New and notable treatments from 2025

Another year, another batch of new treatments coming to market.

The year 2025 may have been a bit of a roller coaster, but along the way, we still picked up a few new drugs and medical devices with major potential.

But what were some of the most impactful treatments of the year? Read on to find out.

Custom gene editing

This year brought the world’s first-ever recorded customized gene edit tailored to a single patient, courtesy of a team at the Children’s Hospital of Philadelphia and Penn Medicine.

The infant patient, KJ, received CRISPR gene editing for a rare, disabling, and often deadly genetic disorder known as CPS1 deficiency. His doctors detailed the treatment in a May New England Journal of Medicine article.

KJ got his first dose of the multidose treatment in February. It’s too soon to say how many doses will be needed, per the New York Times.

In October, some of the scientists who worked on KJ’s treatment announced in an American Society of Human Genetics article they’re planning a clinical trial to bring personalized gene editing to patients with other diseases.

There are about 25 million to 30 million people in the US living with 7,000+ different rare diseases, of which only about 500 have approved treatments, per the National Institutes of Health (NIH).

Stat reported some scientists hope this is a step toward revitalizing the gene editing industry, though others worry it may cost too much to be realistic for everyone.

The cost has not been disclosed. The therapy was funded through grants from entities including the NIH, biotech Acuitas Therapeutics, global life sciences and diagnostics company Danaher, and Danaher subsidiaries Integrated DNA Technologies and Aldevron.

Journavx (suzetrigine)

On Jan. 30, the FDA approved the first nonopioid painkiller in over 20 years.

The medication, Journavx (suzetrigine), is manufactured by biotech Vertex Pharmaceuticals, whose stock jumped about 6% the morning after the announcement and has risen about 8% year to date, as of Dec. 2. Two large randomized controlled trials found the drug eased moderate to severe pain compared to a placebo and at a “similar” level to a combination pain medicine of hydrocodone and acetaminophen.

But unlike hydrocodone and other opioids, suzetrigine is considered nonaddictive because it works on nerves outside the brain rather than actually entering the brain, per Vertex.

In 2024, 16.8% of people living in the US aged 12 or older (around 48.4 million) had a substance use disorder, according to the Substance Abuse and Mental Health Services Administration. Since 1999, 800,000+ people have died from opioid overdoses, including both prescription and illegal forms.

This nonaddictive drug comes with a big price tag, though, at a list price of $15.50 per 50mg tablet. Without insurance, 30 Journavx pills cost over $479 at major pharmacies compared to under $30 for 30 pills of generic hydrocodone/acetaminophen, per drug marketplace GoodRx.

What’s more, uptake has been slower than expected. The drug made $20 million in revenue in Q3 2025, falling $3 million short of expectations.

Yeztugo (lenacapavir)

On June 18, the FDA approved Gilead Sciences’s Yeztugo (lenacapavir), the first HIV prevention method that requires only a twice-yearly injection.

It had a nearly 100% success rate in clinical trials. Leaders in HIV research and treatment have hailed it as a potential “game changer” compared to the current go-to pre-exposure prophylaxis, or PrEP, method of taking a daily pill, which people sometimes have trouble accessing or remembering to take.

About 41 million people across the world were living with HIV at the end of 2024, per the WHO.Some have questioned whether the pill will reach everyone who needs it, however.

The US Department of State and the Global Fund for HIV, Tuberculosis, and Malaria committed in September to buying 2 million doses to donate to low-income countries. Gilead has also committed to providing the drug without profit for up to 2 million people “until generic manufacturers are able to support demand,” according to a Nov. 18 release.

Gilead’s stock is up about 35% year to date, as of Dec. 2.