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.
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Viridian Therapeutics’ Lumvoa is the first FDA-approved treatment for thyroid eye disease that includes data for both active and chronic forms of the illness.
Replimune’s resubmission for RP1 for melanoma comes after the departures of FDA leaders in place at the time of the drug’s first two rejections. The FDA expects to hold an advisory committee meeting in late July.
Policymaking at FDA has been anything but business as usual under the Trump administration, but former regulators cite the agency’s new investigational new drug pilot program as a sign of normalcy.
The approval of Ionis Pharmaceuticals’ Tryngolza for severe hypertriglyceridemia could spur “substantial growth” for the product, according to William Blair.
Gilead’s Trodelvy can now be used as a monotherapy and in combination with Merck’s Keytruda to treat certain patients with triple-negative breast cancer.
The FDA’s recently altered outlook on the evidence required for approval of rare disease drugs could have immediate benefits for companies including Skyhawk Therapeutics, Capricor Therapeutics and Biohaven.
Jubilant HollisterStier received a warning letter over problems that the FDA said necessitate an immediate and comprehensive assessment of the contract manufacturing organization’s global operations.
A few short days after announcing an FDA pivot on a separate asset, REGENXBIO is planning to test the agency’s apparent newfound rare disease outlook on another late-stage gene therapy.
A new report from RBC Capital Markets lists Biotechnology Innovation Organization chief executive John Crowley and military physician Heidi Overton as potential candidates for the role of FDA commissioner—in addition to the agency’s current acting leader, top food regulator Kyle Diamantas.
The FDA has so far secured 600 new hires and is looking for 1,600 more as interim leadership at the agency aims to rebuild the workforce and morale after more than a year of intense attrition.