Jazz Pharmaceuticals plc is prepped and ready for launch following U.S. FDA accelerated approval of dordaviprone as the first systemic therapy for H3 K27M-mutant diffuse glioma. Branded Modeyso, the orally administered first-in-class imipridone was acquired via Jazz’s acquisition earlier this year of Chimerix Inc. in a deal valued at about $935 million.
Cancel culture continues at the U.S. Department of Health and Human Services (HHS) as it observes National Immunization Awareness Month with another strike against vaccines. This time, a $500 million strike specifically targets 22 mRNA vaccine R&D programs at the Biomedical Advanced Research and Development Authority (BARDA), marking the beginning of the end of BARDA’s funding for mRNA vaccines.
After shutting down manufacturers’ efforts last year to offer the mandated 340B discounts on outpatient prescription drugs as a rebate rather than an up-front price, the U.S. Health Resources and Services Administration (HRSA) is now inching the rebate door open for drugs that were selected for the first round of Medicare price negotiations.
Agios Pharmaceuticals Inc. said the patients’ deaths tied to anemia therapy Pyrukynd (mitapivat) that were announced in a securities analyst’s Aug. 4 note to investors hasn’t changed the drug’s already established benefit-risk profile on the drug’s U.S. prescribing information.
Vertex Pharmaceuticals Inc.’s bad news from a phase II pain study and separately on the regulatory front caused shares (NASDAQ:VRTX) to close Aug. 5 at $393.83, down $78.44, or 16%. Vertex rolled out top-line results from the phase II dose-ranging study to test the safety and efficacy of its NaV1.8 pain signal inhibitor, VX-993, in tackling acute pain after bunionectomy.
The other shoe dropped on the U.S. CDC’s Advisory Committee for Immunization Practices (ACIP) when at least nine liaison organizations were informed by email late July 31 that they would no longer be involved in ACIP’s process of reviewing scientific evidence and informing vaccine recommendations.
Spine Biopharma Inc. cited an “inconsistent sham control response” among some of the sites in its phase III study testing SB-01 as an intradiscal treatment for patients with chronic low back pain associated with degenerative disc disease. The study, dubbed MODEL, fell short of statistical significance on pain intensity and pain-related function, despite showing numerical and clinically meaningful improvements.
While inflation in the U.S. is hovering below 3%, increases in some FDA user fees for fiscal 2026 are tripling that rate. PDUFA fees for branded prescription drugs and biological products will see a 9% hike come Oct. 1, and the increase in MDUFA fees will more than double the inflation rate with a 7% hike across the board.
Regeneron Pharmaceuticals Inc. disclosed within a positive second-quarter earnings report that it had received a complete response letter (CRL) two days earlier from the U.S. FDA for its bispecific antibody, odronextamab, for relapsed/refractory follicular lymphoma following two or more lines of systemic therapy.
The U.K. quoted biotech investment firm Syncona plc is facing into the volatile market conditions and the impact U.S. trade policy, regulatory uncertainty and pharma pricing are having on access to capital, with a plan to end its evergreen structure and transition to a closed fund.
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