Bitopertin

Youtube /FDA Without naming a specific product, Commissioner Marty Makary referred to an investigational therapy, delivered surgically into the brain, that the FDA was “pressured” to approve even after finding no clinical benefit to patients. FDA Commissioner Marty Makary is standing by his agency’s drug review decisions amid recent controversial denials. “There’s always going to be two sides to a story,” Makary said Thursday morning in an interview with CNBC . “There are moneyed interests,” he continued. “I get it, if I were an investor and I put in $100 million into a product and you didn’t get the results that you like—it is disappointing.” His remarks were a response to criticism that one of his deputies, Center for Biologics Evaluation and Research Director Vinay Prasad, had been coming in late in the review process to change previous agency advice to a drug sponsor—ultimately resulting in a rejection despite the company having followed that advice. FDA FDA’s Prasad Weathers Personal Controversy, Internal Strife Amid Moderna Imbroglio Vinay Prasad, the FDA’s Center for Biologics Evaluation and Research head, is accused of interpersonal impropriety as pushback builds against his decision to reject Moderna’s influenza vaccine candidate. February 12, 2026 · 2 min read · Dan Samorodnitsky Read more The FDA’s initiative to publicly release complete response letters , Makary continued, is a step toward making the regulator’s rationale behind a rejection as visible as possible, though he conceded that the move has led to “a bit of an effort to find a bogeyman”—a reason that can account for a denial. For example, “There was a product where the researchers drilled a burr hole, literally a hole, in people’s skulls” to directly inject a drug candidate into patients’ brains, Makary said. “At the end of the randomization period, it was found no benefit, and yet this is one of the drugs that we were pressured to approve.” Makary did not name the drug candidate, but his comments seem to match uniQure’s gene therapy AMT-130. AMT-130, a treatment for Huntington’s disease, is delivered directly into the brain via a neurosurgical procedure. In November 2025, the FDA told uniQure that it “ no longer agrees ” that a Phase 1/2 program would be enough to support an approval. The biotech called it a “key shift” after the regulator had previously signed off on the study plan for AMT-130. “Consequently, the timing of the BLA submission for AMT-130 is now unclear,” uniQure said at the time. The biotech’s shares closed Thursday’s trading session at $17.11 apiece, a 31% crash from its previous closing price of $24.87. “To be clear, his specific commentary might/might not have been referring to AMT-130 since QURE never received an actual” rejection letter, analysts at Stifel told investors in a Thursday note. “There has been increasing political pressure on the FDA to be more flexible and one has to wonder if FDA leadership will remain the same during this current Trump term.” FDA Makary, Prasad Under Fire as FDA Turmoil Reaches President Trump Following the FDA’s refusal to review Moderna’s investigational mRNA flu vaccine last week, Commissioner Marty Makary faced questions from the U.S. president about the agency’s handling of vaccines. It’s a clear signal that the tension long brewing at the drug regulator has now gone all the way to the top. February 20, 2026 · 6 min read · Heather McKenzie Read more More broadly, Stifel noted that Makary’s “steadfast defense” of the FDA’s approach to rare disease could be “discouraging for those hoping that the agency can be convinced to reconsider recent negative decisions, in the absence of specific political pressure.” The rare disease space has suffered several high-pro in recent months, including Disc Medicine’s bitopertin for erythropoietic protoporphyria, REGENXBIO’’s RGX-121 for Hunter syndrome and Ultragenyx’s UX111 for Sanfilippo syndrome.

Plus, news about Boehringer Ingelheim, Sitryx Therapeutics, Argenx, Palvella Therapeutics, Vir Biotechnology, Larimar Therapeutics, Bristol Myers Squibb and SystImmune: 📉 AtaiBeckley releases Phase 2a data, stock falls: The company said its drug EMP-01, an oral R-MDMA program, achieved its primary goal of safety and tolerability in individuals with social anxiety disorder. On efficacy, patients saw their symptoms decrease by an average of 28.53 points on a composite score, compared to a 16.67-point reduction in those on placebo. The difference between the two arms resulted in a “one-tailed” p-value of 0.036. (Typically, p-values are two-sided.) AtaiBeckley’s stock $ATAI fell by as much as 17% in Thursday morning trading. — Max Gelman 🥏 Disc to refile bitopertin after Phase 3 study: The company announced it will wait to respond to the FDA rejection letter until a Phase 3 trial reads out, expected some time in the fourth quarter. Earlier this month, the FDA rejected Disc’s experimental drug bitopertin, developed to treat a rare blood disorder called erythropoietic protoporphyria, where patients experience significant pain when exposed to sunlight. Disc had been seeking accelerated approval, but will now aim for full approval with the full Phase 3 dataset. — Max Gelman 🤝 Boehringer Ingelheim licenses a preclinical program: The drugmaker will have access to Sitryx Therapeutics’ small molecule inhibitor program. Boehringer will be fully responsible for developing oral therapies for autoimmune and inflammatory diseases. Sitryx will receive a total of “more than $500 million” for the deal, alongside potential future sales royalties. – Reynald Castaneda 👁️ New Vyvgart data: Argenx said its blockbuster drug, already approved for generalized myasthenia gravis, succeeded in the first Phase 3 trial in people with an ocular form of the autoimmune disease. After four weeks, people on the drug experienced less double vision and drooping of the upper eyelids, and had a roughly 4-point improvement on an 18-point impairment scale compared to a 2-point improvement among those who got a placebo. (Patients had to score greater than 6-points to enter the study.) If approved, the company believes it could expand its US market by 7,000 patients – Ryan Cross 💰 Public offering roundup: 🇨🇳 Bristol Myers, SystImmune tout Phase 3 win in China: At an interim analysis in patients with triple-negative breast cancer, the companies’ drug iza-bren showed statistically significant improvements in progression-free survival and overall survival. The study was conducted in China. Iza-bren is a bispecific antibody-drug conjugate targeting EGFR and HER3. The drug is in an ongoing Phase 2/3 study in first-line TNBC in the US, which is expected to read out in 2028. — Max Gelman