cefepime and 0.5 grams of enmetazobactam. This new antibiotic combo may be particularly useful against ESBL-producing pathogens that have become resistant to other antibiotics.

Duvyzat (givinostat)

The FDA approved Duvyzat on March 21, 2024, for treating Duchenne muscular dystrophy (DMD) in patients 6 years and older. This drug is a histone deacetylase inhibitor that works by slowing down muscle damage and improving muscle function in DMD patients. It’s the first non-steroid treatment for all DMD types, marking a big step forward in care.

Duvyzat is an oral suspension given twice daily with food. The dosage depends on the patient’s weight. Its approval offers a new option for DMD patients and their families, potentially helping to delay symptom progression and improve quality of life. This new drug adds to the growing list of treatments for DMD.

Xolremdi (mavorixafor)

The FDA approved Xolremdi on April 26, 2024. It’s the first drug for WHIM syndrome, a rare disorder that can cause severe infections. Xolremdi works by blocking a protein called CXCR4, which helps increase white blood cells in the body. In clinical trials, Xolremdi reduced the total number of infections by about 40% and cut the annual rate of infections by 60% compared to those who received a placebo.

Patients 12 and older can take Xolremdi capsules. The approval is significant for people with WHIM syndrome who didn’t have any specific treatments before. Now, they have a medicine that might help prevent serious infections and improve their quality of life.

Imdelltra (tarlatamab-dlle)

The FDA approved Imdelltra on May 16, 2024. It’s the first T-cell engager therapy for extensive-stage small-cell lung cancer. This drug works by connecting T-cells to cancer cells, helping the immune system fight the disease.

Healthcare providers start patients on 1 mg for the first dose, then bump it up to 10 mg twice in the first cycle. After that, it’s given every two weeks. Imdelltra’s approval is key for people with this aggressive type of lung cancer who’ve already tried other treatments. It gives them a new option when other drugs haven’t worked.

Piasky (crovalimab-akkz)

Piasky is a new drug that the FDA approved in June 2024. It’s designed to help people with a rare blood disorder called paroxysmal nocturnal hemoglobinuria (PNH). This medicine is for adults and children 13 and older who weigh at least 40 kg (just over 88 pounds). It works by stopping the breakdown of red blood cells, which is a significant problem for people with PNH.

Piasky comes as a shot that patients can give themselves at home after training and instructions from a healthcare provider. That could mean less time at the doctor’s office for these patients. In studies, it worked about as well as an older drug called eculizumab.

Kisunla (donanemab-azbt)

Kisunla is a new Alzheimer’s drug approved by the FDA in July 2024. It’s made by Eli Lilly and works by targeting amyloid plaques in the brain. The medicine is for people in the early stages of Alzheimer’s, including people with mild cognitive impairment or mild dementia. It’s administered as a monthly IV infusion.

In clinical trials, Kisunla showed it could slow down the progression of Alzheimer’s symptoms. It’s not a cure, but it might help people stay independent for longer. Kisunla joins a small group of FDA-approved treatments that target the underlying causes of Alzheimer’s. It gives patients and their families another option in the fight against this tough disease.

Niktimvo (axatilimab-csfr)

The FDA approved Niktimvo in August 2024. It’s a new drug for treating chronic graft-versus-host disease (cGVHD) in adults and children. Niktimvo works by blocking a protein called CSF-1R, which helps control certain immune cells and can help reduce the symptoms of cGVHD.

Niktimvo is given as an IV infusion once every two weeks. It’s used when at least two other treatments haven’t worked well enough. The approval of Niktimvo gives patients with cGVHD a new option when other treatments haven’t helped.

Aqneursa (levacetylleucine)

Aqneursa is a new drug approved by the FDA in September 2024. It’s used to treat a rare condition called Niemann-Pick disease type C (NPC). This medicine helps with the brain-related symptoms of NPC in both adults and children who weigh at least 15 kilograms (just over 33 pounds).

NPC is a genetic disorder that affects how the body manages fats. It can cause serious problems with movement, speech, and thinking. Aqneursa is the first FDA-approved treatment that can help improve these symptoms. It comes as a liquid that patients take by mouth, with the dosage depending on the person’s weight.

Hympavzi (marstacimab-hncq)

Hympavzi is a new drug that the FDA approved in October 2024. It’s used to help people with hemophilia A or B who are 12 years or older. Hympavzi can help stop or cut down on bleeding episodes, which is big news for people dealing with these blood disorders.

The drug, from Pfizer, could make life easier for people with hemophilia. It may be prescribed for hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors. The recommended dosage of Hympavzi includes a loading dose of 300 mg (two 150 mg injections) administered by subcutaneous injection, followed by a maintenance dose of 150 mg every week, starting one week after the loading dose.

Attruby (acoramidis)

The FDA approved Attruby in November 2024 for adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a rare heart condition that can be life-threatening. Attruby works by stabilizing the transthyretin protein, helping to reduce heart-related deaths and the need for hospital visits.

Patients take Attruby tablets orally twice daily. The drug’s approval gives healthcare providers a new option to treat ATTR-CM and may help improve the quality of life for people with this serious heart condition. Attruby joins two other medications, Vyndamax (tafamidis) and Vyndaqel (tafamidis meglumine), as an FDA-approved treatment specifically for ATTR-CM.

Rapiblyk (landiolol)

Rapiblyk is the latest drug to receive FDA approval at the time this article was written. It is a beta blocker approved for the short-term reduction of ventricular rate in adults with supraventricular tachycardia, including atrial fibrillation and atrial flutter. This medication is given through an IV and needs to be closely monitored while being administered.

For most patients with normal heart function, the treatment starts at 9 mcg/kg/min. The dose can be gradually increased every 10 minutes until it reaches a maximum of 36 mcg/kg/min. For patients with heart problems, the starting dose is lower.

The bottom line

2024 saw many exciting new drug approvals that could help lots of people. Some noteworthy medications include Zelsuvmi for molluscum contagiosum, Exblifep for complicated urinary tract infections, and Duvyzat for Duchenne muscular dystrophy. In addition, Xolremdi provides a treatment option for WHIM syndrome, while Imdelltra is a new therapy for extensive-stage small-cell lung cancer. These new meds might change how we treat some common and serious health problems.