EU MDR, FDA, CE Regulations

SHERIDAN, WYOMING – July 25, 2025 – As Q2 2025 earnings season unfolds, leading biotech firms face scrutiny amid a backdrop of regulatory headwinds, clinical trial disruptions, and evolving commercial strategies. Several key players are poised to reveal performance metrics that could reshape market sentiment across the pharmaceutical innovation landscape.

Sarepta Faces Scrutiny Amid Safety Setbacks and Strategic Pivot

SHERIDAN, WYOMING – July 25, 2025 – The U.S. Food and Drug Administration (FDA) has cast doubt over GSK’s attempt to return its antibody-drug conjugate Blenrep to the U.S. market for relapsed or refractory multiple myeloma, citing persistent safety concerns and limited evidence of efficacy across key patient populations.

SHERIDAN, WYOMING – July 25, 2025 – Roche has received CE Mark certification for its Elecsys® pTau181 blood test, marking a pivotal advancement in Alzheimer’s diagnostics by introducing the first IVDR-certified in-vitro diagnostic test to help rule out Alzheimer’s-associated amyloid pathology.

SHERIDAN, WYOMING – July 21, 2025 – Sarepta Therapeutics has entered a critical phase of uncertainty after reports surfaced that the U.S. Food and Drug Administration (FDA) is considering requesting a halt to all shipments of its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys. The potential move follows the disclosure of a third patient death associated with the company's underlying gene therapy platform.

FDA Scrutiny Intensifies After Third Death Linked to Platform

SHERIDAN, WYOMING – July 21, 2025 – GlaxoSmithKline’s ambitions to relaunch its antibody-drug conjugate Blenrep for multiple myeloma treatment faced a critical blow last Thursday, as the U.S. Food and Drug Administration’s Oncologic Drugs Advisory Committee (ODAC) rejected two proposed combination therapies over persistent safety and dosing concerns.

Safety Risks and Dosing Shortfalls Undermine Confidence

SHERIDAN, WYOMING – July 12, 2025 – A landmark release of 200 complete response letters (CRLs) by the U.S. Food and Drug Administration (FDA) has unveiled a decade of regulatory decisions behind once-rejected therapies, now approved and on the market. Among the most revealing cases are Eli Lilly’s Alzheimer’s treatment Kisunla, Sarepta’s Duchenne muscular dystrophy drug Vyondys 53, and Gilead’s long-acting HIV therapy Sunlenca.

SHERIDAN, WYOMING – July 8, 2025 – BrainStorm Cell Therapeutics is once again at the center of the amyotrophic lateral sclerosis (ALS) treatment debate as patients formally petition the FDA to reconsider the approval of its investigational stem cell therapy, NurOwn. Citing new survival data and expanded access outcomes, the Citizens’ Petition seeks regulatory reassessment of the once-rejected therapy, underscoring the shifting landscape of ALS drug development and patient advocacy.

Renewed Push for Regulatory Reevaluation

SHERIDAN, WYOMING – July 1, 2025 – The U.S. Food and Drug Administration’s recent decision to remove Risk Evaluation and Mitigation Strategies (REMS) from approved CAR T cell therapies marks a pivotal shift that could reshape access to these potentially curative treatments and drive significant market expansion across the cell and gene therapy industry.

SHERIDAN, WYOMING – July 1, 2025 – Neurogene has reached alignment with the FDA regarding the design of a registrational study for the investigational gene therapy NGN-401 for Rett Syndrome, enabling the company to convert its current Phase I/II study into a pivotal trial—a milestone that analysts describe as the “best-case scenario.”

SHERIDAN, WYOMING – July 1, 2025 – A groundbreaking collaboration that delivered a custom-made CRISPR therapy to a nine-month-old patient with CPS1 deficiency has ignited optimism that ultrarare diseases could soon be addressed on a broader scale, thanks to advances in delivery systems, precision gene editing, and regulatory pathways.