Vosoritide

iStock, Jcomp A growing number of biotechs are entering the market with newly launched medicines, a pattern indicative of “improving capital markets,” according to Jefferies’ Andrew Tsai. The lion’s share of medicines on the market belong to Big Pharma—drugmakers worth tens of billions of dollars—but increasingly, smaller players are braving the commercial world. “We’re seeing an explosion in the number of new product launches by biotech companies,” Graig Suvannavejh, managing director and senior biotech and biopharma analyst at Mizuho Securities, told BioSpace in an email interview. The group has documented “great successes” across the board, though the industry’s track record is by no means perfect. “Launching drugs is really hard,” he continued, especially with payers “putting in place hurdles and restrictions, making market access to new drugs more challenging.” Still, there is much to be optimistic about. Many biotechs are “preparing better” for their product launches, building up not just their commercial capacities but also their understanding of patient, physician and payer needs, Suvannavejh said. There are larger macro factors at play, too, according to Andrew Tsai, managing director at Jefferies. “Broadly speaking, we think that the improving capital markets has helped,” he told BioSpace over email. Jefferies’ analysis of the trends this year so far “suggests that 2026 financing is on pace to reach an all-time high, implying that investors have an appetite to fund assets they think may have a competitive commercial profile.” A shift in investor attitude may have also helped, Tsai said, however cautioning that he has only anecdotally observed these changes. These days, investors seem to reward companies for early sales beats of recently launched products. This is in contrast to many years ago, when “it was common for investors to ‘short the launch,’” he explained, referring to the investing tactic of actively betting on a drug failing to perform well on the market. Here, BioSpace looks at five biotechs taking the commercial plunge to find out just how favorable the current markets are to smaller companies launching a drug. Big Pharma-partnered Protagonist launches two products Protagonist Therapeutics is having a big year so far. In March, the FDA approved the Johnson & Johnson–partnered IL-23 blocker Icotyde for moderate-to-severe plaque psoriasis, unlocking a market opportunity that Truist Securities estimates could reach $5–10 billion. Then, late last month, the biotech clinched another regulatory victory for the Takeda-partnered Mimrylo for polycythemia vera. The product could hit another $2 billion in peak sales, Jefferies projected at the time. Mimrylo’s uptake is too early to assess, but Icotyde has been performing well. During J&J’s second quarter earnings call in July, CEO Joaquin Duato said that Icotyde was off to “significant early launch momentum, with uptake accelerating and performance outpacing competitors at comparable points in their launches.” While the pharma at the time didn’t provide specific sales figures for Icotyde, Jennifer Taubert, worldwide chairman of J&J’s Innovative Medicine unit, told investors that there were already more than 18,000 prescriptions of the drugs for some 11,000 patients. Psoriasis revenues for Icotyde could reach $1.6 billion by 2028, according to Visible Alpha, a division of S&P Global Market Intelligence. Protagonist and J&J continue to develop Icotyde, with an eye toward expanding to other immunology indications. The partners are running two Phase 3 studies of the drug in psoriatic arthritis, the first of which is expected to read out later this year, according to the pharma’s Q2 presentation . Approvals Takeda, Protagonist score broad FDA nod in rare blood cancer, setting up $2B opportunity The approval of rusfertide—now to be branded Mimrylo—for polycythemia vera marks a key win for Takeda, which now has crossed the FDA finish line for two of three critical product launches slated for the coming year. August 31, 2026 · 2 min read · Tristan Manalac Read more Denali Therapeutics’ Hunter syndrome launch exceeds expectations In March, Denali Therapeutics’ enzyme replacement therapy notched an FDA approval for Hunter syndrome, delivering a much-needed win for the rare disease space that at the time had been battered by a series of negative headlines. Months later, the rare disease news flow remains mixed, but Denali’s drug has continued its victory lap. The therapy, called Avlayah, made $3.6 million in its first quarter on the market, the California biotech revealed in a second quarter earnings report last month. This pleasantly surprised analysts, who had projected $1.3 million in Q2 sales for Avlayah. Given the strength of Avlayah’s first three commercial months, Denali estimates net Avlayah revenues to hit $10–12 million in the third quarter, outpacing William Blair’s original forecast of around $6 million, according to an Aug. 7 note. The firm has now bumped that up to $10.7 million in Q3 and $29.3 million for the rest of the year. What makes Avlayah’s success all the more impressive is that the drug is going up against Takeda’s Elaprase, another enzyme replacement therapy that has a two-decade headstart on the market. Denali’s launch strategy is to entice patients on Elaprase to switch over to Avlayah—an approach that William Blair calls “underappreciated.” “Denali is well positioned and capitalized to drive switches from Elaprase,” the firm wrote on Aug. 7. By the late 2030s, Avlayah sales could surpass $250 million in the U.S. and reach $1 billion worldwide. Avlayah is the first new therapy cleared for Hunter syndrome in nearly 20 years and is the first medicine that directly addresses neurologic complications of the disease, the FDA said at the time of the product’s approval. Earnings Denali climbs past Avlayah expectations with $3.6M in revenue After launching in April, Hunter syndrome drug Avlayah surprised analysts with a strong first quarter, as patients rush to get access to Denali Therapeutics’ new option. August 10, 2026 · 2 min read · Annalee Armstrong Read more BridgeBio has upper hand over ATTR adversaries BridgeBio has more than tripled sales of the transthyretin amyloidosis (ATTR) therapy Attruby in a year. Attruby revenues hit $222.4 million in the second quarter , compared with $71.5 million during the same period in 2025, the company reported last month. Analysts expect the product will continue on this upward trajectory, hitting blockbuster status this year with sales exceeding $1 billion, according to Jefferies. Attruby, a drug that stabilizes the transthyretin protein and prevents pathologic misfolding, was approved for ATTR cardiomyopathy (CM) in November 2024. This gave BridgeBio a few months lead over a crucial competitor: Alnylam’s transthyretin silencer Amvuttra, which won regulatory clearance for the treatment of ATTR-CM in March 2025 . Alnylam, however, had a leg up as a familiar brand, since Amvuttra’s first approval came in June 2022 for ATTR polyneuropathy. But with BridgeBio entering the ATTR-CM market, Amvuttra sales underwhelmed, bringing in $1.01 billion worldwide in the second quarter, below the investor consensus of $1.05 billion. Alnylam has also lowered full-year revenue guidance for Amvuttra, from $4.4 billion to $4.7 billion down to $4.2 billion to $4.5 billion. There appear to be other factors going in BridgeBio’s favor, too. In July, Attruby—alongside the broader stabilizer class more broadly—received a boost when AstraZeneca and Ionis reported a late-stage fail for their antisense oligonucleotide Wainua, which did not significantly improve cardiovascular outcomes in patients with ATTR-CM. Like Alnylam’s Amvuttra, Wainua works by lowering the expression levels of transthyretin. Attruby, on the other hand, binds to the protein and prevents a crucial step in the formation of the disease-causing clumps. “Peak sales estimates for Attruby may edge higher” after the Wainua failure, Mizuho Securities wrote in a July 9 note. Cardiovascular disease AstraZeneca, Ionis’ Wainua woes send waves across ATTR-CM space The failure of AstraZeneca and Ionis’ Wainua in a late-stage study of ATTR-CM casts doubt on Alnylam’s next-generation candidate but is good news for others in the space, including BridgeBio and Intellia Therapeutics. July 10, 2026 · 3 min read · Tristan Manalac Read more Axsome’s Auvelity poised for $6B peak with Alzheimer’s agitation expansion Axsome’s Auvelity first hit the market in 2022 after being approved for major depressive disorder. But in May this year, the non-antipsychotic drug won a key expansion into Alzheimer’s disease agitation—one that Jefferies’ Tsai said could position Auvelity to “capture strong [first-line] market share” in the indication. Alzheimer’s disease agitation is a largely untapped market, with the only other FDA-approved option being Otsuka and Lundbeck’s antipsychotic Rexulti , Tsai said in an email to BioSpace . Auvelity has key edge over its competition because the product doesn’t have a boxed warning flagging a heightened risk of death in older patients, he said. This safety advantage “incentivizes adoption by all parties,” Tsai said. Auvelity also doesn’t come with weight gain, anxiety and motor problems, all typical of antipsychotic medications. Following the launch in Alzheimer’s agitation in June, “we think Auvelity should reach $6B combined peak sales, split between” this indication and major depressive disorder, Tsai told BioSpace. The expansion was granted despite mixed data in January 2025 that showed no significant advantage to Auvelity over placebo in terms of agitation scores, the primary endpoint of the late-stage ADVANCE trial. But the drug met its primary endpoint of lowering relapse risk in the Phase 3 ACCORD-2 study. Approvals Axsome wins first non-antipsychotic FDA approval for Alzheimer’s agitation Alzheimer’s disease agitation could mean peak sales of over $2.1 billion for Axsome’s Auvelity, according to analysts at William Blair. May 1, 2026 · 2 min read · Tristan Manalac Read more Despite patent hump, Ascendis continues achondroplasia ascent The FDA signed off on Ascendis’ Yuviwel in March, but months earlier, in October 2025, BioMarin had already started bracing for impact. BioMarin markets Voxzogo, which was approved for achondroplasia in November 2021 and for years reigned as the only therapy for the disease on the market. With competitive pressures on Voxzogo’s horizon—from Yuviwel and other achondroplasia products in development—BioMarin in October abandoned a goal of hitting $4 billion in revenue in 2027. So far, Yuviwel seems to be living up to investors’ high expectations. In the second quarter, Ascendis reported €8 million ($9.3 million) in sales for Yuviwel, which as of July 31 had reached 220 patients in the U.S. since the April launch. “Early uptake is strong,” H.C. Wainwright wrote on Aug. 27. “Voxzogo built a blockbuster market that Yuviwel only needs to convert,” the firm added. This could prove to be a relatively simple task for Ascendis, given Yuviwel’s convenience edge. While Voxzogo is given daily , Yuviwel is a weekly injection . “Yuviwel has a stronger competitive pro Voxzogo,” Leerink Partners said in an Aug. 18 note. “Weekly-versus-daily is a genuine convenience edge over BioMarin.” Last month, however, BioMarin was able to eke out a victory against Ascendis in a settlement agreement that gives BioMarin 18% to 20% royalties on net Yuviwel sales through May 20, 2030. BioMarin in early 2025 filed a complaint alleging that Ascendis infringed on a European patent for Voxzogo. The settlement will allow BioMarin to “participate economically in Yuviwel’s commercial success,” William Blair wrote on Aug. 31, but the market is still likely to shift in Ascendis’ favor. Rare Diseases Ascendis Readies BioMarin Challenge in Achondroplasia While Others Search for Root Cause Therapies from industry leaders BioMarin and Ascendis Pharma supply a key hormone that promotes bone growth. In order to move the field forward, challengers are looking to address the underlying cause of the rare, genetic disease. October 13, 2025 · 7 min read · Tristan Manalac Read more

BioMarin is pumping the brakes on yet another indication for its CNP analog, Voxzogo. The rare disease biotech announced that it would discontinue efforts to develop Voxzogo for children with Noonan syndrome, a genetic disorder that can affect height, facial features and the heart. BioMarin said the decision to discontinue was not based on the safety or efficacy of Voxzogo, but instead related to the “feasibility of the study and the current treatment landscape for children with Noonan syndrome,” according to a securities filing. Voxzogo is already FDA-approved to treat achondroplasia, a form of dwarfism. And BioMarin filed for an indication expansion with the FDA earlier this month based on strong phase 3 results for the drug in children with hypochondroplasia, a milder form of dwarfism. But Noonan syndrome is not the first indication trimmed from the Voxzogo pipeline this year. Earlier this spring, BioMarin discontinued dosing and enrollment in phase 2 trials testing Voxzogo in three other indications: Turner syndrome, SHOX deficiency and aggrecan (ACAN) deficiency. At that time, BioMarin said the choice to stop the initial trials was based on reports of possible adverse effects, namely slipped capital femoral epiphysis (SCFE) events, but trials investigating Noonan syndrome would continue as planned. The new decision to further refine Voxzogo’s pipeline strategy likely points to the drug’s changing competitive landscape. In February, the FDA approved Ascendis’ Yuviwel to treat achondroplasia. The rival treatment offers logistical advantages, requiring only weekly injections compared with daily injections of Voxzogo. The growing competitive pressures have been significant, with analysts at Leerink Partners saying that BioMarin’s success in the new hypochondroplasia indication may not be enough to offset competitive losses due to Yuviwel. BioMarin did manage to recoup some of those losses last month. In a deal to end patent disputes in five countries where BioMarin alleged that Yuviwel infringed on its intellectual property, Ascendis agreed to pay BioMarin a 20% royalty on U.S. net sales and an 18% royalty on net sales in the European Union, Brazil and South Korea. In exchange, BioMarin agreed to dismiss pending proceedings and grant Ascendis a broad license to patents related to Yuviwel and navepegritide products. In August, BioMarin upped its expected 2026 revenue from Voxzogo to $1 billion to $1.05 billion, citing strong patient demand and adherence. The company’s stock slipped just under 3% after it announced the discontinuation of the Noonan syndrome program.