Novo Nordisk fired the first salvo in the next battle for obesity drug supremacy by launching an oral version of its popular Wegovy medication on Jan. 5. But its lead on chief rival Eli Lilly is likely to be short, as a similar, oral GLP-1 drug Lilly calls orforglipron is already under a faster-than-usual FDA review.

An approval decision should come in just weeks. Commissioner Martin Makary has granted orforglipron one of the agency’s new national priority vouchers, which drastically shorten review times by spurring the FDA to take an all-hands, interdisciplinary approach for experimental drugs that fit with the Trump administration's health agenda.

Awarding orforglipron the voucher may have removed a key competitive edge for oral Wegovy, as the first-mover advantage Novo enjoys may be trimmed from months to mere weeks if Makary’s controversial promise of one- to two-month reviews for beneficiaries comes to pass. And though the two drugs appear similarly effective in helping people with obesity lose weight, orforglipron has a couple other benefits that could boost its market share.

One possible edge is that, unlike with oral Wegovy, people who take orforglipron don’t have to fast for 30 minutes afterwards. Another is that as a chemical, or “small-molecule” drug, orforglipron is simpler and cheaper to manufacture than a peptide like Wegovy. And with the price deals cut between the two obesity drug manufacturers and the White House, Novo’s flexibility to negotiate a preferred position on private insurance formularies may be more limited than it has been with other metabolic medications.

Some analysts and investors have been skeptical of the data supporting orforglipron, particularly in obesity, where its weight loss effects haven’t matched injectable GLP-1 drugs. Still, it’s widely viewed as a future blockbuster that’ll further cement Lilly’s dominant position treating weight loss. Leerink Partners analyst David Risinger in late 2025 projected orforglipron sales will reach $20 billion in 2030. — Jonathan Gardner

Five years ago, Disc Medicine hatched a new plan for a drug Roche had unsuccessfully tested against schizophrenia. In a deal worth up to $200 million, Disc licensed rights to the therapy, bitopertin, and turned it against a rare and potentially fatal inherited condition called erythropoietic porphyria. In 2022, Disc went public to support the effort. 

Fast forward to 2026 and Disc is now worth nearly $3 billion and bitopertin potentially on the verge of its first approval. In a pair of studies called Aurora and Beacon, bitopertin improved markers of disease severity and lowered levels of a light-sensitive molecule that toxically builds up in patients’ red blood cells. The results made bitopertin, like Lilly’s orforglipron, the beneficiary of a new FDA program promising extra-speedy drug reviews.

It’s a “potentially game changing product,” the agency’s Deputy Chief Medical Officer Mallika Mundkur, said on an FDA podcast in October.

The award put Disc on the path to a regulatory decision by the end of January. However, a published report in Stat News in December suggested a fast approval isn’t a given. According to the report, the FDA requested more time to review bitopertin and Vinay Prasad, a top deputy of Commissioner Martin Makary, expressed skepticism about the drug’s efficacy. The report has depressed Disc’s stock price, though multiple Wall Street analysts remain optimistic about bitopertin’s approval chances. 

The drug “clearly checks off” many of the requirements of that FDA program, including “addressing a large unmet medical need,” wrote Jefferies analyst Roger Song. — Ben Fidler

Back in 2021, the FDA approved for the first time a medicine for children with a type of dwarfism known as achondroplasia. Developed by BioMarin Pharmaceutical, Voxzogo is designed to overcome a genetic mutation and spur bone growth. It remains the only achondroplasia treatment on the market, and now accounts for more than a quarter of BioMarin’s revenue.

But that monopoly may end soon, as the FDA is set to issue a verdict by the end of February on a rival therapy from Ascendis Pharma.

That therapy, “TransCon CNP,” works similarly to Voxzogo, but is given as a once-weekly injection rather than a daily shot. Analysts expect this dosing schedule to win over patients and help Ascendis take market share.

BioMarin has tried taking legal action to stop TransCon CNP from entering the market, though those efforts have yet to pan out. European regulators last year revoked a patent for Voxzogo, leading BioMarin to withdraw its infringement claim against Ascendis. Meanwhile, a decision from The United States International Trade Commission might not come until early 2027, according to Li Watsek of the investment firm Cantor Fitzgerald.

In a recent note to clients, Watsek wrote that his team sees the pending U.S. case as a “high hurdle to clear” for BioMarin, since it must “establish the validity of the patents, infringement of the patents, and pulling [Ascendis’ therapy] from the market is in the interest of the public.” The ruling in Europe also suggests “there might not be a high probability of success.”

The FDA had previously planned to make a decision on TransCon CNP by Nov. 5, but pushed back the deadline after Ascendis submitted more information on post-marketing requirements. The agency considered those additions a “major amendment” to the approval application.  

“[W]hile this delay will give BioMarin one more quarter to shore up its position in the market, we do not think this reprieve changes the overall Voxzogo narrative,” wrote Christopher Raymond, an analyst at Raymond James.

BioMarin recorded $654 million in Voxzogo revenue from January through September last year. — Jacob Bell

Rhythm Pharmaceuticals is already worth more than six-times the market value it debuted at nearly a decade ago. Shares could continue their ascent if the Boston-based biotechnology company’s top drug, a once-daily shot called Imcivree, wins a key additional clearance by a March 20 decision deadline. 

Rhythm already markets Imcivree for two rare, genetically driven forms of obesity. Since its initial 2020 approval, sales have steadily grown, topping $130 million in 2024 and reaching about $138 million over the first nine months of last year. But Rhythm is eyeing much broader use, starting with a type of obesity driven by injury to the hypothalamic region of the brain. 

In clinical testing, Imcivree helped people with this “acquired hypothalamic obesity” lose about 20 percentage points more of their body weight than placebo recipients. Those results surpassed Wall Street expectations and opened up what analysts believe to be a more than $1 billion market opportunity.

On a conference call in September, executives said that, through market research, they’d already identified some 2,000 “diagnosed or suspected” U.S. patients and expect the total number to be north of there to be at least 10,000. Their condition is also distinct from general obesity, driven by a defect that causes “rapid, uncontrollable” weight gain and that can, in this case, be addressed by a targeted therapy, noted Leerink’s Faisal Khurshid, in a report following the discussion. 

Rhythm’s path to approval hasn’t been clear-cut, however. The FDA in October called for additional analyses of the efficacy data in Rhythm’s trial. Those results were deemed a “major amendment” to the company’s application and delayed by three months the agency’s verdict. Multiple companies under current FDA leadership have seen surprise changes in guidance and slipping regulatory timelines, raising at least some concern about Rhythm’s application. 

Still, the delay is “likely just a minor speedbump on the road to large success,” asserted Khurshid, in November, noting that the drug’s “efficacy profile remains strong.” — Ben Fidler

Rocket Pharma was once one of the industry’s most valuable gene therapy companies. Sporting a market capitalization north of $3 billion in 2021, Rocket was advancing a pipeline of “ex vivo” treatments built from patient stem cells as well as “in vivo” therapies administered via simple infusions. 

Like many of its peers, though, Rocket has since had a rough run. Manufacturing issues led the FDA to reject one of its treatments in 2024. Safety concerns then slowed a closely watched program for a rare heart condition, leading to layoffs, a pipeline restructuring, and a decision to pull a planned application for a blood disease treatment. The myriad setbacks have sent shares once worth over $60 spiraling to less than $4 apiece. They’ve also “undermined confidence in execution,” wrote Leerink’s Mani Foroohar, last November. 

Rocket has a chance to turn things around in 2026, though. The treatment the FDA turned back in 2024, for a rare and severe immune disorder called leukocyte adhesion deficiency-I, could finally be approved by March 28. Called Kresladi, that treatment has shown in testing the potential to keep patients alive, out of the hospital, and dramatically reduce the rate of bacterial and fungal infections they struggle to fight off. 

Rocket recently prioritized its “in vivo” gene therapies over more complex treatments like Kresladi. Still, an approval could help the company re-establish needed momentum. Should Kresladi win an FDA clearance, Rocket would receive a “priority review” voucher that could be sold for nine figures, extending its ability to operate beyond the second quarter of 2027. That prospective funding is seen as “key” to supporting development of the rest of Rocket’s gene therapy pipeline, which is led by the once-stalled cardiovascular program that’s now in pivotal testing, Foroohar wrote. 

That cardiovascular program, for what’s known as Danon disease, could unlock a more than $1 billion market opportunity for Rocket. Analysts at William Blair have estimated that Kresladi, by comparison, could generate $294 million peak yearly sales. — Ben Fidler