FDA
The approval of Tregzi—the first regulatory greenlight for Orca Bio—was based on a Phase 3 study in which patients on the therapy were twice as likely to survive without cancer relapse and without chronic GVHD compared with conventional allogeneic transplant.
FEATURED STORIES
A recent FDA reversal sparked new hope for patients with Huntington’s disease. Flying under the radar, Skyhawk Therapeutics revealed 12-month functional data from a midstage trial of its own candidate showing improvements on a key disease measurement scale.
The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
When the variance can’t be modeled, even disciplined biotech investors stop deploying. Here’s the cheapest fix for biotech’s investability problem.
Subscribe to ClinicaSpace
Clinical trial results, research news, the latest in cancer and cell and gene therapy, in your inbox every Monday
THE LATEST
While requests by government officials for anonymity when speaking to the media are nothing new, the practice attracts more scrutiny when the Department for Health and Human Services has pledged a commitment to “radical transparency.”
After the FDA’s first-ever public listening meeting on data-sharing in the cell and gene therapy space, new draft guidance aims to standardize the practice. But recent decisions call into question whether shared evidence and prior knowledge will accelerate development in rare diseases.
Among the unreported adverse events potentially linked to Ozempic are two deaths and one case of “completed suicide,” according to an FDA inspection report.
Capricor Therapeutics’ deramiocel was rejected in July 2025, potentially caught between Nicole Verdun, a former top biologics regulator at the FDA, and outgoing Vinay Prasad, director of the Center for Biologics Evaluation and Research.
The U.S. Senate has a plan to improve drug development for rare disease patients. The exit of controversial CBER chief Vinay Prasad will help clear the path.
Rare disease biotech stocks pop on the news that Vinay Prasad, the FDA’s chief biologics regulator, will depart the FDA at the end of April; Sen. Ron Johnson launches an investigation into recent rare disease drug rejections; and Roche and Zealand’s amylin analog fails to match investor expectations—and Eli Lilly’s rival candidate—in a mid-stage trial.
The senator, who has long advocated for expanding access to experimental therapies, reportedly called the FDA’s request for a sham surgery–controlled Phase 3 trial for uniQure’s Huntington’s disease gene therapy “bureaucratic idiocy.”
Industry and FDA representatives have reached a general agreement on planned pre-submission facility meetings but have expressed different views about the specifics.
The FDA’s cancer advisors will discuss AstraZeneca’s application for the oral SERD camizestrant in breast cancer and the AKT inhibitor Truqap in prostate cancer.
Some biotechs that had seen regulatory setbacks under Center for Biologics Evaluation and Research director Vinay Prasad experienced stock bumps Monday morning. Under Prasad’s leadership, the rare disease space has suffered a series of controversial rejections.