Children with solid tumors who relapse are being treated with the same chemotherapy they would have been given 40 years ago, as “there have been no major approvals for pediatric solid tumors,” Catherine Bollard, senior vice president and chief research officer at Children’s National Hospital, said at a June 5 FDA roundtable on cell and gene therapies (CGTs). The problem isn’t the science. Bollard said many groups are working on curative CGTs “for these children who have lost all other hope for survival.” The real gap is that “big pharma doesn’t see the business model because it’s a rare disease,” she added.
The U.S. FDA gave Sarepta Therapeutics Inc.’s rAAVrh74 viral vector, used in an investigational gene therapy for the treatment of limb-girdle muscular dystrophy, a step up, making it one of the first platforms to receive the agency’s platform technology designation.
Moderna Inc. once again emerged the winner in a court skirmish over claims that its COVID-19 vaccine infringed two Alnylam Pharmaceuticals Inc. patents. The U.S. Court of Appeals for the Federal Circuit issued a precedential opinion May 4, agreeing with a federal district court in Delaware that Moderna didn’t infringe the patents. For both courts, the decision was based on a single issue of claim construction.
A court decision blocking President Donald Trump’s reciprocal and trafficking tariffs was hardly a day old before the U.S. Court of Appeals for the Federal Circuit stepped in late May 29 to grant a temporary stay while it considers the administration’s appeal. The stay adds further uncertainty to the path ahead for drug and device companies.
Without convening the U.S. CDC’s Advisory Committee on Immunization Practices, Health and Human Services Secretary Robert Kennedy decided to bring the government’s COVID-19 vaccine recommendations in line with the FDA’s new “evidence-based” approach to the shots.
A former board member of Chinook Therapeutics Inc. and four others were charged May 22 in a 19-count indictment stemming from an alleged insider trading scheme that produced more than $600,000 in profits after the June 2023 announcement that Novartis AG was acquiring the Seattle-based Chinook in a $3.5 billion deal.
It’s been nearly five years since the U.S. FDA, under the first Trump administration, issued a final rule and guidance on state importation plans (SIP) for importing less expensive prescription drugs from Canada. Yet only one state, Florida, has an approved SIP. In hopes of adding to that number, the FDA is enhancing its drug importation program to ease the way for states and tribes to bring prescription medicines in from Canada, at Canadian prices.
The recent 8-1 adcom vote against the U.S. applicability of Genentech Inc.’s Starglo trial is being seen as a warning signal expanding beyond the confirmatory trial for Columvi (glofitamab) as a treatment for relapsed/refractory diffuse large B-cell lymphoma.
More telling than the U.S. FDA’s Oncologic Drugs Advisory Committee’s 4-5 vote May 21 on the overall benefit-risk of Urogen Pharma Inc.’s UGN-102 (mitomycin) is that the panel’s urology specialists and the patient representative all voted yes, saying the drug would be an important alternative to what is often a continuing cycle of surgery for patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer.
Five years after the COVID-19 pandemic gripped the world, the U.S. FDA is moving away from annual routine boosters for all children and adults. Instead of that one-size-fits-all regulatory framework by which it has granted broad COVID-19 vaccine marketing authorization for all Americans older than 6 months, the agency said it’s adopting a policy akin to that followed in Europe, which now restricts the vaccines to older adults and those at high risk for severe disease.
