Regulatory Oversight

The agency’s CHMP recommended drugs to treatments for hemophilia, two biosimilars for psoriasis, and two flu vaccines at their October meeting.

The agency has reconfirmed its recommendation to not give positive opinion to Translarna (ataluren) after a reexamination of the available data.

The draft joint network strategy for the EU until 2028 will be open for public consultation until Nov. 30, 2024.

Regulators, EMA and HMA, have published principles and recommendations on the use of LLMs, which are being increasingly used for daily tasks.

The agency is recommending that the market authorization for Oxbryta (voxelotor) be suspended so that emerging data may be reviewed.

Approvals were recommended for treatments of ovarian cancer, lung cancer, bleeding disorders, respiratory disease, and more.

The company said this new commitment to its facility in Germany goes hand in hand with its recently announced ReciPredict, an initiative intended to streamline the product development cycle.

What impact will the AI Act have on pharma and digital medicine?

Germany’s Medical Research Act allows pharmaceutical companies to keep drug prices confidential.

Innovative solutions are making personalized cell and gene therapies accessible to all.

PRAC is recommending that patients who use glucagon-like peptide-1 receptor agonists (GLP-1 RAs) consider the risk of delayed gastric emptying before undergoing surgery with general anesthesia or deep sedation.

This paper reflects upon the past 15 years of experience in the application of QRM and KM within the pharmaceutical GMP environment.

EMA’s CHMP gave positive opinions on a nasal delivery for epinephrine and for a first-in-class medicine to treat pulmonary arterial hypertension, among others, including a biosimilar for treatment of autoimmune diseases.

Diversity Action Plans are now required to be submitted by medical product sponsors after changes governed by the Food and Drug Omnibus Reform Act.

Yoni Tyberg, associate director of the Special Program Staff in the Office of New Drugs, provided an update on efforts to modernize CDER processes.

This alert follows a similar one from the European Medicines Agency in October 2023 amid a rise in demand for the diabetes medication that, in turn, created a shortage.

PRAC is reviewing the risk of secondary malignancies in patients treated with CAR T-cell medicines.

USP and FDA publish reports detailing drug shortage numbers and challenges through 2023.

CGT Catapult and CATTI aim to standardize advanced therapy manufacturing with new aligned training standards.

An ordered process describing calculations activities for compounded dosage forms is described.

After a review, the agency concluded there was a possible risk of cancer to those exposed to 17-hydroxyprogesterone caproate in the womb.

Determining E&L risk from single-use components can be used to build the level of extractable profiling and PERLs.

EMA’s CHMP recommended treatments for hemophilia A, colorectal cancer, hypertension, overactive bladder, and metastatic breast cancer.

This study reviews changes in in-country testing (registration testing, import testing) requirements and analyzes current trends. In the context of international harmonization of good practices and standards as well as the improved information exchange between national regulatory authorities, in-country testing is considered outdated and redundant in many cases.

A new draft guidance issued by FDA covers human- and animal-derived materials used in the manufacture of advanced therapy medicinal products.

The report covers the major achievements of the office during 2023, including notable drug approvals, publications, guidances, workshops, webinars, and meetings.

In the new draft guidance, FDA provides recommendations on cell safety testing of human-sourced allogeneic cells for sponsor companies.

The agreement, which will allow participants to share confidential information on medical and medicinal products, builds on the cooperation fostered between the two regulatory authorities during the COVID-19 pandemic.

The majority of PMCs/PMRs are proceeding according to schedule.

Regulations for mRNA products are evolving as the market for mRNA expands.