Regulatory Oversight and Compliance

The seven chosen sponsors will help accelerate development of novel drugs and biologics for rare diseases.

Results of a Phase III trial showed that Tagrisso with the addition of chemotherapy reduced the risk of disease progression or death associated with EGFR-mutated non-small cell lung cancer by 44%.

Fruzaqla was previously approved for use in patients with metastatic colorectal cancer in the US in November 2023.

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

Sarepta Therapeutics received expanded approval from FDA for Elevidys in the treatment of DMD in non-ambulatory patients as ell as ambulatory patients.

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

FDA has approved argenx's VYVGART Hytrulo for a new indication, treating chronic inflammatory demyelinating polyneuropathy.

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.

Imfinzi (durvalumab) combined with chemotherapy decreased the risk of disease progression or death by 58% in a 700-patient trial.

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

The new meeting program will create a dialogue around AI and be administered through CDER’s Emerging Drug Safety Technology Program.

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

EMA is promoting the use of RWD in decision-making and has started a public consultation to review its draft recommendations.

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

FDA granted priority review to Sanofi's sBLA for Sarclisa in combination with VRd in treating transplant-ineligible patients who are newly diagnosed with multiple myeloma.

Bkemv (eculizumab-aeeb) is the first interchangeable biosimilar to Soliris (eculizumab) to treat paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.

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.

Purna Thakker, founder and CEO of ADPT Solution, discusses common issues that companies face with FDA regulations at CPHI North America.

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.

The IDAP pilot scheme is designed to accelerate access to innovative medical devices and is a pioneering legislative proposal in the UK medical device space.

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.

FDA has granted approval for the use of Novartis’ radioligand therapy, Lutathera, to treat pediatric patients with gastroenteropancreatic neuroendocrine tumors.

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