Quality/GMPs

The authors discuss subjectivity in the ICH Q9 (R1) guidance document.

Understanding how to apply phase-appropriate GMPs is crucial for achieving successful regulatory approval.

EMA has validated two MAAs submitted by AstraZeneca and Daiichi Sankyo for datopotamab deruxtecan in two types of cancer.

The Data Analysis and Real World Interrogation Network, DARWIN EU, is planning to add 10 new data partners in 2024.

The agency has published final guidance documents regarding validation and development of analytical procedures.

The license holder should negotiate, educate, contract, and then oversee the transportation contractor, says Siegfried Schmitt, vice president, Technical at Parexel.

Steffen Thirstrup, chief medical officer, EMA, emphasizes the importance of transparency in investigating potential safety issues of CAR-T therapies, while highlighting the need to balance regulatory diligence with maintaining trust within the community and among stakeholders.

The agency has recommended granting marketing authorization to ALS treatment, Qalsody (tofersen), for adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.

Steffen Thirstrup, chief medical officer of the EMA, discusses manufacturing supply, drug shortages, commercial incentives, politics, and patient wellbeing.

Daiichi Sankyo is investing approximately €1 billiion (US$1.08 billion) to expand its Pfaffenhofen an der Ilm, Germany, site for ADC development and production.

FDA's Drug Safety Priorities FY23 describes the center’s key safety programs and activities involved in promoting and protecting public health.

FDA has approved Iovance Biotherapeutics’ Amtagvi (lifileucel) for treating patients with unresectable or metastatic melanoma.

Two public electronic catalogues will be available for real-world data sources and for real-world data studies.

An interview with Steffen Thirstrup, chief medical officer, European Medicines Agency (EMA).

The three non-commercial developers of ATMPs will benefit from enhanced support from the agency.

The guidance discusses how to timely notify the agency of changes in production of APIs and finished products.

NICE has recommended the use of AbbVie's Tepkinly as a treatment option for eligible adults with diffuse large B-cell lymphoma.

The agency will coordinate intra-regional medicines regulation to strengthen the African regulatory network.

In the ATMP space, CGTs are hitting their stride with unprecedented approvals in the past year alone.

The milestone approval of a gene-edited therapeutic paves the way for gene-editing technologies.

The final guidance provides specific recommendations for CMC, pharmacology, toxicology, and clinical study design for CAR-T cell products.

Validating computerized systems is required to demonstrate adherence to data integrity, says Susan J. Schniepp, distinguished fellow at Nelson Laboratories, LLC.

The immune-resetting peptide, developed by Revolo Biotherapeutics, received positive results from its Phase IIb study.

The final guidance provides recommendations for developing gene therapy products incorporating genome editing of human somatic cells.

Takeda has received FDA approval for HYQVIA, a subcutaneous immunoglobulin for the maintenance treatment of chronic inflammatory demyelinating polyneuropathy.