2024's Most Influential Drug and Vaccine Approvals So Far — As Selected by GoodRx Pharmacists

GoodRx Health

The FDA approves hundreds of new medications every year. Some of them are innovative medications that are the first of their kind. Others are medications that follow in the footsteps of a larger family of drugs. Many more are new generics or biosimilars that aim to offer additional savings opportunities.

Every new medication deserves its time in the spotlight. But due to the nature of a medication's expected impact, some new FDA approvals represent especially meaningful steps forward.

Here, we'll review some of the most influential drug and vaccine approvals of 2024 to date, all selected by GoodRx Health pharmacists. But keep in mind this isn't an exhaustive list, and it only reflects products approved through June 30.

Disclaimer: While creating this list, we prioritized first-in-class medications and vaccines that are expected to have a significant impact on human health. This was defined as either the number of lives touched or the expected degree of impact for future medication recipients. We also prioritized medications with effectiveness or safety data that represent a notable advantage over other medications recommended by clinical treatment guidelines or mainstay medical organizations.

Summary of 2024 approvals

Many medications have been approved so far in 2024 - and more are on deck. Here's a summary of 11 of the most notable approvals.

Let's review each of these medications and vaccines in more detail.

1. Ohtuvayre for COPD

What is it:Ohtuvayre (ensifentrine) is an inhaled medication that treats chronic obstructive pulmonary disease (COPD) in adults. It's set to launch in the second half of 2024.

FDA approval date:June 26, 2024

How it's taken: Ohtuvayre is inhaled using a nebulizer. It's designed to be used twice daily as a maintenance breathing treatment.

Why Ohtuvayre's approval matters

Ohtuvayre is the first new type of maintenance COPD medication in over 10 years. It works differently than existing COPD treatment options.

Most COPD medications enhance breathing by either lowering inflammation or by relaxing muscles and widening airways (bronchodilation) in the lungs. Inhalers often contain one of each medication type.

Ohtuvayre offers bothof these benefits in a single active ingredient. It works by blocking a protein called phosphodiesterase (PDE) 4 to lessen inflammation, similar to roflumilast (Daliresp). But it also blocks PDE 3, which is what provides its bronchodilator effects.

Ohtuvayre, though, isn't meant for quick relief. And if using a nebulizer isn't ideal for you, inhaler forms of ensifentrine are currently in phase 2 clinical trials and may be available later on.

2. Wakix

What is it: Wakix (pitolisant) is an oral medication that's approved to treat excessive daytime sleepiness associated with narcolepsy, a chronic sleeping disorder, in adults and kids.

FDA approval date:June 24, 2024

How it's taken: Wakix is an oral tablet that's taken once daily. You'll likely start with a low dose and gradually increase your dose over a few weeks until you reach your target dose.

Why Wakix's approval matters

Wakix, initially approved in 2019 for narcolepsy in adults, is now a treatment option for pediatric narcolepsy. It can treat excessive daytime sleepiness in kids ages 6 and older.

Before Wakix, Xyrem (sodium oxybate) and Xywav (calcium oxybate / magnesium oxybate / potassium oxybate / sodium oxybate) were the only medications approved for pediatric narcolepsy. But they're both controlled substances, meaning that they have a risk of dependency and misuse. Xyrem and Xywav also have inconvenient dosing schedules, requiring a dose at bedtime, followed by a second dose a few hours after your child goes to sleep.

Wakix isn't a controlled substance, and it's only taken once a day.

3. Capvaxive

What is it: Capvaxive is a vaccine that helps prevent pneumococcal disease in adults, including pneumococcal pneumonia. It's categorized as a 21-valent pneumococcal conjugate vaccine.

FDA approval date:June 17, 2024

How it's taken: Capvaxive is given as a single dose into the muscle. A pharmacist, physician, or another healthcare professional can administer it to you. However, since it's newly approved, the CDC hasn't incorporated it into its official vaccine recommendations yet. These recommendations provide specific guidance about who should receive vaccines and when.

Why Capvaxive's approval matters

There are several vaccines available to prevent pneumococcal disease. Vaxneuvance, Prevnar 20, and Pneumovax 23 are all CDC-recommended for certain kids and adults. Each vaccine contains pieces of different types of pneumococcal bacteria (serotypes).

Capvaxive, made by the same company as Pneumovax 23, is now approved as a pneumococcal vaccine specifically for adults. People ages 18 and older are eligible to receive it.

What makes Capvaxive unique is that it addresses the serotypes responsible for about 84% of invasive pneumococcal disease (e.g., meningitis) in adults ages 50 and older. This includes eight serotypes that aren't currently covered by the other pneumococcal vaccines.

4. mRESVIA

What is it: mRESVIA is a mRNA vaccine for respiratory syncytial virus (RSV). It's approved for adults ages 60 and older. mRESVIA should be available by the start of 2024-2025 respiratory illness season.

FDA approval date:May 31, 2024

How it's taken: mRESVIA is given as a single dose into the muscle by a healthcare professional. You and your primary care provider can decide if mRESVIA or another RSV vaccine is right for you.

Why mRESVIA's approval matters

mRESVIA is the first mRNA vaccine to be approved for something other than COVID-19. This breakthrough is encouraging for other mRNA vaccines moving forward.

mRESVIA isn't the first RSV vaccine, though. Two other RSV vaccines, Arexvy and Abrysvo, were approved in 2023. But these are both protein subunit vaccines - a more conventional vaccine technology. If you want or need another option, mRESVIA is a good choice to consider.

5. Winrevair for pulmonary arterial hypertension

What it is: Winrevair (sotatercept) is a biologic medication that treats pulmonary arterial hypertension (PAH), a type of high blood pressure in the lungs. It's classified as an activin signaling inhibitor.

FDA approval date:March 26, 2024

How it's taken: Winrevair is an injectable medication that's given under the skin. You can inject it at home once every 3 weeks.

Why Winrevair's approval matters

Medical advancements have helped improve the survival rate among people with PAH. But PAH remains a progressive and life-threatening condition. For those living with PAH, medications can help to slow progression of the condition and improve quality of life. Winrevair is one treatment that can have an additional impact on top of existing PAH treatments.

Taking a step back, PAH happens when the arteries in the lungs become thickened or narrowed. This can make it harder for your heart to pump blood through the lungs, since pressure and resistance is increased. Common PAH symptoms include shortness of breath, fatigue, and swelling in your extremities.

Most PAH medications reduce this pressure by widening blood vessels in the lungs or preventing them from narrowing. But Winrevair works differently. It targets the pathway that contributes to structural changes (such as thickening) in pulmonary artery walls. It helps reverse some of these structural changes.

During clinical trials, Winrevair was studied in adults with PAH as well as slight-to-marked limitations in their physical activity even with existing treatments. After nearly 6 months of use, people receiving Winrevair had a significant improvement in the distance they could walk in 6 minutes compared to those taking placebo. They also had significantly reduced pulmonary vascular resistance, which is the force against blood flowing through arteries in the lungs.

6. Nexletol for high cholesterol

What it is:Nexletol (bempedoic acid) is an oral medication that treats high cholesterol in adults alongside a low-cholesterol diet. It's similar to Nexlizet, a combination pill that contains both bempedoic acid and ezetimibe. Ezetimibe is the active ingredient in Zetia.

FDA approval date:March 22, 2024

How it's taken: Nexletol is an oral pill that's taken once daily. It's considered to be a statin alternative if statins aren't working for you or are causing bothersome side effects. You also have the option to combine it with a statin for added cholesterol benefits.

Why Nexletol approval matters

Nexletol isn't actually a new medication. It was initially approved in 2020 for relatively specific uses. Its approval in March 2024 is a broad label expansion that permits many more people to take it for cholesterol management.

Now, this oral medication can treat high cholesterol with or without a statin by its side. It's also approved to lower the risk of serious cardiovascular problems stemming from high cholesterol, such as a heart attack.

Nexletol offers a unique feature compared to statins, too. It targets a specific enzyme (protein) called adenosine triphosphate-citrate lyase (ACL). This enzyme is active at a different part of the cholesterol creation process than statins are. It also only works in the liver, helping to reduce the risk of muscle-related side effects that are normally associated with statins.

Nexletol's expanded approval ultimately allows for more flexibility in treating high cholesterol, giving people and their prescribers more options to find the best treatment for them. This can lead to better adherence and overall success in managing cholesterol.

7. Tryvio for high blood pressure

What it is:Tryvio (aprocitentan) is a blood pressure medication that's approved for adults with hard-to-treat high blood pressure. A type of endothelin receptor antagonist, it's meant to be combined with other blood pressure medications for added benefits. It's not designed to be taken by itself.

FDA approval date:March 20, 2024

How it's taken: Tryvio is a once-daily oral tablet. You can take it with or without food.

Why Tryvio's approval matters

Tryvio's approval is eye-catching because it's the first new type of blood pressure medicine in close to 4 decades. But its actual benefit goes far beyond the headline.

For some people, following a diet and exercise plan is enough to keep their blood pressure within range. For others, adding one or more blood pressure medications to the mix can keep things well managed. Unfortunately, for many adults, lifestyle changes and traditional blood pressure medications aren't enough to keep their blood pressure readings where they should be.

This is where Tryvio steps in. It works differently than other blood pressure medicines. Tryvio stops a protein, called endothelin, from binding to specific receptors in your blood vessels. This helps prevent blood vessel constriction and fluid retention, among other effects.

D. Edmund Anstey, MD, MPH, an Assistant Professor of Medicine at Columbia University Vagelos College of Physicians, gave GoodRx Health more context about why Tryvio's approach is unique.

"Tryvio is not just a new medication, but [it] represents a novel mechanism for controlling blood pressure. Identifying new and effective mechanisms for blood pressure control may help expand our understanding of this disease and help us continue to find effective ways to treat it," said Anstey. "For individuals with hypertension, in particular for people whose blood pressure is inadequately controlled on their current regimen, Tryvio represents a new tool to help in the fight to get blood pressure under control and ultimately prevent a major heart attack or stroke."

8. Wegovy for cardiovascular risk reduction

What it is:Wegovy (semaglutide) is an injectable medication commonly prescribed for weight loss. It also helps lower the risk of serious cardiovascular problems in adults with larger bodies and heart disease. Wegovy is a type of glucagon-like peptide-1 (GLP-1) agonist.

FDA approval date:March 8, 2024

How it's taken: Wegovy is injected just under the skin once a week. The best places to inject it are your stomach, upper thigh, or upper arm. You can conveniently inject your doses at home, too.

Why Wegovy's approval matters

In 2021, Wegovy became the first once-weekly medication in its class approved for weight loss. And this more recent approval marks another milestone. It's now the first weight-loss medication to be approved to prevent major adverse cardiovascular events (heart attack, stroke, or cardiovascular death), or MACE, in adults

Ozempic, a lower-dose semaglutide injection, is already approved to lower the risk of MACE in people with both diabetes and heart disease. Wegovy, on the other hand, offers this benefit in people with heart disease without diabetes, too. During clinical trials, people with heart disease who are considered overweight or obese saw roughly a 20% reduction in MACE with Wegovy.

Dave L. Dixon, PharmD, FACC, a member of the American College of Cardiology CV Team Pharmacists Work Group, elaborated more on what this benefit could mean for people.

"There is a strong link between obesity and increased risk of heart disease but until now we didn't have a pharmacological intervention that reduces that risk. It's important to recognize that the benefits of Wegovy aren't just about weight loss. It's about reducing one's risk of heart disease, which remains the leading cause of death," said Dixon. "Wegovy's approval means we can now use both lifestyle and pharmacological intervention to reduce the heart disease risk associated with obesity."

9. Xolair for food allergies

What it is:Xolair (omalizumab) is an injectable biologic medication that can treat allergic reactions to foods, asthma, and other related health conditions. It works by targeting immunoglobulin E (IgE), a substance that drives the immune system's response to an allergen.

FDA approval date:February 16, 2024

How it's taken: Xolair is an injectable medication that can be given at home. It's injected under the skin once every 2 to 4 weeks. Your prescriber will determine your specific dose based on your IgE levels and body weight.

Why Xolair's approval matters

Xolair is the first medication that can lower the risk of allergic reactions to multiple foods after accidental exposure; it can be given to people as young as 1 years old with food allergies. In clinical trials, this included people allergic to peanuts and at least two of the following food items:

• Cashews

• Hazelnuts

• Walnuts

• Milk

• Egg

• Wheat

Xolair does this through its effects on IgE. A type of antibody, IgE plays a role in certain food allergies, potentially resulting in severe allergic reactions (anaphylaxis). And while people with food allergies try to avoid the foods that could trigger a reaction, accidental exposures can happen.

It's in situations such as these that Xolair could play a role. Xolair could make small, accidental exposures to these foods a far less risky situation.

10. Amtagvi for advanced melanoma

What it is:Amtagvi (lifileucel) is an injectable medication that treats advanced melanoma (skin cancer). It's a tumor-infiltrating lymphocyte (TIL) therapy - a type of immunotherapy.

FDA approval date:February 16, 2024

How it's taken: Amtagvi is infused into your vein a single time. You'll receive it at an authorized treatment center after you go through a series of preparatory treatments.

Why Amtagvi's approval matters

Amtagvi is the first medication to use TIL therapy technology. It's also the first cellular therapy that treats a solid tumor (a non-blood-related cancer). It's a big step forward, scientifically speaking. But it can also make a meaningful difference in the lives of those with advanced melanoma.

In an interview with GoodRx Health, Kim Margolin, MD, the medical director of the melanoma program for Medical Oncology at St. John's Cancer Institute, discussed how TIL therapy like Amtagvi (or lifileucel) works and how it differs from other treatments.

"Lifileucel is one form of what's broadly called adoptive T-cell therapy. You're taking the patient's own cells and you're manipulating them in such a way that they become even more active killers [against their cancer]. In the case of lifileucel, which is a form of TIL therapy, it's actually the tumor itself that's the source of the cells," Margolin explained.

According to Margolin, TILs are lymphocytes (a type of white blood cell) found inside the tumor that can recognize and attack specific cancer cells. TIL therapy involves taking a small piece of tumor, dicing it up, and growing the TILs in a laboratory. Then, they'd be infused back into the body after a brief course of chemotherapy. After the TIL cells are infused, the patient receives a few doses of interleukin-2, previously known as T-cell growth factor. This creates an "army" of killer cells that can travel directly to the tumor to fight it.

During a phase 2 trial (all patients treated with the same therapy), the percentage of people whose tumors shrunk down to undetectable or reduced by about half (objective response rate) with lifileucel was about 31%. This trial, however, included people who had received three lines of prior therapy, on average. Margolin noted that the response rate may look different for people who haven't received as much prior therapy.

"TIL cell response rates are somewhere in the ballpark of 30% to 40%," said Margolin. "But they may be as high as 50% in people who've had the least amount of prior therapy, whose tumors are likely to be the least resistant to prior therapy." Margolin compared this to response rates of about 30% with alternative treatments, namely combinations of ipilimumab (Yervoy) and nivolumab (Opdivo).

11. Dupixent for eosinophilic esophagitis in young children

What it is:Dupixent (dupilumab) is a biologic medication most often prescribed for asthma. But it can treat eosinophilic esophagitis (EoE) now, too. It works by targeting inflammation caused by specific immune cells in the body.

FDA approval date:January 25, 2024

How it's taken: Dupixent is an under-the-skin injection that's given every 2 weeks for EoE. The amount of medicine that you'll inject with every dose will depend on your body weight.

Why Dupixent's approval matters

In May 2022, Dupixent became the first FDA-approved treatment for EOE. EOE is a chronic medical condition caused by inflammation in the esophagus. It's associated with high levels of eosinophils and can affect people of all ages.

Dupixent was initially approved to treat EOE in people ages 12 and older. But it's now approved for children as young as 1 years old. This is important for giving young kids access to an effective inflammation-lowering treatment.

Good to know: Dupixent may be approved for chronic obstructive pulmonary disease (COPD) by late September 2024. If approved, it'll be the first biologic treatment for COPD. Dupixent is also being studied for hives. Results from the study are expected in late 2024.

A year in review: 2023's top 9 drug and vaccine approvals

Many medications and vaccines were approved in 2023. Here's a recap of some of the most notable ones.

1. Paxlovid for COVID-19

Paxlovid (nirmatrelvir / ritonavir) is an antiviral medication available for people with mild-to-moderate COVID-19 that are at high risk for severe illness. It was initially authorized for emergency use in late 2021. But fast forward more than 11 million treatment courses later, it obtained full FDA approval in May 2023.

Paxlovid has been the subject of frequent news coverage. Understandably, too. The National Institutes of Health (NIH) recommends Paxlovid as a first-choice treatment option for nonhospitalized adults and adolescents ages 12 and older. It's preferred over other options, such as Veklury (remdesivir) and Lagevrio (molnupiravir).

In an interview with GoodRx Health, Christina Madison, PharmD, the founder and CEO of The Public Health Pharmacist - a public health consulting firm - described the importance of Paxlovid's approval.

"Paxlovid has been a mainstay of COVID therapeutic management since its initial [Emergency Use Authorization] designation in December 2021. Having full FDA approval also helps us with hesitant patients not wanting to take a medication that isn't 'FDA approved,'" said Madison. "It's great news that this [medication] has gone through the rigorous approval process and has been found to be safe and effective."

2. Arexvy, Abrysvo, and Beyfortus for RSV

RSV often causes symptoms of the common cold. But for certain people - especially infants and older adults - RSV can be especially dangerous.

It's ideal to prevent RSV complications before they happen. In 2023, the FDA approved three new products that can prevent illness from this respiratory virus. Previously, the only RSV prevention option was a medication for certain babies called Synagis (palivizumab).

• Arexvy (RSVPreF3): This is the first FDA-approved RSV vaccine. Adults ages 60 and older can receive one dose for the 2023-2024 RSV season. In June 2024, it was approved for high-risk adults as young as 50.

• Abrysvo (RSVpreF): This RSV vaccine was approved a few months after Arexvy. Adults ages 60 and older and certain pregnant women can receive one dose for the 2023-2024 RSV season.

• Beyfortus (nirsevimab): This preventative medication reduces the risk of RSV-related hospitalizations and clinic visits. One Beyfortus dose is recommended for all infants younger than 8 months old and some toddlers younger than 19 months old.

Good to know: In October 2023, the CDC noted that Beyfortus availability may be limited in certain areas. Synagis is a good alternative for kids who are eligible for it.

3. Opill for contraception

Opill (norgestrel) is the first over-the-counter (OTC) birth control pill in the U.S. The FDA approved it in July 2023, and it's set to launch in early 2024. You'll be able to purchase this progestin-only pill at pharmacies, big box retailers, online, and more.

Accessibility is what makes Opill unique. Many people have trouble obtaining prescription-only contraceptives. In fact, one study found that as many as 33% of women encounter hurdles while trying to obtain birth control. Opill has the ability to significantly reduce this number.

Veronica Vernon, PharmD, who serves as an assistant professor and Vice Chair of Pharmacy Practice at Butler University, told GoodRx Health why Opill's approval offers such promise.

"This is a tremendous step forward in terms of increasing access to contraception. A prescription for contraception can be a significant barrier for some patients due to the time and cost involved in having a visit," said Vernon. "There is a wealth of evidence to support over-the-counter contraception. I hope this is the first of several OTC options. I advocate for insurance coverage of OTC contraception [too]."

4. Zurzuvae for postpartum depression

Up to 20% of women experience postpartum depression (PPD), or a significant period of depression shortly after giving birth. It can cause long-term harm for kids and mothers alike if it's left untreated.

Zurzuvae (zuranolone) is a notable step forward for efficient PPD treatment. This fast-acting antidepressant was FDA approved in August 2023. It can start making a noticeable difference in PPD symptoms in as little as 3 days. By comparison, traditional antidepressants can take weeks to work. And it's more convenient than IV medications for PPD that are time consuming to receive.

Sarah Gupta, MD, a board-certified psychiatrist, highlighted why Zurzuvae is important for maternal health outcomes, saying its speedy effects will make a big difference in PPD cases.

"Though we've had some success in the past treating PPD with therapy, off-label antidepressants, and Zulresso (brexanolone), the truth is none of these treatments are ideal. Therapy and antidepressants can take weeks or months to fully take effect," said Gupta. "And though Zulresso can work in as little as 3 days, it has to be given as an IV infusion over 60 hours in a supervised healthcare setting - which is a dealbreaker for many patients. It seems likely that Zurzuvae will be a total game-changer when it comes to postpartum depression and maternal outcomes. After all, we're looking at the very first FDA-approved oral treatment for PPD."

5. Leqembi for Alzheimer's disease

Most Alzheimer's disease medications can have a positive impact on thinking, judgment, and memory. But they don't necessarily target any underlying problems behind the condition. In some ways, they're like a bandage for a larger issue.

Leqembi (lecanemab) is a new advancement in Alzheimer's research. It's the first clinically backed Alzheimer's medication that targets a root cause of the condition. Compared to placebo, an infusion with no medication, Leqembi lowered the worsening of dementia symptom severity by an average of 27% over a 1.5-year period.

Data such as this contributed to Leqembi's full FDA approval in July 2023. A similar medication - called Aduhelm (aducanumab) - received accelerated approval back in 2021, but it has yet to demonstrate a real clinical benefit.

In an interview with GoodRx Health, Rebecca M. Edelmayer, PhD, the Senior Director of Scientific Engagement at the Alzheimer's Association, noted some of the real-life benefits of Leqembi.

"By slowing progression of the disease in the early stages of Alzheimer's, people may have more time to participate more fully in daily life and live independently. This means more time for a person to drive safely, accurately handle family finances, and participate in hobbies and interests," said Edelmayer. "[And] because Leqembi has received traditional approval for the treatment for Alzheimer's disease, it is covered by the Centers for Medicare and Medicaid Services (CMS) and many other insurers."

Edelmayer went on to say that in order to receive Medicare coverage, people will need to meet a few qualifications. According to CMS, they will need to be enrolled in Medicare, be diagnosed with mild Alzheimer's-related dementia - with documented evidence of beta-amyloid plaque on the brain - and have a physician who participates in a qualifying registry.

6. OTC naloxone for opioid overdoses

The fight against the opioid overdose epidemic has been a decades-long journey. There's still a long road ahead, but one bright spot has been naloxone. It's a life-saving medication that reverses opioid overdoses.

Naloxone was previously only available through your pharmacist or healthcare provider. This was a barrier for many people. Now, it's even more accessible with the FDA approving the first OTC naloxone products. OTC Narcan nasal spray was approved in March 2023, and RiVive followed suit in July 2023.

In an interview with GoodRx Health, Anita Jacobson, PharmD - a clinical professor at the University of Rhode Island College of Pharmacy and the director of the Community First Responder Program - provided some context when asked if OTC naloxone's approval is a big step forward for responding to the opioid epidemic.

"I think it is [a big step forward] for quite a few reasons. The first one is stigma reduction. The fact that people now see this as an OTC product, like ibuprofen, cough medicine, or any other product that they can buy, can really help to remove fear," said Jacobson. "People try to distance themselves from anything related to the opioid epidemic, understandably, because there's loss of life, it's scary, and they may have a loved one that they're worried about. [But its OTC approval] also allows us to distribute naloxone across state lines in a way that we could not do when it was prescription only."

7. Zepbound for chronic weight management

Zepbound (tirzepatide) is a new long-term option for managing body weight. It's a once-weekly injectable medication that shares the same active ingredient as Mounjaro, a popular Type 2 diabetes treatment.

Zepbound's effectiveness is what makes it stand out. In clinical studies, it helped people lose an average of 34 lbs to 48 lbs after 72 weeks (16.5 months). About a third of people taking a high dose lost up to 25% of their initial body weight, too. Zepbound does this by helping you eat less and feel full for longer.

Zepbound isn't approved for everyone, though. It's approved for adults who are considered obese (BMI of 30 or higher). It's also approved for adults who are considered overweight (BMI of 27 or higher) and haveanother weight-related health condition, such as Type 2 diabetes, high blood pressure, or high cholesterol.

Minisha Sood, MD, an assistant professor at the Donald and Barbara Zucker School of Medicine at Hofstra/Northwell, described to GoodRx Health how and when Zepbound can make a difference.

"From the patient perspective, this potent treatment 'finally' affords persons with obesity enough appetite suppression to result in consistent reduction in caloric intake - a challenge which persons with obesity generally do not feel able to surmount on a long-term basis," said Sood. "Patients treated with tirzepatide describe feeling 'freedom' around food, no longer feeling trapped in the cycle of caloric reduction followed by increased hunger and weight regain."

Sood went on to say that Zepbound stands out from similar medications because of not only its weight-loss results, but also how well it's tolerated by patients

"It's thus potentially useful for patients who do not respond well to semaglutide (Wegovy) or liraglutide (Saxenda)," she said. "In the current climate of medication shortages, it also affords those who have been struggling with lack of access to semaglutide or liraglutide an opportunity to receive incretin-based treatment for obesity."

8. Roctavian for hemophilia A

Roctavian (valoctocogene roxaparvovec) is the first gene therapy for adults living with severe hemophilia A, a genetic bleeding disorder. It causes uncontrolled and prolonged bleeding. Reducing the frequency and severity of these bleeds is a priority for everyone living with the condition.

Roctavian's effectiveness and safety data is catching the attention of the hemophilia A community. A one-time dose can reduce the number of bleeds that people experience per year by more than 50%. It can also significantly reduce the need for other maintenance medications. And while it has a high sticker price, some analyses suggest Roctavian is more cost-effective than competitor medications like Hemlibra (emicizumab).

Leonard Valentino, MD, is a hematologist and serves as President/CEO of the National Bleeding Disorders Foundation. He told GoodRx Health why exactly Roctavian's approval offers hope.

"Roctavian marks a groundbreaking leap in treating hemophilia A. Its FDA approval is pivotal for folks dealing with hemophilia A because it offers a one-time infusion aiming to address the condition," said Valentino. "This contrasts with the everyday or frequent treatments that can seriously disrupt someone's daily life."

Valentino also acknowledged, however, that there is still much to know about Roctavian when it's used long-term.

"While this therapy brings the promise of hope and freedom, it's essential to remember ongoing research is still evaluating its long-term effects," he said. "Considering gene therapy is a significant decision, continuous discussion with medical professionals and family members is crucial to weigh the potential pros and cons."

9. Casgevy and Lyfgenia for sickle cell disease

Sickle cell disease (SCD) is a genetic blood disorder. It's a painful and expensive health condition to live with, and it often requires frequent blood transfusions and routine maintenance medications.

In December 2023, the FDA approved the first two gene therapies for SCD. They're designed to be single-dose infusions that help adults and kids ages 12 and older with SCD avoid pain crises and health complications over time.

Casgevy - also called exagamglogene autotemcel (or "exa-cel" for short) - is one such therapy. In addition to its potential benefits for SCD, the way it works is notable too. Casgevy is the first FDA-approved medication to use CRISPR-Cas9 gene editing technology. This innovative medication can target and turn off a gene that causes SCD.

"We at the Sickle Cell Disease Association of America Inc. have tremendous hope in [Casgevy], a potential cure for people living with sickle cell disease that could change their lives for the better," said Lewis Hsu, MD, the chief medical officer of the Sickle Cell Disease Association of America.

Hsu went on to say that Casgevy's "clinical trials have shown remarkable success in reducing the severe pain of sickle cell disease. We recognize that getting broad access to this therapy and fully understanding risks and benefits will be the next big tasks to tackle. [But] overall, we're excited about what that means for people living with the condition."

Casgevy's approval shouldn't take attention away from Lyfgenia (lovotibeglogene autotemcel, or "lovo-cel"), the other gene therapy approved by the FDA in December. This new and promising gene therapy uses more traditional gene editing technology to help eliminate pain crises. It's expected to be available in early 2024.

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