Science and medicine typically move in spurts and stumbles. But every once in a while, a genuine breakthrough does come along and substantially improve how doctors can manage or treat a particular health problem.
2024 has seen its fair share of these important advances, from novel therapies for debilitating genetic conditions to a pivotal vaccine-like medication for HIV. Here’s a rundown of this year’s drugs and innovations that are likely to be game-changers moving forward.
New weapons in the war against superbugs
Antibiotic resistance is one of humanity’s greatest public health challenges. Many disease-causing bacteria have steadily adapted to the most common drugs used to treat them, including those that cause urinary tract infections. In October, the FDA approved a drug designed to fight back against these hardy UTI bugs: Iterum Therapeutics’ Orlynvah.
Orlynvah is approved solely for certain UTIs caused by Escherichia coli, Klebsiella pneumoniae, or Proteus mirabilis bacteria—specifically UTIs that haven’t responded or aren’t expected to respond to other antibiotics. It’s the first antibiotic of its kind, combining a drug that extends the duration of antibiotics in the body with a compound from a subclass of antibacterials known as penems. Penems have shown promise in treating a wide variety of commonly resistant germs, but Orlynvah is the first oral penem to be approved.
A steady pipeline of innovative treatments is essential to effectively combat antibiotic resistance, given how quickly bacteria evolve in response to new drugs. But drugs like Orlynvah, so long as they’re carefully managed, are a crucial weapon in our everlasting battle against superbugs.
A groundbreaking drug for schizophrenia
In September, the FDA approved Bristol Myers Squibb’s Cobenfy—the first truly novel drug for schizophrenia seen since the 1950s.
Over the years, scientists have created less harsh antipsychotics, but these drugs have all worked on the same basic principle of targeting the neurotransmitter dopamine in our brain. Dopamine isn’t the only neurotransmitter involved in schizophrenia, however, and simply tweaking this one lever often isn’t enough to keep people’s symptoms in check.
Cobenfy is the first drug for schizophrenia to use a new mechanism of action, by specifically targeting the neurotransmitter acetylcholine. The drug is actually a combination of two medications: xanomeline, which stimulates acetylcholine receptors in the brain, and trospium chloride, which is designed to block any off-target effects of xanomeline elsewhere in the body. In clinical trials, Cobenfy noticeably reduced people’s schizophrenia symptoms without causing major side effects.
As meaningful as Cobenfy will be for people with schizophrenia who haven’t responded to existing options, its value extends beyond that. Scientists and drug companies will now be motivated to develop other acetylcholine-based drugs for not only schizophrenia but other neurological conditions, such as Alzheimer’s disease.
A vaccine-like barrier against HIV
A widely viable cure for HIV is likely still many years away, if at all possible. But science has made incredible strides in both treating and preventing the infection, which used to be universally fatal. Antiretroviral therapy has made it possible for people living with HIV to have a normal life expectancy, for instance, often without having any risk of spreading the infection to others. People can also take versions of these drugs as pre-exposure prophylaxis (PrEP), substantially reducing their chances of catching the infection in the first place.
Today, PrEP is typically taken as a daily pill, though the FDA also approved an injectable PrEP treatment that is taken every two months in 2021 (Viiv’s Apretude). This past June, the future of PrEP became even brighter, with the released results of Gilead’s PURPOSE 1 trial, which tested out a twice-yearly injection of their antiretroviral lenacapavir (the drug is already approved to treat HIV). The Phase III trial found that twice-yearly lenacapavir performed just as well or even better than daily PrEP options at preventing HIV infections in cisgender women over a year’s time. The preliminary results from the PURPOSE II trial, reported last fall, also demonstrated the drug’s effectiveness among cisgender men, transgender men, transgender women, and non-binary people who have sex with men. Across both trials, twice-yearly lenacapavir was estimated to be over 99% effective at preventing HIV, with side effects similar to those of existing PrEP medications.
Given the clearly impressive findings, lenacapavir is almost certain to be approved as a new form of PrEP in the near future. While oral PrEP is highly effective at preventing HIV, it can be difficult for people to stick to the daily regimen needed for optimal protection. Some people can also face stigma and discrimination if they’re discovered to be taking or storing PrEP pills, experts have noted. So the vaccine-like nature of lenacapavir could provide a more convenient and safer option for preventing HIV, especially in parts of the world where HIV prevalence remains high.
Many outside scientists have already lauded the arrival of twice-yearly lenacapavir as the research breakthrough of the year. And just this week, Gilead announced that it would soon trial a once-yearly formulation of lenacapavir, which would make it even more like a typical annual vaccine (if everything goes well, this version could be out by 2027). So it’s likely this drug and others will help us push HIV ever closer to the brink of eradication—a now feasible goal that once seemed impossible.
As with past PrEP treatments, however, there remain important questions about how affordable and accessible the therapy will be once it’s available to the public.
The first ever medications for a life-shortening dementia
Niemann-Pick disease type C (NPC) is an incredibly rare but life-sapping genetic disorder, perhaps affecting around 1,000 people in the U.S. People with NPC are unable to move cholesterol and other lipids within their cells, causing it to build up and eventually damage many organs, including the liver, spleen, and brain. The progression and symptoms of NPC can vary, but often include dementia, and the median life expectancy of sufferers is currently only 13. In September, the Food Drug Administration approved the first-ever medications designed to treat NPC, just days apart: Zevra Therapeutics’ Miplyffa and IntraBio’s Aqneursa.
Both drugs, taken as a pill, were found to delay the worsening of people’s symptoms compared to placebo, but there are important differences between them. While Aqneursa is taken alone, Miplyffa will be prescribed alongside an enzyme. Miplyffa’s rollout also took longer, as the drug only became commercially available this month. It will take time to know whether these medications can significantly alter the course of people’s progression of their disease and to what degree. But given the lack of other options until now, these approvals are monumental.
A new era of hot flash treatment
In August, drugmaker Bayer published the results of two successful Phase III trials testing its experimental drug elinzanetant as a treatment for moderate to severe hot flashes in women over 40. By the end of the studies, women taking elinzanetant were noticeably better off than those taking a placebo, with over 80% of women on the drug seeing a more than 50% reduction in their symptoms.
The FDA still has to formally approve the drug, with a final decision expected by next July. But given the compelling results, an approval appears very likely. If it does happen, it would signal an important change in the treatment of this common and often disruptive condition. Elinzanetant would become the second non-hormonal drug of its kind to treat symptoms by affecting certain neurons associated with hot flashes—following the approval of fezolinetant in 2023—and the first to do so by blocking two key receptors. While hormonal therapy for hot flashes is safe and effective for most women, there are some unable or unwilling to receive it. So the more options available for this condition, the better.
Future possibilities
2024 has seen plenty of scientific breakthroughs, but there’s plenty more set to arrive in the coming years.
On the near horizon to look out for: the potential approval of Vertex’s suzetrigine, a novel, non-opioid treatment for moderate to severe acute pain; the start of phase III trials testing out new incretin-based drugs for obesity and diabetes, which could prove to be even better than the already blockbuster drugs semaglutide (Ozempic, Wegovy) and tirzepatide (Mounjaro, Zepbound), and the possible arrival of new and improved flu vaccines (including some meant to be combined with a covid-19 shot).
There are also potential innovations a bit further out that could have a tremendous impact in medicine if research continues to show promising results. Early trials this year have found that gene therapy can substantially restore the sight of people with certain inherited vision disorders, for instance. As of 2024, scientists have also begun to successfully transplant the organs of genetically modified pigs into living and terminally ill people, though, so far, these treatments have only extended lives by a few months. And we may even be able to someday help paralyzed people with certain kinds of spinal cord injuries to walk again using brain implants, based on early findings published this year.
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