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Ascendis Pharma of Denmark has gained its third FDA new drug approval in the last six years as the U.S. regulator has signed off on Yuviwel as a treatment for achondroplasia. Ascendis Pharma is spiraling upward by way of its TransCon platform. For the third time in six years, the Copenhagen-based biopharma has scored an FDA approval for a rare disease medicine that was developed with its “transient conjugation” drug delivery technology. The U.S. regulator has signed off on Yuviwel, a once-weekly injection to treat children with achondroplasia, a rare genetic disorder that causes dwarfism. The accelerated approval covers children age 2 and older who have open growth plates, which are areas where cartilage develops at the end of bones, allowing them to lengthen.By providing continuous exposure to C-type natriuretic peptide (CNP) receptors, Yuviwel can counter the effects of the fibroblast growth factor receptor 3 (FGFR3) gene, which causes the protein to become overactive and restrict growth. This continuous systemic exposure to CNP over the weekly dosing interval sets Yuviwel apart in the achondroplasia treatment landscape, according to Ascendis.“The approval of once-weekly Yuviwel is a major step forward in the treatment of children with achondroplasia, giving physicians for the first time the option of prescribing a once-weekly medicine backed by compelling efficacy and excellent tolerability data from three randomized, double-blind, placebo-controlled clinical trials,” Carlos Bacino, M.D., of Baylor University and Texas Children’s Hospital, said in a release.The three trials and up to three years of open-label extension data demonstrated the ability of Yuviwel to provide improvements in annualized growth velocity versus placebo.Achondroplasia is the most common form of short-limbed dwarfism, affecting between 1 in 10,000 and 1 in 30,000 live births and preventing adults from growing taller than four feet,10 inches. The condition can increase the likelihood of spinal cord compression and respiratory blockages early in life along with other muscular and neurological problems.The company said it expects to make Yuviwel available early in the second quarter of this year. In a conference call Monday morning, Ascendis said it will reveal the price in the coming days.The nod comes with a valuable pediatric disease priority review voucher, which can be used to speed the review of a future drug application."Most of the products we're developing are getting priority review, so I'm not sure that we actually need to keep it. I think we'll sell it," Scott Smith, Ascendis' chief financial officer, said in a lighter moment during the conference call.Yuviwel’s approval comes after the FDA delayed its decision on the medicine by three months back in November. Ascendis will compete with BioMarin in the indication. The California company gained approval for its CNP treatment Voxzogo in 2021. The daily injected medicine generated sales of $927 million last year, which were up 26% and accounted for 29% of the company’s revenue in 2025.BioMarin is working on a longer-acting version of Voxzogo, BMN 333, which has shown promise in a phase 1 trial and could be launched by 2030, the company has said. BMN 333 has shown area-under-the-curve (AUC) levels greater than three times those observed in other long-acting CNP studies, BioMarin said in August of last year, referring to Ascendis’ trials for Yuviwel. AUC is a pharmacokinetic measure indicating the total exposure to a drug over time.Ascendis’ current advantage with Yuviwel is its consistent, sustained release over its weekly dosing as opposed to Voxzogo’s daily administration.Last month, BridgeBio revealed the success of a phase 3 trial of its FGFR1-3 selective tyrosine kinase inhibitor infigratinib in children with achondroplasia. BridgeBio and its partner Kyowa Kirin plan to apply for approval of infigratinib in the second half of this year.Ascendis used its TransCon technology to develop Skytrofa, a long-acting growth hormone for children which was approved in the U.S. in 2021, and Yorvipath, a hormone replacement therapy for hypoparathyroidism, which scored an FDA nod in 2024.  Sales for Skytrofa reached 206 million euros ($242 million) last year, which were up 5% year over year. Yorvipath generated sales of 477 million euros ($561 million) last year, with 187 million euros ($220 million) coming in the fourth quarter."We expects Yuviwel revenue to be light this year but don't think that reflects on long-term value," analysts from ISI Evercore wrote in a note to investors. "We continue to believe our $1 billion CNP franchise peak is quite conservative."Evercore sees more sales potential for Yuviwel in Europe than in the U.S., citing sales results for Voxzogo. There are also other potential indications for the treatment. The company also is exploring the potential of Yuviwel in combination with other treatments.

With quarterly earnings underway, BioPharma Dive is providing a snapshot of some companies results and how theyre being received by investors. Today, were offering insight into the latest numbers from Neurocrine Biosciences, Alnylam Pharmaceuticals and Ascendis Pharma. Neurocrine again disappoints some investorsShares of Neurocrine Biosciences fell by over 10% on Thursday, wiping more than $1 billion in value from the brain-focused biotechnology company.The drop followed a Wednesday afternoon earnings report in which Neurocrine said net product sales in the fourth quarter and 2025 overall totaled $798 million and $2.83 billion. Those figures are respectively 29% and 22% higher year-over-year. Ingrezza, a drug for movement issues associated with Huntington's disease, accounted for more than 80% of overall sales. The remainder mostly came from Crenessity, a treatment approved in 2024 for a rare, genetic condition that can cause life-threatening shortages of crucial hormones.Brian Abrahams, an analyst at RBC Capital Markets, suspects a combination of factors may be concerning some investors. One is Neurocrine's guidance. The company expects that, over the course of this year, net sales from Ingrezza will be in the range of $2.7 billion to $2.8 billion. That might not be as high as shareholders had hoped. Neurocrine executives also recently disclosed plans to spend another $150 million building out the sales teams for those two leading products a decision that could be seen as bad for near-term margins.Additionally, Abrahams noted how some are worried about Crenessitys growth prospects, specifically with regard to the number of new patients starting on the drug. Neurocrine says there are roughly 20,000 people in the U.S. with that rare condition, congenital adrenal hyperplasia, and, across last year, about 2,050 began taking Crenessity. Sales figures indicate the final three months of 2025 averaged fewer starts, at 431, than previous quarters.Even so, Abrahams and his team see the fears as overblown. Though we acknowledge investor consternation around margins and Crenessity launch dynamics could persist ... we believe todays downside reaction will prove to be a compelling buying opportunity, he wrote in a note to clients.Ingrezza looks poised for a major beat, he added, and Crenessity should be fine [as] we have always anticipated some continued slowdown in new patient starts.Overall, we see much growth left in both key franchises, wrote TD Cowen analyst Phil Nadeau in his own client note.Alnylam bitten by its successA similar situation played out with Alnylam Pharmaceuticals. The company, which specializes in so-called RNA interference drugs, gave a preliminary look at fourth-quarter and full-year earnings last month, then released its official report Thursday. It recorded nearly $3 billion across all of 2025 and almost $1 billion over those final three months, reflecting increases of 81% and 121% compared to the same periods the prior year.Despite that performance, Alnylam shares had slipped around 4% by the time trading closed Thursday afternoon.Though the company sells half a dozen drugs, nearly 80% of its product revenue comes from Amvuttra, a treatment for the nerve damage and heart failure associated with a rare disorder known as ATTR. Alnylam has said this disorder, which has a couple subtypes, affects north of a few hundred thousand people worldwide. For 2026, the company expects net product revenue to reach $4.9 billion to $5.3 billion, with Amvuttra and another ATTR medicine, Onpattro, responsible for up to $4.7 billion of that sum.To Paul Matteis, an analyst at Stifel, the main debate among investors is whether Amvuttra can meet or beat this guidance, and whether Alnylam can keep pace finding new patients to get on the drug.Alnylam is somewhat a victim of its own success after two very strong quarters for Amvuttra, Matteis wrote. Yet, while some investors may have wanted even more from the latest earnings, the underlying fundamentals here still seem strong.To that end, Alnylam said Amvuttras approval last spring for those cardiovascular problems tied to ATTR was a primary reason why patient demand for the drug grew roughly 250% in the fourth quarter. The company estimates that, globally, around 80% of patients with this condition, which stiffens the heart and impairs its function, remain undiagnosed.The number of identified patients has, on average, grown 40% annually since 2019, according to Alnylam.There are simply many tailwinds for the mid-to-long term, even if short-term expectations had gotten ahead of themselves, Matteis wrote.Ascendis numbers overshadowed by competitorDenmark-based Ascendis Pharma also provided an early glance at earnings in January. Its full report, out Wednesday, detailed fourth-quarter product revenue of 240 million euros, or about $279 million. Full-year product revenue hit 684 million euros, with 70% coming from Yorvipath, a drug for a rare illness where the body doesnt produce enough of a hormone that regulates vital minerals.The results were in-line with analyst forecasts. Joseph Schwartz, of Leerink Partners, expects Yorvipaths momentum to continue throughout this year, and described Ascendis goal of the drug surpassing $1 billion in revenue as achievable.The companys share price, however, slumped 5% at markets open Thursday. Though the stock would rebound over the following hours, Ascendis earnings appeared to take a back seat to data from a rival.Early that morning, BridgeBio Pharma disclosed positive findings from a late-stage study evaluating one of its most advanced drugs an oral enzyme-blocker named infigratinib as a growth-inducing treatment for achondroplasia, the most common type of dwarfism. Ascendis has been testing a once-weekly injectable therapy in this setting, and could receive approval from the Food and Drug Administration before the end of February.With few surprises in Ascendis results, the update from BridgeBio served as bigger news and reinforced that this oral option creates competition, wrote Li Watsek of Cantor Fitzgerald.Yaron Werber, a TD Cowen analyst who covers Ascendis, acknowledged that BridgeBios drug appears solid, with clean safety. And, if approved, it will likely dominate the achondroplasia market as both an initial and second-line treatment.In Werbers view, the key to Ascendis being able to compete is securing strong data from a Phase 3 study of its TransCon CNP therapy combined with its already approved product for growth hormone deficiency. In earlier testing, Ascendis said this pairing, when used in certain people with achondroplasia, tripled the effectiveness seen when TransCon CNP was used alone.The combination should eventually become the standard-of-care in achondroplasia, Werber wrote. '