EU MDR, FDA, CE Regulations

SHERIDAN, WYOMING – August 12, 2025 – The U.S. Food and Drug Administration (FDA) has significantly tightened prescribing criteria for bluebird bio’s Skysona, an autologous hematopoietic stem cell-based gene therapy for cerebral adrenoleukodystrophy (CALD). The treatment is now limited to patients with no other therapeutic options or available stem cell donors, following post-marketing findings of a substantially higher incidence of hematologic malignancies than previously reported.

SHERIDAN, WYOMING – August 11, 2025 – Boehringer Ingelheim has secured FDA accelerated approval for its kinase inhibitor Hernexeos (zongertinib), marking the German pharmaceutical company’s first entry into the oncology market in its 140-year history. The approval positions Hernexeos as a new targeted therapy for adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) harboring HER2 tyrosine kinase domain (TKD)–activating mutations.

SHERIDAN, WYOMING – August 10, 2025 – Gilead Sciences is signaling strong confidence in its HIV prevention portfolio, including the twice-yearly injectable PrEP therapy Yeztugo, despite growing uncertainty over the future of the U.S. Preventive Services Task Force (USPSTF). The federally recognized panel’s recommendations currently mandate insurance coverage for preventive services, but a postponed meeting has fueled speculation that Health Secretary RFK Jr. could overhaul or dismantle the group.

SHERIDAN, WYOMING – August 10, 2025 – In a rapid reversal, Dr. Vinay Prasad has resumed leadership of the U.S. Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) less than two weeks after stepping down. The reinstatement, confirmed by the Department of Health and Human Services (HHS), ends speculation over the agency’s direction during a critical period for advanced biologics oversight.

FDA Leadership Transition Reverses Course

SHERIDAN, WYOMING – August 9, 2025 – The cell and gene therapy (CGT) sector stands at a pivotal moment. Once buoyed by multibillion-dollar valuations, companies like Sarepta Therapeutics and bluebird bio have faced sharp declines, underscoring the sector’s commercial growing pains. Industry leaders now agree: sustainable success will depend on advancing manufacturing, aligning global regulations, and better communicating value to payers.

Next-Generation Manufacturing as a Growth Engine

SHERIDAN, WYOMING – August 8, 2025 – In a move aimed at reducing the nation’s dependence on overseas pharmaceutical production, the U.S. Food and Drug Administration (FDA) has launched PreCheck, a two-phase program designed to speed regulatory review and strengthen domestic drug manufacturing capacity.

SHERIDAN, WYOMING – July 29, 2025 – In a swift regulatory reversal, the U.S. Food and Drug Administration (FDA) has lifted its recommendation for a voluntary hold on Sarepta Therapeutics’ gene therapy Elevidys for ambulatory Duchenne muscular dystrophy (DMD) patients—restoring immediate commercial viability for the treatment and stabilizing investor confidence after weeks of uncertainty.

FDA Reinstates Ambulatory Access Following Brief Hold

SHERIDAN, WYOMING – July 29, 2025 – The American Medical Association (AMA) has issued a public plea urging Health Secretary Robert F. Kennedy Jr. to preserve the U.S. Preventive Services Task Force (USPSTF), following reports that the panel could be dissolved due to ideological concerns. The potential shakeup, while not yet finalized, has sparked widespread concern across the healthcare community and could significantly impact access to preventive care services nationwide.

SHERIDAN, WYOMING – July 29, 2025 – Sarepta Therapeutics is once again under regulatory spotlight following the death of an 8-year-old boy in Brazil who had received its gene therapy Elevidys, triggering an FDA investigation and fresh market turbulence for the embattled biotech.

SHERIDAN, WYOMING – July 25, 2025 – Genentech, a member of the Roche Group, announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its supplemental Biologics License Application (sBLA) for Columvi® (glofitamab-gxbm) in combination with gemcitabine and oxaliplatin (GemOx). The application sought approval for this regimen as a second-line treatment for patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are ineligible for autologous stem cell transplant.