Regulatory Authority Actions

The draft ICH Q9(R1) document details the importance of quality risk management principles.

The new draft guidance describes a standards recognition program for regenerative medicine therapies.

The European Commission has granted conditional marketing authorization to the Janssen Pharmaceutical Companies of Johnson & Johnson for CARVYKTI (ciltacabtagene autoleucel), a cell therapy for treating multiple myeloma.

The agency’s Medicines Shortages Steering Group has adopted a list of critical COVID-19 vaccines and treatments so they may be monitored for potential shortages.

Roche has received approval from the European Commission for Lunsumio (mosunetuzumab), the first CD20xCD3 T-cell engaging bispecific antibody for treating follicular lymphoma.

FDA has approved GSK’s vaccine for the prevention of measles, mumps, and rubella in individuals 12 months of age and older.

EMA has been assigned a formal role in preparing for and managing future public health crises within the EU through the formalization of ad-hoc structures and processes that were employed during the COVID-19 pandemic.

FDA will use virtual site visits even after resuming active inspections.

Manufacturers are aligning with pharmacists and providers to blame high drug costs and limited patient access on pharmacy benefit managers.

FDA once again is taking steps to facilitate the import of less costly prescription drugs.

Dupixent (dupilumab), developed by Regeneron Pharmaceuticals in partnership with Sanofi, has received FDA approval for a new indication—treating eosinophilic esophagitis, a chronic inflammatory disease.

A new initiative aims to speed the approval of and access to new drugs for young patients around the world, while limiting the number of children needed for testing in clinical trials.

PTC Therapeutics has received a positive opinion from EMA for Upstaza for the treatment of AADC deficiency.

The final guidance addresses safety aspects of container and carton labeling design.

The agency is asking drug manufacturers to ensure a strong supply chain by developing risk management plans.

FDA has approved Mounjaro (tirzepatide) injections as a treatment to improve blood sugar control in adults with type 2 diabetes.

Pfizer and BioNTech’s COVID-19 vaccine has been granted EUA for booster doses in children five through 11 years of age.

The companies have reached an agreement with the European Commission to update their COVID-19 vaccine supply agreement.

The agency’s Center for Drug Evaluation and Research has launched the new program to increase development of treatment options for patients with rare diseases.

The draft guidance describes the benefit-risk principles used to conduct product quality-related assessments of CMC information submitted to FDA for assessment as part of NDAs, BLAs, or supplements.

The drug is the first immunomodulatory COVID-19 treatment approved by FDA.

FDA is limiting the use of Janssen’s COVID-19 vaccine to certain individuals.

Issues related to mifepristone prescribing and dispensing are emerging in the debate over legislation to reauthorize FDA user fees for drugs and medical products.

Janssen has submitted a marketing authorization application to EMA for the approval of a treatment for patients with HRR gene-mutated metastatic castration-resistant prostate cancer.

Purolite has received the Queen’s Award for Enterprise in the International Trade category.

Kapruvia has been approved by the European Commission for the treatment of moderate-to-severe pruritis in hemodialysis patients.

Members of Congress asked FDA about multiple contentious issues, including expedited approvals, vaccines for young children, orphan drug exclusivity, access to medical abortions, conflicts of interest, and hiring initiatives.

FDA granted Quanterix’s neurofilament light chain plasma test a breakthrough device designation as a prognostic aid for relapsing-remitting multiple sclerosis.

VYDURA has been granted marketing authorization by the European Commission for both acute treatment of migraine and prophylaxis of episodic migraine.

Moderna has filed for emergency use authorization of its COVID-19 vaccine for use in young children six months to under six years of age.