Quality Systems

FDA granted accelerated approval to Skysona (elivaldogene autotemcel), which is designed to slow the progression of neurologic dysfunction in adolescents with CALD.

Regulatory officials are addressing challenges in ensuring that clinical data can be relied on in making regulatory decisions.

Choosing low-adsorption glass vials helps ensure detection accuracy.

Otsuka announced that the EC has approved Lupkynis as the first oral treatment for active lupus nephritis.

EMA has recommended approval of Enjaymo for the treatment of hemolytic anemia in adult patients with cold agglutinin disease.

The White House hopes to expand US modern biotech manufacturing and reduce reliance on foreign sources of bioindustrial materials.

WHO has awarded prequalification to GSK’s Mosquirix, an approved malaria vaccine.

FDA is in jeopardy of losing its fees for assessing and approving new drugs and medical products.

Lynparza has been approved in Great Britain as an adjuvant treatment for patients with germline BRCA-mutated HER2-negative high-risk early breast cancer.

Sanofi’s Xenpozyme (olipudase alfa-rpcp) is now the only approved treatment for acid sphingomyelinase deficiency.

The patent applications are for programs concerning lupus, idiopathic pulmonary fibrosis, and chronic kidney disease.

A new survey has demonstrated that remote audits, brought to the fore thanks to COVID-19, are not necessarily a preferred option for clients.

A Post Approval Change (PAC) to replace identity (ID) testing of incoming liquid drug substance with ID by visual inspection is considered a low risk provided questioned listed in this example have been answered favorably. In that case the PAC can be downgraded from a prior approval to a notification to health authorities, and managed in the PQS with immediate implementation effect.

Innovation is driven through a strong digital backbone.

An integrated approach to the data lifecycle is key to successful digital transformation.

Creating building blocks for good manufacturing practices is essential.

It is argued that orthogonal quantitative methods can be used to establish primary working standards. An example details how to calculate the expanded measurement uncertainty (U) for the certified assay value by considering two orthogonal assay methods.

The IQ Working Group has defined a pragmatic risk-based approach to audit trail review, where it is only required for high impact GxP data.

FDA has issued an emergency use authorization for both the Pfizer-BioNTech and Moderna bivalent COVID-19 boosters.

Change of lot or extension of reference standard shelf-life is considered low risk and should be downgraded from prior-approval to being managed in the Pharmaceutical Quality System (PQS) only based on a science and risk-based approach.

EMA has announced a change of plan for the IDMP.

Pharma loses battle to block price negotiations, but implementation faces many challenges.

A new unitary patent system is due to come into effect in Europe later on this year.

A process flow diagram can help create an agreed-upon process flow for a third-party standard operating procedure, says Siegfried Schmitt, VP Technical at Parexel.

A Post Approval Change (PAC) in size of thermal shipping solution used for transport of product is considered a low risk provided it has been qualified and temperature monitored, and therefore can be downgraded from a prior approval to a notification to health authorities, and managed in the PQS with immediate implementation effect.